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Rebecca Hersher, NPR

April 20, 2017

The Food and Drug Administration says children under 12 should not be given prescription medicines that contain codeine or another narcotic, tramadol, and that such drugs can also be dangerous to youth between 12 and 18.

On Thursday, the FDA said it will require that prescription drugs containing codeine or tramadol carry a warning on the label against using them in children under 12 or in women who are breast-feeding. The agency cited evidence that the drugs could cause dangerously slowed breathing in some children, which could lead to death.

Multiple prescription drugs contain codeine or tramadol. For example, the painkiller Tylenol 3 contains acetaminophen and codeine. Drugs containing codeine already carry a black-box warning against using it to treat pain in children who have their tonsils removed.

“This is something we’ve been watching for several years,” says Douglas Throckmorton, the deputy director for regulatory programs at the FDA’s Center for Drug Evaluation and Research. “They are powerful, effective medicines when used right [but] they can cause a lot of harm when they’re not.”

The agency also warned against using the drugs in young people between 12 and 18 who are obese or have breathing problems such as sleep apnea or lung disease.

The new warnings did not further restrict over-the-counter medicines that contain codeine, such as popular types of cough syrup and medication marketed for cold and flu symptoms. The FDA recommends that parents talk to a doctor before giving their children such drugs and notes that colds and coughs in kids “are generally mild and go away in a few days, so they may not need to take any medicine.”

Thursday’s announcement was a partial acceptance of 2015 recommendations by an independent advisory committee that recommended the agency restrict prescription of codeine-containing drugs for children and also voted overwhelmingly against over-the-counter sale of codeine-containing cough syrup for children.

At the time, pharmacist Maria Pruchnicki, an associate professor at the Ohio State University College of Pharmacy, told NPR’s Rob Stein, “My concern, were I to be prescribing codeine in children, would be that I would, frankly, kill them.”

As Stein reported, the FDA is not required to follow the committee’s 2015 advice, although the agency generally acts in line with advisory committee recommendations.

In this case, the agency did act on prescription drugs but did not act on the over-the-counter recommendation.

It was unclear why more than a year passed between the committee recommendations and their partial adoption by the agency. In a press briefing, Throckmorton said the agency had received “additional information.”

Diana Zuckerman, president of the consumer group National Center for Health Research, called the move a “long-overdue step to protect our children”:

“The science is clear, so why did it take almost 18 months since the FDA’s public meeting on the exact same issue? FDA needs to do more to warn patients about the known risks of medical products as soon as data are available. A change in the label is important, but unfortunately many doctors and patients don’t read these labels. We need a public education campaign to warn parents and nursing women about these risks.”

In 2015, the FDA acknowledged that although tramadol is not approved for use in children, it is used off-label to treat pain in kids. In that warning, the agency noted that some people are what’s known as ultra-rapid metabolizers of tramadol and codeine. Those people have a variant of a liver enzyme that breaks down the drugs more quickly, leading to a rapid and potentially dangerous spike in the level of active opioids in the bloodstream.

The drugs can make their way into breast milk in nursing mothers and potentially harm infants, the FDA warns. The proportion of people with the enzyme variant, which is genetic, is thought to be between 1 and 10 percent.

“Because we can’t easily determine which children or nursing mothers specifically are at greater risk of ultra-rapid metabolism of codeine or tramadol, we are requiring manufacturers … to make important labeling changes to protect those children who are at the greatest risk,” explains Throckmorton.

As for future action on over-the-counter medicines that contain codeine, the American Academy of Pediatrics has made its position clear.

In a report published last year in the journal Pediatrics, members of the academy’s committee on drugs wrote that although over-the-counter cough and cold medicines containing codeine were available in 28 states and Washington, D.C., “neither the value of suppressing cough nor the effectiveness of codeine in children with acute illnesses has been shown.”

Regulators in Europe, Canada and Australia have all restricted the use of medicines containing codeine for children. According to the FDA’s public label database, more than 100 medications containing codeine are approved for sale without a prescription in the U.S.

Read the original article here.

Diana Zuckerman, PhD, National Center for Health Research

April 17, 2017

My friend Gwen went from being a healthy woman living a happy life to being diagnosed with a fatal cancer in a very short period of time. The doctors tried one Alzheimerstoxic chemotherapy after another. All were approved by the FDA, which means they had been proven to work for some patients. But none of them worked for Gwen, and she became a shadow of her former self, living her last months on earth in a limbo of a life, hoping one of these treatments would do some good.

The Goldwater Institute, a conservative think tank that promotes “Right to Try” legislation, has a different view of medical care for dying patients. In their fantasy world, experimental drugs that haven’t even been proven to work forany patients will save the lives of thousands of desperately ill patients, if only the U.S. government would mind their own business and get out of the way.

Unfortunately, this doesn’t make sense. If patients can’t be saved by drugs that are proven to work, what’s the chances that an unproven experimental treatment will save them?

Even more important, under current law all patients in the U.S. have the right to try experimental drugs that have at least some evidence of safety and effectiveness. So why lower the standard to include treatments with no evidence that they work at all?

Many people don’t understand how the current system works, and — understandably — want dying people to have another chance. As a result, Goldwater lobbyists have convinced more than 30 states that they should give patients the “Right to Try” experimental drugs. Those laws haven’t worked very well, however, and there is no evidence to show how many patients have benefited compared to how many have been harmed.

Now the Goldwater Institute is going a giant step further: they are trying to convince Congress to pass a federal Right to Try law that is much, much more dangerous than the state laws.

Here’s what the Goldwater Institute and their supporters are saying. Let’s set the record straight.

They say that the proposed federal law would merely give patients in all 50 states the rights that patients have in states with state Right to Try laws. NOT TRUE.

Under the state laws, companies can’t charge more for the investigational treatments than their “actual cost” to the manufacturer, because federal law prevents profits on experimental drugs. However, they are lobbying for a national Right to Try Act that specifically allows companies to determine what they will charge for their experimental drugs. That means desperate patients and their frantic loved ones can be exploited by scam artists and greedy companies selling unproven treatments.

They say that “promising new treatments” take more than a decade to be approved by FDA. NOT TRUE.

Effective treatments may take a decade to develop, but that is not the fault of the FDA.  The average drug is approved about a year after its pivotal studies are submitted to the FDA. An article in Cancer World concluded that the FDA approves drugs an average of 6 months faster than the European medical agency.

They claim that the law would greatly increase the number of patients gaining access to promising experimental drugs. They complain that in 2015, FDA only allowed 1,300 patients to have access to experimental drugs through their Expanded Access program. NOT TRUE.

The FDA is approving 99% of the applications submitted to them by doctors whose patients want to participate in the program. Only 1,300 patients gained access to experimental drugs because most doctors aren’t requesting it and the companies that are asked to make their drugs available are not always willing or able to do so. They may not have enough of the experimental drug available, or they may believe that the specific patient requesting the drug is more likely to be harmed than helped by the drug.

They even give an inaccurate example. The Goldwater Institute has stated that “most” of the 78 patients treated by an “oncologist” under the Texas Right to Try law “are doing very well.” NOT TRUE.

The doctor they are referring to, Ebrahim Delpassand, is a radiologist, not an oncologist, and he has not said “most are doing very well.” He testified before the Senate that the treatment “has helped many” of the patients, but he doesn’t say how many or to what extent the treatment helped.  He also hasn’t said how many were hurt by the treatment.  None of that information has been made public and the doctor has refused to answer questions from skeptical reporters.

They say “something is wrong” because “fewer than one-tenth of 1 percent of terminal patients can take advantage of the FDA’s compassionate use exception.” NOT TRUE.

Experimental drugs are just that: not proven to work or to be safe. Patients can die sooner and in agony from experimental drugs, especially those that have only gone through tiny, preliminary studies of a few healthy volunteers or patients (as the proposed national law would allow).

Only about 15% of the drugs that complete those types of preliminary studies are eventually proven to be safe or effective. The other 85% are either not safe, not effective, or both. That is a very important reason why most patients (and their doctors) do not seek experimental treatments even when they know they can.

Our patient protections are working. Major pharmaceutical companies are not lobbying for this legislation, but those who oppose FDA safeguards are. If you want to prevent the exploitation of desperate patients, contact your Senators and Congress and let them know.

This article originally appeared on Our Bodies, Our Selves. 

Emily Hartman and Langan Denhard


Many sellers of health foods and supplements claim that the acai (AH-sah-EE) berry will help you lose weight. Dieters beware: these claims are false. Ads promising weight loss have been spotted on popular social networking sites and search engines. Before you give out your credit card number, read on!

The acai palm produces a very small berry that is found in South and Central America. It has become popular in the United States over the last few years. While there are plenty of good things about the fruit, make no mistake-there are no special properties that make you suddenly or quickly lose weight. Acai does have antioxidants-a substance our bodies need-but no more than cranberries or cherries, and less than blueberries, pomegranate, and grapes.1

Here are some tips that have been scientifically proven to help you lose weight:

  • Exercise on a regular basis-most days of the week
  • Eat well-balanced, nutritious meals
  • Start each day off with breakfast
  • Get enough sleep (at least 7-9 hours)

It is important to remember that if a product claims fast weight loss, either the product is risky or the claims are false.  Federal regulators from the Federal Trade Commission filed temporary restraining orders in April 2011 against six online marketers who participated in the scam—the accused have all since ceased online advertising. More than simply advertising these false claims, the online marketers created websites that masqueraded as a legitimate news source. Recently, Contrave, a drug which was approved by the FDA for weight loss, was put on the 2017 “Watch List” for risks of losing consciousness. Sustained and healthy weight loss cannot be achieved by a pill!

Healthy, safe weight loss consists of losing no more than two pounds per week-that’s 7,000 fewer calories consumed than burned off each week, since one pound equals 3,500 calories.  Weight loss at the rate of .5 to 2 pounds per week is more likely to be permanent.  Several of the marketers promised buyers a weight-loss of 25 pounds in 4 weeks.  That’s the equivalent of 6.25 pounds, or 21,875 calories burned, per week. If acai really were a miracle weight loss product, there would be no obesity epidemic in the United States! So please, use your better judgment next time you are tempted to buy an acai weight loss product.

All articles on our website have been approved by Dr. Diana Zuckerman and other senior staff. 

1. Navindra P. Seeram, Michael Aviram, Yanjun Zhang, Susanne M. Henning, Lydia Feng, Mark Dreher, David Heber. Comparison of Antioxidant Potency of Commonly Consumed Polyphenol-Rich Beverages in the United States. Journal of Agricultural and Food Chemistry 2008 56 (4), 1415-1422.

Megan Polanin, PhD, April 5, 2017

Thank you for the opportunity to speak today. My name is Dr. Megan Polanin. I am a licensed clinical psychologist in Washington D.C. and a Senior Fellow at the National Center for Health Research. I previously trained at Johns Hopkins University School of Medicine. Our research center analyzes scientific and medical data and provides objective health information to patients, providers, and policymakers. We do not accept funding from the drug or medical device industry, and I have no conflicts of interest.

The development of opioids formulated to prevent abuse is a public health priority, and we support the FDA’s efforts to encourage the creation of opioid analgesics that deter abuse. The FDA states that a product that has abuse-deterrent properties means that “the risk of abuse is lower than it would be without such properties.” According to the FDA materials provided, it appears that RoxyBond is more abuse-deterrent compared with Roxicodone. However, there is still abuse potential for the intranasal and intravenous use of RoxyBond.

The studies about RoxyBond’s abuse are limited. In the laboratory setting, it appears to meet the FDA’s current standards for “abuse-deterrent.” Whether its abuse-deterrent properties are effective in the real world and whether Roxybond is a “better” drug are much more difficult questions that will require postmarketing data. We know from previous experience with opioids that the FDA has designated as abuse-deterrent that once this drug is on the market, it may be abused more widely than current laboratory studies suggest. That is exactly what happened with reformulated Opana ER, as several members of this panel are aware.

Compared with the FDA-approved extended-release/long-acting abuse-deterrent opioids, RoxyBond’s characteristics are similar regarding “drug liking” and “taking the drug again.” Thus, it does not appear more likely to be abused than extended-release/long-acting opioids.

Unfortunately, this comparison is rudimentary and less than ideal for several reasons. First, a direct comparison is impossible given a lack of sufficient information. Second, we are utilizing extended-release/long-acting opioids currently on the market as a comparison, which does not set a high standard.

The FDA’s guidelines state that a drug’s label should reflect and describe a product’s specific abuse-deterrent properties, such as an abuser’s ability to crush a tablet and extract the opioid. Thus, RoxyBond’s label should include its specific abuse-deterrent properties and clearly specify the potential risks of intranasal and intravenous abuse. Most important, the FDA should require opioids to have a black box warning indicating that, although the drug may be more difficult to crush or inject, it is still highly addictive.

Opioid addiction is an epidemic in the U.S. and labeling a drug as abuse-deterrent influences doctors, patients, and family members. Unfortunately, many doctors think abuse deterrent means an opioid is less addictive. To be part of the solution rather than part of the problem, the FDA should be diligent in analyzing whether this drug’s abuse-deterrent properties result in meaningful reductions in abuse, misuse, and related adverse clinical outcomes compared with Roxicodone once it is marketed to consumers. Although current data suggest that this drug will be less likely to be abused, abusers of the drug can be more creative or implement unique techniques to overcome those deterrents. Thus, sufficient follow-up is critical in order to determine if this is actually the case.

If approved with abuse-deterrent labeling, this will be the first immediate-release abuse-deterrent opioid, and it will likely be favored for prescriptions and will set a standard for future drugs to meet. Thus, it is important for this panel and the FDA to make approval decisions based on good science and strong data.

To reduce the opioid epidemic, the FDA must hold pharmaceutical companies to a high and truthful standard. We urge this Advisory Committee to advocate for patient safety by demanding that the FDA include labeling regarding RoxyBond’s specific abuse-deterrent properties as well as the specific routes of abuse that the product has been developed to deter. We also urge this Committee to recommend that, if RoxyBond is approved, postmarket studies should be required immediately to evaluate its use and abuse once it is on the market.

Sheila Kaplan, STAT News

April 5, 2017

Dr. Scott Gottlieb has been preparing for this job his whole life.

He’s a physician and a conservative policy wonk. He’s been a federal regulator scrutinizing new drugs. He’s a writer. An investor. And a consultant: He’s made a lot of money advising biopharma executives on how to get through the bureaucratic thickets of the Food and Drug Administration.

As his confirmation hearing opens Wednesday for the post of FDA commissioner, that resume is both Gottlieb’s strongest asset and his deepest vulnerability.

President Trump has made clear he wants to revamp the FDA to slash regulations and speed drug approvals. Gottlieb, 44, should be able to hit the ground running: He knows how new drugs are developed, tested, and reviewed. A cancer survivor, he also understands what such therapies can mean to desperate patients.

But if he wins confirmation, he’ll also soon be in a position to make or break the fortunes of many a biopharma executive he’s crossed paths with — or even worked for — in recent years. He’s promised to recuse himself when conflicts arise.

Still, Democrats are wary.

And CEOs are ecstatic. […]

The Trump administration named Gottlieb to head the FDA late on a Friday afternoon, with a bland press release that simply recapped the bare outlines of his resume. It didn’t include even a perfunctory quote of praise from the president — or, for that matter, from anyone in the administration. […]

The nomination drew quick praise from former FDA commissioners of both political parties, including Democratic appointee Dr. Margaret Hamburg and Republican appointee Dr. Mark McClellan.

“Scott has long been committed to finding better ways to advance science and improve health outcomes,” McClellan said. “He is deeply committed to getting these issues right.”

President Obama’s last commissioner, Dr. Robert Califf, praised the nomination, but also expressed some doubts. “It seems like the main question is, ‘Which Gottlieb are we going to get?’” Califf told Kaiser Health News. […]

Despite his promises of recusal, Gottlieb’s close ties to industry worry consumer advocates — a concern echoed by some Democratic senators.

“It is essential that the FDA commissioner be very knowledgeable about how the FDA works,” said Diana Zuckerman, president of the National Center for Health Research, an advocacy group.

“But when so much of a nominee’s career has been spent helping companies get their products approved, that creates the kind of perspective and conflicts of interest that can’t be recused away,” Zuckerman said. “It’s an integral part of who that person is.” […]

Read the full story here

Megan Polanin, PHD, April 4, 2017

Thank you for the opportunity to speak today. My name is Dr. Megan Polanin. I am a Senior Fellow at the National Center for Health Research, and I previously trained at Johns Hopkins University School of Medicine. Our research center analyzes scientific and medical data and provides objective health information to patients, providers, and policymakers. We do not accept funding from the pharmaceutical industry, so I have no conflicts of interest.

Like many public health experts and advocates, we are very concerned about treatments that are marketed and promoted for hangovers that contain either acetaminophen or aspirin. Both acetaminophen and aspirin have well-known health risks, particularly when consumed in conjunction with alcohol. Acetaminophen can cause liver damage, and aspirin can cause stomach bleeding. The FDA’s review points out that most research indicates that moderate alcohol consumption may be associated with a higher risk of acetaminophen-related adverse events, including liver toxicity.

And unfortunately, substituting other nonsteroidal anti-inflammatory drugs is not any safer.

Consumers are likely to take pills for hangovers shortly after, just before, or even during alcohol use. Since these are over-the-counter drugs, most consumers assume they are completely safe and are unlikely to read warnings about the risks. Adolescents and young adults may be at particular risk for the cumulative effects of binge drinking paired with analgesic consumption because they are even less likely than adults to read the label carefully or be cautious about avoiding possible adverse events. They are unlikely to realize that they are drinking heavily enough to be at risk.

We work closely with patients and consumers, and we know that many are unaware of all the ingredients in over-the-counter combination products that they use, whether they are for hangovers or colds. As the FDA noted, research indicates that most acetaminophen overdoses are unintentional or due to the failure to recognize the risks. For example, unintentional overdoses can occur when the consumer does not realize that acetaminophen is in a hangover medication and additionally takes acetaminophen or aspirin to treat or prevent a hangover.

FDA’s standard for drug approval is that the benefits of taking the drug for that indication must be greater than the risks. There is well-documented evidence citing the risks of consuming alcohol and these drugs around the same time. A person who has been drinking enough to expect a hangover or to already have a hangover is likely not in a condition to carefully read the label of an over-the-counter medication.

The FDA should base its decision on how to label antacid-analgesic combination drugs on scientific evidence, and scientific evidence clearly indicates that the combination of alcohol with these drugs can be dangerous. The bottom line: Medications should not be labeled for hangovers if they contain ingredients that can cause serious harm when taken before, during, or within a few hours after drinking alcohol.

These are the key issues that the FDA should address:

  1. Antacid-analgesic combination over-the-counter medications that are marketed and sold for hangovers should not contain acetaminophen or aspirin. Ideally, the label should clearly say that the reason they do not include these types of painkillers is because of the uncommon yet potentially very serious risks of combining them with alcohol. Antacid-analgesic combination drug labels should clearly indicate that acetaminophen or aspirin are active ingredients.
  2. All products with acetaminophen or aspirin should have warnings that they are risky to use for hangovers because of the risks of taking those analgesics before, during, or within a few hours after consuming alcohol. If it were possible to include a clear description of under what circumstances (e.g. number of drinks, length of time following alcohol consumption, etc.) alcohol increases the risk of these drugs, that would be best to include, but we don’t seem to have that information available at this time.
  3. The labels on aspirin and acetaminophen currently have warnings about their risks for individuals who consume the drug and three or more drinks every day, which implies the risks of long-term use of alcohol and these analgesics. The FDA should modify these warnings to also include one episode of heavy drinking. It is important to keep in mind, however, that many people do not consider 5 or even more alcoholic beverages in a short time to be binge drinking or heavy drinking.

Rather than warning consumers, the current labels for over-the-counter treatments for hangovers instead encourage consumers to use these treatments around the same time that they are consuming large quantities of alcohol.

We encourage you to strongly urge the FDA to focus on patient safety by removing the treatment of hangovers from the label of any medications containing aspirin and acetaminophen, because the risks outweigh the benefits. At the same time, we urge the FDA to issue a press release and host a press advisory phone call to publicize their concerns.


Diana Zuckerman, PhD, National Center for Health Research
Our Bodies Ourselves

March 28, 2017

Last week the media discovered that breast implants can cause cancer. Rather than causing breast cancer, experts now say that breast implants can cause a type of lymphoma (cancer of the immune system) called anaplastic large cell lymphoma (ALCL).

You’ll be excused for thinking this is news. The truth is that experts have known that breast implants cause ALCL since at least 2013, and some of the foremost plastic surgeons in the country were discussing it behind closed doors since at least 2010.

The U.S. Food and Drug Administration (FDA), which is responsible for making public information about the risks of medical devices, including breast implants, first published a report on its website about ALCL and breast implants in 2011. At that time, they said there was evidence that implants might possibly cause ALCL. The FDA’s report came months after anarticle published in Allure magazine stated that plastic surgeons and their medical societies were studying the possible link between breast implants and ALCL.

Articles subsequently published in medical journals concluded that breast implants cause ALCL. But despite the growing evidence, the FDA didn’t update its website to officially report that breast implants really can cause ALCL until last week. That’s when the media realized it was a real story.

If you think women should have been told this sooner, here’s what you need to know:

In May 2016, the World Health Organization published a report that included the term breast implant associated ALCL (BIA-ALCL). A few months later, the National Comprehensive Cancer Network (NCCN) released the first worldwide oncology standard for the disease. The guidelines (you need to sign up for a free account to see them) include a guided algorithm for surgeons and oncologists to test for and diagnose the disease. The authors conclude that any abnormal accumulation of fluid or a mass that develops near the breasts months after breast implants are  implanted must be evaluated.

They also state that even if the BIA-ALCL is confined to the scar capsule that surrounds the implant and even if that capsule is totally removed through proper explant surgery, the patient must be followed for 2 years to make sure the ALCL is eliminated.

Why didn’t plastic surgeons or the FDA make that information more widely available? I’m sure there are women and their doctors who would have benefited from that information in the last few months.

In 2015, plastic surgeons who had denied any link between breast implants and cancer for more than two decades published an article in a plastic surgery journal about 173 women with ALCL that was caused by their breast implants.

However, plastic surgeons across the country focused on reassuring women that BIA-ALCL is “very rare” and the FDA echoed that mantra.  But, although rare, it seems that BIA-ALCL is not “very rare.”  In Australia, which can track medical problems from any kind of implants better than the tracking of implants in the U.S., the Australian Department of Health estimates that BIA-ALCL affects as many as one in 1,000 women with breast implants.

The estimates of plastic surgeons and the FDA are much lower in the U.S., but there is no reason to think BIA-ALCL is less likely to develop in women in the U.S. than in Australia. Given the dramatic increase in BIA-ALCL diagnoses in recent years, it is clear that BIA-ALCL was under-diagnosed and under-reported for many years.

For women with ALCL, it doesn’t matter how rare it is. The sooner it is diagnosed, the more likely it can be cured easily by removing the implants and scar capsule surrounding it. At later stages, women will need chemotherapy and are less likely to survive, according to research conducted at the MD Anderson Cancer Center that was published in 2013.

The study followed women for 5 years and found that ALCL related to breast implants sometimes requires chemotherapy, and approximately 25% of the implant patients with the more serious type of ALCL died during the 5 years following their diagnosis. You can read more about the study here.

ALCL caused by breast implants can result in swelling, which is often mistaken for an infection and treated with antibiotics. Antibiotics are ineffective against ALCL and the delay in timely and appropriate treatment for ALCL is dangerous.

A published response in the same medical journal urged physicians to respond quickly and to check patients who have swelling near their implants for ALCL. This would require cytology testing rather than testing for bacteria.

This news is especially important to women who undergo mastectomies to prevent cancer or for DCIS or very early breast cancer, either of which is equally likely to be cured with a lumpectomy instead. Women trying to beat cancer by undergoing a radical surgery they don’t need are unlikely to do so if breast implants will put them at risk of developing a different type of cancer.

The news is equally frightening to cosmetic surgery patients. Many health insurance companies refuse to cover the cost of medical tests or treatment for women with breast problems related to cosmetic breast implants. We now know this can result in undetected ALCL, which can be fatal. In addition, delays in treatment for ALCL can be extremely expensive for patients and their insurance companies; the companies would be required to pay for treatment for ALCL when it is eventually diagnosed at a later stage.

Women deserve to know the facts.  And they deserved to know them years ago.

Read the full article here

Bronwyn Mixter, Bloomberg BNA
March 24, 2017

Over three-hundred class II medical devices made the list of those that might become exempt from premarket notification requirements once the FDA takes final action.

The Food and Drug Administration March 14 published a proposed list of class II devices that will be exempt from the requirements, also called 510(k)s, when the list is made final (82 Fed. Reg. 13,609). Class II devices are moderate-risk devices. A 510(k) clearance demonstrates that a device is substantially equivalent to another (or predicate) device already on the market. Clinical data typically aren’t generated for a 510(k).

The 21st Century Cures Act (Pub. L. No. 114-255), a 2016 law to speed new drugs and devices to market, required the FDA to publish the list. The FDA said the final list will decrease regulatory burdens on the industry by eliminating time and resources needed to submit 510(k) notifications. The devices include dentures, umbilical clamps, obstetrical forceps and surgical clamps.

Comments on the list are due May 15 (Docket No. FDA-2017-N-1129). The agency said it will consider the comments and determine whether the list should be modified before publishing the final version.

Safety Concerns

The National Center for Health Research, a nonprofit that encourages new and more effective medical treatments, is concerned these devices won’t be reviewed by the FDA.

“We are very concerned about this huge list of devices that will no longer go through any kind of FDA review,” Diana Zuckerman, president of the nonprofit, told Bloomberg BNA in a March 20 email. “The FDA’s faulty justification is that the FDA already knows quite a bit about these devices, but that ignores the fact that when a new company starts making a device, or when any company makes changes to a device, the device can become substantially less safe or substantially less effective, or both.”

Zuckerman said “it is very disturbing that the FDA’s Center for Devices is so focused on relieving device companies of the burden of providing evidence of safety and effectiveness, but not at all concerned that now doctors and patients have an impossible burden of trying to figure out which devices they can rely on and which they can’t. And if we are ever going to have a value-based reimbursement system for medical care, increasingly devices will not be covered because so often there is no scientific evidence that a specific device will benefit patients.”

List Longer Than Expected

Bradley Merrill Thompson, a Washington-based attorney with Epstein Becker & Green PC, told Bloomberg BNA in a March 20 email the FDA’s list is longer than he expected, which is good news because he was afraid the FDA would exempt just a few categories of medical devices. Thompson counsels medical device, drug and combination-product companies on a wide range of FDA issues. He’s also a Bloomberg BNA health-care advisory board member.

“The proposed list is pretty comprehensive, covering most areas of medical devices,” Thompson said. “The purpose of this exercise is both to save industry resources developing unnecessary premarket submissions, but also to conserve agency resources for those products that really need oversight.”

Thompson said the list “will help FDA focus its attention on those technologies that really need premarket review.”

The Advanced Medical Technology Association (AdvaMed) “is still reviewing FDA’s proposed 510(k) exemption list,” Janet Trunzo, senior executive vice president, technology and regulatory affairs at AdvaMed, said in a March 20 statement provided to Bloomberg BNA. “We commend the agency’s progress in implementing this important provision of the 21st Century Cures Act, which supports a risk-based review approach by creating a regular process for identifying lower-risk products appropriate for exemption.”

Read original article here.

Rita Ruben, Forbes

March 22, 2017

The Food and Drug Administration has received nine reports of women dying of a rare blood cancer years after getting breast implants, according to information the agency released Tuesday.

The FDA says it now agrees with the World Health Organization that such cases are linked to the breast implants and not some unfortunate coincidence. As of Feb. 1, the FDA says, it had received a total of 359 reports of breast implant-associated anaplastic large cell lymphoma (BIA-ALCL).

The FDA reports suggest that implants with a textured surface are more likely to be associated with the cancer than smooth implants—of the 231 reports that contained information about the implant’s surface, 203 were reported to be textured implants, while 28 were reported to be smooth. The Australian Therapeutic Goods Administration (TGA) analyzed 46 confirmed cases of BIA-ALCL, including three deaths, and none of the cases occurred in women with smooth implants.

BIA-ALCL on average is diagnosed about a decade after implant surgery, according to the WHO. The first reported case of a woman with breast implants developing ALCL was published in a 1997 letter to the journal Plastic & Reconstructive Surgery. While that case was a woman with saline-filled implants, the FDA says the filling, be it saline (salt water) or silicone, doesn’t seem to make much of a difference, although no well-designed studies have yet been conducted to settle that issue.

BIA-ALCL is rare, but just how rare isn’t clear. As the FDA notes, it medical device reports can’t answer that question, because they don’t represent all cases, and the denominator—the total number of women who’ve received breast implants—isn’t known.

ALCL is more common in the breasts of women who’ve had implants than in those who don’t have implants, in whom the cancer almost never develops in the breast. A U.S. studypublished in January concluded that over their lifetime, 3.3 women out of every 100,000 with textured breast implants will develop BIA-ALCL. But the TGA estimates that the disease is more common, affecting 1 in 10,000 to 1 in 1,000 women with breast implants (that agency says it has received no reports of BIA-ALCL in women with smooth implants).

“There is no reason to think it is less likely to develop in women in the U.S., and given the dramatic increase in diagnoses in recent years, it is clear that it was under-diagnosed and under-reported for many years,” Diana Zuckerman, a health advocate who has long questioned the safety of breast implants, told me.  Zuckerman serves as president of both the National Center for Health Research and the Cancer Prevention and Treatment Fund, nonprofits based in Washington, D.C. […]

Read the full article here.

Megan Polanin, Ph.D.

March 14, 2017

Thank you for the opportunity to speak today. My name is Dr. Megan Polanin. I am a licensed clinical psychologist in Washington D.C. and a Senior Fellow at the National Center for Health Research. I previously trained at Johns Hopkins University School of Medicine. Our research center analyzes scientific and medical data and provides objective health information to patients, providers, and policymakers. We do not accept funding from the drug or medical device industry, and I have no conflicts of interest.

The development of opioids formulated to prevent abuse is a high public health priority. Although the reformulated Opana ER was designed to prevent abuse by making it more difficult to abuse via intranasal or injection routes, the reality is very different. Compared with other opioids, reformulated Opana ER, along with its generic counterpart, had the highest injection abuse rates following reformulation.

The FDA states that a product that has abuse-deterrent properties means that “the risk of abuse is lower than it would be without such properties.” Instead of lowering the risk of abuse, however, the reformulation of Opana ER seems to have resulted in significantly increased rates of abuse via injection.

The term “abuse-deterrent” is not accurate for reformulated Opana ER because the drug is widely abused. The FDA’s guidelines state that a drug’s label should reflect and describe a product’s specific abuse-deterrent properties, such as an abuser’s ability to crush a tablet and extract the opioid. Despite the drug’s incorporation of physiochemical properties aimed at making it more difficult to abuse by intransal or injection routes, it is misleading to doctors, patients, and family members to say or imply that the drug is more difficult to abuse. In fact, the drug’s black box warning should be amended to more clearly specify the risks of injection abuse.

Compared to other types of opioid abuse, the injection of opioids is associated with increased infection risk. This risk is even greater because of Opana ER’s high potency and short duration, which results in more injections per day. In addition, the high cost of this drug can lead to equipment sharing. Individuals who injected the reformulated version have been especially likely to develop thrombotic microangiopathy (TMA). Abuse by injecting “melted” tablets resulted in an HIV outbreak in Scott County, Indiana. This drug is not only failing to deter abuse, but it is generating additional public health problems.

Opioid addiction is an epidemic in the U.S. and labeling a drug as abuse-deterrent which is actually widely abused would greatly contribute to the problem, by misleading doctors, patients, and family members.

To be part of the solution rather than part of the problem, the FDA should be more specific and accurate when claiming that a drug is abuse-deterrent. Research indicates that many physicians believe that a drug labeled “abuse deterrent” is less addictive. If a drug is crush-resistant, or difficult to crush in a specific way, it should be labeled as crush-resistant, not as abuse-deterrent. Only those drugs that significantly reduce the chances of abuse should be labeled as abuse-deterrent, and the reasons for that label should be clearly explained.

We strongly agree with the FDA’s 2013 denial of Endo Pharmaceuticals’ citizen petition to label Opana ER as abuse-deterrent, and we strongly urge the Advisory Committee to recommend that the FDA continue to deny this company’s requests to include abuse-deterrent labeling. To reduce the epidemic, the FDA must hold pharmaceutical companies to a truthful standard: Only abuse-deterrent drugs should have that label.

We also agree with the FDA’s 2013 denial of Endo Pharmaceuticals’ requests to take the original Opana ER off the market. This company has not proven that the original Opana ER poses an increased potential for abuse compared with reformulated Opana ER. We urge the FDA to continue to deny Endo’s request to withdraw the original Opana ER from the market for safety and effectiveness reasons.

We urge this Advisory Committee to advocate for patient safety by rejecting the companies’ requests and instead demanding that reformulated Opana ER have a stronger, more specific black box warning.

The joint Advisory Committee, composed of the Drug Safety and Risk Management Advisory Committee and the Anesthetic and Analgesic Drug Products Advisory Committees, voted on this question: “Do the benefits of reformulated Opana ER continue to outweigh its risks?” The Committee voted 18-8, with 1 abstention, that the benefits of this drug do not outweigh its risks. Read more about the meeting here.

Anna Edney and Robert Langreth, Bloomberg Politics

March 10, 2017

  • Nominee was senior FDA executive under George W. Bush
  • Choice represents mainstream pick for pharmaceutical industry

Scott Gottlieb, a former deputy commissioner of the U.S. Food and Drug Administration, is President Donald Trump’s choice to lead the agency, according to an emailed statement from the White House.

Gottlieb, 44, served in several senior positions at the FDA during the George W. Bush administration. He has talked extensively about how to lower the cost of prescription drugs by modernizing the agency’s approval process and speeding cheaper generic competitors to market.

Since leaving the FDA, Gottlieb has worked as an adviser to investment firms and as a fellow at the conservative-leaning American Enterprise Institute, a Washington think tank. He has been the drug industry’s preferred choice for the FDA job and has worked as a consultant to some of its companies.

Gottlieb must be confirmed by the Senate before he takes over at the agency.

Mainstream Pick

Gottlieb is a more mainstream nominee than some of the other candidates who were said to be under consideration. He’s a partner at one of the world’s largest venture capital firms, New Enterprise Associates, which has a portfolio of more than 300 businesses in the technology and health-care industries, according to its website.

Other names for the FDA post that Trump considered in the past, according to people familiar, were Jim O’Neill, a Silicon Valley investor who has suggested that drugs need to be assessed only for safety, not efficacy, before they’re approved. Another was biotech executive Balaji Srinivasan, who’s tweeted that the FDA “bears responsibility for many deaths” because it “blocked many good drugs.”

While Gottlieb has focused on easing regulations, he “understands the agency and has some respect for it,” Diana Zuckerman, president of the National Center for Health Research, said in an interview before the news that Gottlieb is the leading candidate. The center is a Washington think tank that promotes education and research on policies that benefit patients.

Consulting, Speaking Fees

The government watchdog group Public Citizen called Gottlieb’s deregulatory approach “dangerous.” He received at least $413,000 in consulting and speaking fees from medical-product companies in 2013 to 2015, Public Citizen said. Those payments came from drugmakers including GlaxoSmithKline Plc, Valeant Pharmaceuticals International Inc. and Vertex Pharmaceuticals Inc., according to the U.S. government database that tracks physician payments from drugmakers.

“Gottlieb’s appointment would accelerate a decades-long trend in which agency leadership too often makes decisions that are aligned more with the interests of industry than those of patients,” Public Citizen said in a statement.

Most of Trump’s public comments about the drug industry have focused on prices. On Tuesday, Trump tweeted that he’s working on a “new system” that will bring costs “way down.”

Faster Approvals

In past speeches, Gottlieb has indicated a link between lowering prices and speeding approval. He’s praised the FDA cancer division’s efforts in getting drugs approved faster, and suggested that other parts of the agency need to emulate its approach, particularly for rare genetic diseases.

Making drug testing more efficient could ultimately “lower the costs that need to be recouped after a drug is approved,” he said at a May 2016 conference in Washington.

If the FDA can accelerate approval for older generics, “the market will basically have a self-correcting mechanism for those distorted prices on old generics,” said Alex Azar, a former Eli Lilly & Co. executive and health official under President George W. Bush, in an interview last week before Gottlieb emerged as the front-runner.

Gottlieb was trained as a physician and completed his residency in internal medicine at Mount Sinai Medical Center in New York after graduating from the Mount Sinai School of Medicine in 1999. He earned a degree in economics from Wesleyan University in 1994.


“Throughout his career, including his time at the agency, Scott has shown that he has the skills necessary to continue to lead the FDA to be patient-centered and science-focused,” said Ellen Sigal, founder and chair of Friends of Cancer Research. The group works with patients and regulators to try to accelerate the approval of promising drugs.

In his past stint at FDA, Gottlieb served as deputy commissioner for medical and scientific affairs and helped coordinate with the White House and the Department of Health and Human Services. Part of his work included facilitating talks that led to a system in which generic drug manufacturers pay a fee to help accelerate the review of their products, similar to one in place for brand-name drugs.

Gottlieb also worked at the Centers for Medicare and Medicaid Services, where he helped implement the Medicare Part D program that provided prescription drug coverage for seniors.

Read original article here.

Diana Zuckerman, Ph.D. and Anna E. Mazzucco, Ph.D.

In recent years, there has been a growing concern that annual mammograms starting at age 40 may do more harm than good for many women. That is why the U.S. Preventative Services Task Force, an expert group that reviews the latest research findings, recommends that mammography screening for most women start at age 50 rather than 40, and that the frequency be every two years (instead of annually) through the age of 74.

The Task Force is widely used as a gold standard for determining medical treatment and screening. In this case, they recommended raising the age to 50 after the American College of Physicians recommended the same thing, and they also recommended that women continue to undergo mammograms until age 74. They say that there is no evidence of what the benefits might be for women 75 and older.

For many years, the American Cancer Society (ACS) recommended annual mammograms starting at age 40, but in October 2015, they issued new recommendations that moved in the direction of those of the medical experts. They now recommend that women at average risk of breast cancer start mammography at 45, that they undergo annual mammograms from 45 – 54, and continue to undergo mammography every other year after that. In contrast, some experts point out that screening mammograms usually do more harm than good, because there is no evidence that they save lives or result in less radical surgery. 1 Experts do not recommend MRIs for screening women of average risk, but clinical studies are being done to determine whether they should be.

So what is best for you?

A key reminder: These recommendations are for screening mammograms. Mammograms are still needed at almost any age if a lump is found. The mammography recommendations also do not apply to all women, only for the average woman. Experts agree that women at especially high risk of breast cancer, such as those with mothers or sisters who had breast cancer, may want to start mammograms between the ages of 40 and 50 or in rare cases, even earlier.

The bottom line is that mammograms have the potential to help detect breast cancer earlier. However, like most medical procedures, there are risks as well as benefits.

Whether to start at age 50, age 40, or earlier or later or never depends on several different factors.

For most women, who are not at especially high risk of breast cancer, regular mammograms do not need to start before age 50. Or, to be cautious, a woman can get one mammogram earlier (around age 45), and then if it is normal, wait until she is 50 for her next mammogram. This is the advice that the National Center for Health Research and their Cancer Prevention and Treatment Fund have been giving since 2007.

Women at higher risk of breast cancer should not wait until they are 50 to have regular mammograms. Please remember that the higher age– 50– is only a guideline (not a strict rule), and only for women with no symptoms and who are not at high risk of breast cancer. In addition, if a woman finds a lump on her breast, a mammogram is still very important, regardless of the woman’s age. For a woman at high risk of breast cancer because of her family history or environmental exposures, regular screening before age 50, or even before age 40, may be a very good idea.

Women who are carriers of the BRCA genetic mutation were previously recommended to begin yearly mammograms between ages 25-30, since this mutation puts them at much higher risk of getting breast cancer. Newer studies have found that starting yearly mammograms before age 35 has no benefit and may instead be harmful. Women end up with higher exposure to radiation for mammograms over their lifetime, which increases their chance of getting radiation-induced breast cancer that they may not have gotten otherwise.2

Most women who have a mother, sister, or grandmother who had breast cancer at the age of 50 or older, or who are at high risk of breast cancer because of obesity or other reasons, may want to have regular mammograms (every two years) starting between ages 40 and 50. If their relatives had breast cancer at a young age, women may consider mammograms even before age 40. Unfortunately, younger women tend to have denser breasts, which often look white on a mammogram. Since cancer also shows up as white, mammograms are less accurate for younger women (and other women with dense breasts). For those women, a breast MRI is likely to be more accurate than a mammogram, and they are safer than mammograms.

Breast MRIs are more expensive than mammograms, costing an average of $2,000 (compared to about $100 for a mammogram). The Task Force says there isn’t enough information to recommend for or against MRIs. For that reason, insurance may not cover the cost. If you want insurance to pay for an MRI, you probably need your doctor to recommend it because of your high risk. Women with dense breasts are at higher risk, especially women with mothers or sisters who had breast cancer at a young age. It is logical that they could potentially benefit from regular breast MRIs, but research is lacking to draw conclusions.

Which kinds of cancer risks can help me decide?

A 2011 article by Dr. John Schousboe and his colleagues examined mammography for women at different ages and with different risk factors. They concluded that mammography screening once every two years (biennial) had health benefits and was cost effective for all women 40-79 with high breast density or with both a family history of breast cancer and a breast biopsy, regardless of breast density. Biennial mammography was also beneficial for women aged 50-69 with average breast density and women 60-79 with low breast density and either a family history of breast cancer or a previous breast biopsy. Annual mammography was not cost-effective for any group.

The study’s authors concluded that each woman’s decision about mammography screening should be based on the following risk factors: age, breast density, history of breast biopsy, family history of breast cancer, and personal beliefs about the benefits and harms of screening. This study supports the Task Force guidelines that women at an average risk of breast cancer can start biennial screening at age 50, and that women at a higher breast cancer risk should consider screening before age 50.3

The chances of getting breast cancer increase with age, and the disease is much more common after age 50. So, from a public health and cost-effectiveness perspective, annual screening mammograms do the most good after age 50. Earlier mammograms are less accurate and more likely to result in unnecessary anxiety or unnecessary biopsies. Unlike Schousboe and his colleagues, the Task Force did not recommend routine screening for women 75 and older, because there was not enough evidence to conclude whether or not the benefits outweigh the risks. However, the American Cancer Society recommends that screening every other year continue for older women whose health is good enough that they are likely to live at least 10 years. That is a difficult standard to implement: How many doctors want to tell their healthy older patients that they don’t need mammography because they are not likely to live at least 10 more years?

Isn’t more frequent mammography better?

Remember that mammograms expose women to radiation, which can increase the risk of breast cancer. Increasing the age of mammograms to age 50 for most women, and reducing the frequency to every two years could save lives because it would drastically reduce radiation exposure. Experts believe that less frequent mammograms also means a lower false alarm rate, and that means fewer unnecessary tests, anxiety, and possibly fewer unnecessary surgeries.45

The big debate: Do mammograms save lives?

Between 1975 and 2000, dramatic improvements in treatments for breast cancer became available. Surgery options were improved, important chemotherapy agents were discovered, and tamoxifen, a hormonal treatment for estrogen-sensitive breast cancer, came into widespread use. At the same time, mammography became more popular. In 2000, about 70% of women 40 and over reported that they had a mammogram within the previous two years. Mammography rates more than doubled between 1987 and 1999, but more recently rates have decreased slightly.

The result of these important advances has been a dramatic decrease in the number of breast cancer deaths, even while more cases of breast cancer were being diagnosed. The five-year survival rate for breast cancer increased from 75% between 1974 and 1976, to 91% between 2005 and 2011.6 Have the survival rates improved because of mammography or because of better treatments?

This became a full-fledged medical controversy in recent years. Two issues were at the root of the debate: 1) Was mammography simply uncovering more tiny, slow-growing abnormalities or cancers that would never have developed into a health threat even if they had never been discovered? and 2) Were we doing more harm than good by subjecting so many women to cancer treatment without knowing whether some of these very early cancers would really become dangerous? Since 2009, research has shown that some tiny cancers disappear on their own without treatment. For example, experts now conclude that most ductal carcinoma in situ (DCIS) will never become an invasive breast cancer, even without treatment.

Regular screening mammography can possibly help diagnose cancer earlier, but the latest research suggests it may not have as much benefit for earlier diagnosis as expected. In January 2017, the Annals of Internal Medicine published a Danish study which examined whether the use of mammography can prevent the number of bigger, more advanced cancers that are difficult to treat.5 Dr. Karsten Juhl Jorgenson and colleagues looked at 30 years of data and compared women living in areas covered by screening programs to those in areas without the programs. Overall, mammography was not associated with fewer advanced cancers. However, in the areas with screening programs, diagnoses of non-advanced cancers increased. It is estimated that up to one third of diagnosed breast cancer cases would never have caused noticeable health problems or death.

Other research indicates mammography may not be saving lives, except possibly for the highest risk women. Researchers estimate that for 1,000 40-year-old women who have annual mammograms, two fewer women will die of breast cancer.7 During that time, approximately 600 of these 1000 women will have false alarms, and approximately 5 – 10 will have unnecessary surgical treatment that could be harmful to them. This latest research did not consider the benefits compared to the risks of regular mammography (every two years) after age 50. It is possible that starting less frequent mammography at 50 (and for women at high risk between the ages 40 and 50) could provide benefits that may outweigh the risks for most women. Although about 90% of worrisome findings from mammograms turn out to be false alarms — not cancer — many experts continue to believe that the overall benefits have been established for women over 50.

Having fewer women die of breast cancer does not, however, mean that fewer women die.  None of the studies that evaluate the impact of mammography do so in terms of lives saved. Instead, they evaluate the number of women who die of breast cancer specifically.

What about breast self-exams? The Task Force recommends against teaching women to do breast self-exams, because evidence suggests the risks outweigh the benefits. There are many “false alarms,” and by the time a cancer is large enough to be felt in a self-exam, it will soon be found anyway, in the shower or while dressing. The Task Force and the American Cancer Society no longer recommend that doctors do breast exams on their patients for the same reason. Nevertheless, women should be familiar with how their breasts normally look and feel and report any changes to a doctor right away.

For more information:

U.S. Preventive Services Task Force, Breast Cancer Screening Final Recommendations, 

For information about insurance coverage for free mammograms:

Related Content:
Should I “upgrade” to digital or 3D? A mammography guide
Breast implants and mammography: what we know and what we don’t know
DCIS: Mostly good news

February 21, 2017

Division of Dockets Management (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852

National Center for Health Research’s Public Comments on Lead in Cosmetic Lip Products and Externally Applied Cosmetics: Recommended Guidance for Industry on Maximum Levels
[Docket No. FDA-2014-D-2275]

The National Center for Health Research is a nonprofit think tank that conducts, analyzes, and scrutinizes research, policies, and programs on a range of issues related to health and safety. We do not accept funding from companies that make products that are the subject of our work.

We support the efforts of the Food and Drug Administration (FDA) to provide a recommended maximum level of lead as in impurity in cosmetic lip products and externally applied cosmetics that are marketed in the United States. We are very concerned, however, about the FDA’s rationale for concluding that 10 parts per million (ppm) is an acceptable level of lead in these products and strongly encourage the FDA to hold cosmetics manufacturers to a safety standard based on solid scientific evidence.

The FDA’s 10 ppm limit is based on the recommended maximum level set by the International Cooperation on Cosmetics Regulation and by other countries, such as Canada and the European Union. In addition, the FDA evaluated lead levels in a sample of products currently on the U.S. market and found that about 1% of samples had lead levels that exceeded 10 ppm. As a result, the FDA concluded that the 10 ppm level is safe and achievable so long as manufacturers follow “good manufacturing practices” and seek to achieve even lower levels “whenever feasible.”8

There is no known level of lead exposure that is considered safe. With consumers’ health in mind, the first question the FDA should ask is: How much lead in one’s system is considered “safe?” The FDA proposal is linked to the Center for Disease Control and Prevention’s (CDC) threshold for lead in the blood of 5 micrograms per deciliter of blood (5µg/dL), above which children should be targeted for treatment. However, the CDC lead threshold is not based on evidence that 5µg/dL is the minimum lead level that causes harm, but instead it arbitrarily identifies children with the highest levels of lead exposure (i.e. the 2.5% of children aged 1 – 5 years with the highest levels of lead in their blood).9 This is particularly problematic because blood lead level tests do not measure lead’s total burden on the body but instead reflect recent or ongoing lead exposure. It should be unacceptable because blood lead level concentrations as low as 5µg/dL may result in decreased intelligence in children, behavioral difficulties, and leaning problems. The CDC and World Health Organization both state that there is no safe level of lead in the blood and that lead poisoning is preventable.10 11

Because of the serious risks of lead, the status quo should not be used to justify the proposed lead level for cosmetic lip products. The maximum level of lead in these products should be based on sound science. The FDA’s proposed maximum level of 10 ppm is not based on scientific safety data and instead on the status quo according to the FDA’s analysis of a sample of lip products sold in the U.S. and the CDC’s similarly questionable threshold.

When considering the maximum level, it is important to acknowledge that some women will use these lip products several times per day, seven days per week for years. One study found that women applied lipstick on average 2.35 times per day and an average of 10 mg of product at each use.12 Thus, researchers estimated that metal intakes for average use were 24 mg/day and 87mg/day for high use of lip products. In addition, many consumers begin using lip products as children or adolescents and continue using them for decades. These multiple exposures over time can lead to increased buildup of lead in the body.

No research has been conducted to determine whether or not the FDA’s proposed recommendation for cosmetic lip products and externally applied cosmetics is safe for consumers. Basing this recommendation primarily on the status quo in the U.S. and other countries creates a disincentive for the cosmetic industry to reduce lead levels in these products. The FDA has a responsibility to set high standards for manufacturers so that consumers are not inadvertently exposed to products that harm them. This is especially true for a guidance document that has no enforcement mechanism.

As the FDA draft guidelines state, the toxicity of lead is well documented.13 Lead is a toxic metal that disrupts brain development and causes problems with behavior, such as one’s ability to stop and think before acting.14 Lead exposure has been linked with reduced IQ and educational attainment as well as increased antisocial behavior. Furthermore, exposure to lead before birth increases risk for low birth weight.15 Early exposure to lead can increase risk for childhood obesity and diabetes, heart disease, or stroke in adulthood.16 As little as a 2µg/dL blood lead level is associated with increased death due to heart attack and stroke.17

Children, infants, and fetuses are particularly vulnerable to lead as their brains are not fully developed.18 Thus, smaller amounts of lead will have a greater effect than when compared to adults. Young children absorb 4-5 times the amount of lead that an adult ingests from the same source. Since lead stored in women’s bones and teeth may be released into the blood during pregnancy and lactation to expose the developing fetus and infants,19 women may cause significant harm to their children by ingesting lip products.

It is also essential to consider that some children, teenagers, and adults using lip products will be exposed to other sources of lead, including exposure to lead paint, water from leaded pipes, and lead-contaminated dust and soil.20 Though lead-based paints were banned for use in housing in 1978, approximately 24 million housing units in the U.S. still contain deteriorated lead paint and elevated levels of lead-contaminated house dust, and individuals living at or below the poverty line are at greater risk.21 A recent report found that 5,363 community water systems, serving over 18 million Americans, exceeded the established lead limits for water.22 The body accumulates lead over a lifetime and gets rid of it very slowly. It takes 28 to 36 days to remove about half of the lead from the blood of an adult.23

Lead has been identified as 1 of 10 chemicals of “major public health concern.”24 Researchers estimated that lead exposure resulted in $5.9 million in medical costs and an additional $50.9 billion in lost economic productivity resulting from reduced cognitive potential due to childhood lead exposure.25

In conclusion, although we support the FDA’s efforts to provide a recommended maximum level of lead as in impurity in cosmetic lip products and externally applied cosmetics, we reject the FDA’s rationale for the maximum level of 10 parts per million of lead in these products. Instead, the FDA should base this level on sound safety data. We believe that the maximum level of 10 parts per million is a reasonable temporary standard, but should be labeled as such in the guidance while the FDA requires research to determine what the level should be. This will ensure that the FDA accomplishes its stated goal to encourage companies to ensure that cosmetic lip products and externally applied cosmetics do not pose a health risk to consumers.

Liz Szabo, Kaiser Health News

February 9, 2017

Marlene McCarthy’s breast cancer has grown relentlessly over the past seven years, spreading painfully through her bones and making it impossible to walk without a cane.

Although the 73-year-old knows there’s no cure for her disease, she wants researchers to do better. It’s been years, she said, since she has found a drug that has actually helped. McCarthy said she’s frustrated that the Food and Drug Administration is approving cancer drugs without proof that they cure patients or help them live longer.
Pushed by patient advocates who want earlier access to medications, the Food and Drug Administration has approved a flurry of oncology drugs in recent years, giving some people with cancer a renewed sense of hope and an array of expensive new options. A few of these drugs have been clear home runs, allowing patients with limited life expectancies to live for years.
Many more drugs, however, have offered patients only marginal benefits, with no evidence that they improve survival or quality of life, said Dr. Vinay Prasad, assistant professor of medicine at the Oregon Health and Sciences University, who has written extensively about the FDA’s approval process for cancer drugs.
Overall cancer survival has barely changed over the past decade. The 72 cancer therapies approved from 2002 to 2014 gave patients only 2.1 more months of life than older drugs, according to a study in JAMA Otolaryngology-Head & Neck Surgery.
And those are the successes.
Two-thirds of cancer drugs approved in the past two years have no evidence showing that they extend survival at all, Prasad said.
The result: For every cancer patient who wins the lottery, there are many others who get little to no benefit from the latest drugs.
In a November study published in JAMA Internal Medicine, researcher Diana Zuckerman looked at 18 approved cancer drugs that didn’t help patients live longer. Only one had clear data showing that it improved patients’ lives, such as by relieving pain or fatigue.
Two drugs harmed quality of life. For example, thyroid cancer patients taking the most expensive drug, cabozantinib, scored worse on a scale measuring five symptoms: diarrhea, fatigue, sleep disturbance, distress, and difficult remembering, Zuckerman said.
“We cannot have a system where drugs that may not even work are being sold for these amazingly crazy amounts of money,” said Zuckerman, president of the National Center for Health Research, a nonprofit in Washington that aims to explain research to consumers.
Recognizing the slow pace of progress, the American Society of Clinical Oncology has set goals for new cancer drugs of extending life or controlling tumors for at least 2.5 months. The bar was set relatively low because “it’s not very often that we come across a transformative treatment,” said Dr. Sham Mailankody, an assistant attending physician and myeloma specialist at Memorial Sloan Kettering.
Yet in a study published in September in JAMA Oncology, Mailankody found that only one in five cancer drugs approved from 2014 to 2016 met those standards.

The FDA wants to give patients the chance to benefit as soon as possible, rather than waiting for definitive proof of improved survival, Pazdur said. In some cases, the FDA requires pharmaceutical companies to perform long-term studies after drugs are approved, to measure whether drugs live up to their early promise.

But many of these studies never provide an answer, Zuckerman said. Once a drug is approved and is available to anyone, patients have no incentive to participate in a clinical trial. So studies can end with no clear conclusion.

Unless the FDA requires companies to provide survival data before approving a drug, “we may never have answers,” Zuckerman said. “We will have all of these expensive drugs on the market and we will never have the information we need about how well they work or even how safe they are.”

President Donald Trump has vowed to cut regulations at the FDA and recently told pharmaceutical industry leaders that he wants to further speed up the drug approval process.

Read the entire article here.

Jay W. Belle Isle, Legal Reader

As I sit here writing, I have to wonder how much of this post will be true tomorrow. We have a saying in Michigan: “Don’t like the weather? Wait five minutes, it’ll change.” The saying seems to fit the new administration, too. Changes – and executive orders – are flying around at the speed of light.  Trump met with some of the world’s biggest Big Pharma executives Tuesday of this week. His message was pretty clear: lower drug prices, bring jobs back to the U.S., and he’ll gut the already weakened FDA, doing away with those pesky regulations that barely keep the American people safe now.

How comforting. If successful, we may as well start calling the FDA the Former Drug Agency.

The execs in attendance at this diabolical gathering came from Johnson & Johnson, Celgene Corporation, Eli Lilly & Company, Merck & Company Inc., Novartis AG (a Swiss company) and the chief of the Pharmaceutical Research and Manufacturers of America (PhRMA), the industry’s puppet lobbying group, among others. PhRMA is behind the plan for a multi-year, multi-million dollar ad campaign aimed at justifying increases to drug prices. One wonders if PhRMA will cancel this foolish waste of time and money following this meeting.

During the meeting, Trump said, “So you have to get your companies back here. We have to make products … We have to get rid of a tremendous number of regulations. I know you have some problems where you cannot even think about opening up new plants. You can’t get approval for the plant and then you can’t get approval to make the drugs. We have no choice. For Medicare, for Medicaid, we have to get prices way down, so that’s what we’re going to be talking about. We’re also going to be streamlining the process so that from your standpoint so that when you have a drug you can actually get it approved — if it works — instead of waiting for many, many years.”

The CEOs who jumped on the opportunity to highlight their long histories in the U.S. and all of the U.S. plants they have and a story in the Wall Street Journal states that over half of the drugs sold in the U.S. are made here. However, according to the WSJ, most of the raw ingredients in those drugs come from places such as India and China. It has been pointed out that regardless of a drug’s country-of-origin, its manufacturer must adhere to U.S. regulations to be sold here. Essentially, that means Big Pharma has to meet U.S. regulations for drugs made here or abroad. So, the only reason for moving jobs overseas is basically a boost to Big Pharma’s bottom line in the form of less taxes, etc.

A bit of tension still exists between the Commander-in-Chief and Big Pharma. Drug prices. While he hopes the industry will respond favorably to his offer to lower taxes and gut the FDA, Trump also added that he’s willing to whip out the pen and correct the problem with drug prices himself if needed. This could include letting Medicare negotiate drug prices with the industry, an idea that Big Pharma despises with all of its corporate greed.

Dr. Diana Zuckerman, president of the National Center for Health Research, an organization that works for strong safety and effectiveness standards in the industry said, “Streamlining drug approvals sounds good, but the agency has already weakened approval standards and patients are paying the price — hugely expensive drugs that don’t even work.”

Dr. Zuckerman has done her homework, too. In a study published on the JAMA Network, Dr. Zuckerman and her co-author, Tracy Rupp, looked at certain new – and expensive – cancer drugs. They found that many of these drugs failed to improve patient quality of life or extend patient life.

Read the full article here.

Maura Duffy


Urinary incontinence is the loss of bladder control, a condition that affects 13 million women in the United States. 26 Stress incontinence, the most common type in women, is when leakage occurs during activities that put pressure on the bladder, such as coughing, sneezing, laughing, lifting, or running. These accidents happen when the muscles that contract to hold urine in (known as pelvic floor muscles) are too weak to handle sudden pressure on the bladder during “stressful” activities.

Though stress incontinence affects people of all ages, races, and both sexes, it is twice as common in women as in men, and affects one in three women at some point in their lifetime. 27 It is more common in women because pregnancy, childbirth, and menopause may weaken the pelvic floor muscles. The condition is especially common in older women post-menopause because the pelvic floor muscles tend to weaken with age, especially when hormone levels drop.

If stress incontinence is affecting your lifestyle, there are treatments that can lessen the problem or eliminate it entirely. There are many effective treatments, and surgery should be considered only as a last resort.

Pelvic floor muscle exercises, which you can perform yourself at no cost, is the treatment that has been shown to have the most consistent success to help women achieve bladder control. 28  The fact that it is free is probably the reason it is not more widely used: nobody is advertising it because they can’t make money.

Before you go to a doctor about your stress incontinence, here is information that will help you decide what kind of doctor or treatment is best for you.



The easiest first step is pelvic floor muscle training, done by performing Kegel exercises. Invented by and named after gynecologist Dr. Arnold Kegel, these exercises are performed by squeezing and holding the muscles you contract to stop yourself from going to the bathroom. A doctor can help you develop an exact regimen, but usually these exercises should be performed about twice daily, and they can be done almost anytime: while watching TV, sitting at a stop light, or working at your desk, for example. 29 Research proves that consistent pelvic floor muscle training decreases stress urinary incontinence incidents. 30 In a review of 905 studies, 3/10 women achieved full control of their bladder (continence), and an additional 4/10 reduced their symptoms by pelvic floor muscle training alone, making it the most successful treatment studied. There are no risks from Kegel exercises, but do not perform them while urinating or with a full bladder, as this may lead to urinary tract infections.

If you are having trouble with Kegel exercises, there is help available. The biofeedback method involves an electrical patch that a therapist places over your bladder and links to a TV screen. You and your therapist can see on the screen when your pelvic muscles are contracting as you perform Kegel exercises, so you learn to identify the right muscle groups. There are no harmful effects from biofeedback, but there is no evidence that it will improve your bladder control more than Kegel exercises alone – unless you need the biofeedback to identify the muscles to exercise. 31

Vaginal cones (or vaginal weights) are another tool to help women increase pelvic muscle strength. While standing, the patient inserts the small metal cone like a tampon and then contracts the pelvic floor muscles to prevent the cone from slipping out, usually for about 15 minutes at a time once or twice a day. Research has shown that while vaginal cones are inexpensive and have no harmful effects, they are not more effective at reducing stress leakage than basic Kegel exercises.

Just as your arms, legs, and abs do not instantly tone from going to the gym a few times, it takes time for pelvic floor muscles to strengthen enough to noticeably improve bladder control. Most women see a reduction in leakage after about six to twelve weeks of regular Kegel’s exercises, so be patient and stick with your routine. Though biofeedback and vaginal cones are not necessary, they are safe tools if you are having trouble with Kegel exercises.


Weight Loss

For women who are overweight, reduction in bladder leakage is yet another health benefit of weight loss. In one study, 388 overweight and obese women were assigned to an intensive 6-month weight-loss program that included diet and exercise. Almost three quarters of the women who lost weight noticed a significant reduction in the weekly number of incontinence episodes. Though it is not known for sure why extra weight may exacerbate stress incontinence, some scientists believe that excess belly fat puts more pressure on the abdominal muscles, which in turn increases pressure on the bladder. Weight loss may reduce these extra forces on the bladder, resulting in fewer incontinence episodes.


Medical Devices

Another way to strengthen the pelvic floor muscles is by electrical or magnetic stimulation. With electrical stimulation, a silicone rubber tube that is attached to a battery-operated unit is inserted into the vagina, similar to a tampon. A small amount of electricity is sent through the tube into the surrounding nerves, causing the muscles to contract. This therapy, which can be done at home or in a doctor’s office, has a success rate similar to Kegel exercises. However, it has no additional benefits and costs much more money.

Magnetic stimulation is done in a doctor’s office with the woman sitting in a special chair that has magnetic coils. An electric current is sent into the coils, creating a magnetic field that tenses and relaxes the muscles of the pelvis. Research has shown that magnetic stimulating devices work just as well turned off as they do turned on, so there is no reason to spend money on these treatments. 32

A pessary, also known as intravaginal bladder neck prosthesis, is a stiff ring that a doctor or nurse will fit you for and insert into the vagina. The rigid ring, made out of latex or silicone, repositions the urethra, leading to less stress leakage. A pessary requires regular checkups with a doctor, as it can increase the risk of developing urinary tract infections with complications. There are other disposable inserts that you can apply yourself, like urethral plugs and continence tampons. Two scientific reviews have concluded that there is not enough consistent evidence to show that pessaries and other disposable vaginal devices improve stress urinary incontinence. So again, Kegel exercises, which are proven to work, are a better option.


Prescription Drugs

There are several drugs that are prescribed for stress incontinence, but all have side effects and risks, and many don’t help at all. Estrogen, which is made under many brand names and can be taken in a variety of ways (orally, applied to the skin, or inserted in the vagina), may be prescribed for stress incontinence in post-menopausal women. This use is “off label,” which means it was not proven to work for stress incontinence.  However, doctors can still prescribe it for that. Studies have shown that low doses of vaginally administered estrogen – tablets, creams, rings – may improve stress incontinence symptoms for some women. Estrogen patches that you place on the skin, however, may worsen symptoms. There are health risks associated with estrogen, as there are with any post-menopausal hormone therapy.

Duloxetine, under the brand name Cymbalta, is an anti-depressant that some doctors prescribe “off-label” for stress incontinence. A review of clinical trials showed that duloxetine did not cure stress incontinence when compared to a placebo. Though some women did have improved bladder control while taking duloxetine, many women experienced unpleasant side effects like dry mouth, nausea, and fatigue and therefore stopped taking it. In fact, more women stopped taking Cymbalta because of side effects than the number of women who noticed an improvement in bladder control.



Another therapy involves injecting “bulking agents” to thicken the tissue around the urethra so it can better take the pressure that causes leakage. The bulking material varies: it can be carbon beads, calcium particles, bovine collagen, swine collagen, or silicon particles. 33 Repeat injections every 6 to 18 months are usually needed because the body breaks down bulking agents over time. A scientific review of 14 clinical trials of various bulking agents that included over 2,000 women reported that the only bulking agent to show an advantage over Kegel exercises was silicon particles. However, the follow-up for this trial was only 3 months, so it is not known if these results hold up in the long run or what the long-term risks are. Known side effects of urethral injections are urinary retention (inability to urinate), painful urination, and pain or infection at the injection site. Given the lack of long-term data on the success of injection therapy and the high cost of needing repeat injections, there is no reason to spend money on this type of treatment.



Renessa is the brand name for a procedure known as “radiofrequency collagen denaturation.” A probe is inserted into the upper urethra (near the bladder) and emits radio waves that generate heat, burning tissue in the urethra. When the tissue rebuilds itself and forms scars, it becomes firmer, which may decrease involuntary leakage. 34 This procedure can be done in a doctor’s office in less than half an hour, but it requires anesthesia. The only published studies have been funded by the manufacturers of Renessa, and none have follow-up periods longer than 3 years, so it is not known what the consequences of burning your urethral tissue are in the long run. There are some online reports by women whose incontinence was actually worse after Renessa, which is important in light of the lack of unbiased research. It is also important to note that the FDA has only approved Renessa for women who have tried and failed at conservative treatments, meaning Kegel exercises. 35 Harvard Women’s Health Watch recommends against Renessa because of the lack of independent studies on its safety and effectiveness.


Surgery as a Last Resort

Although there are surgical devices such as bladder slings, mesh implants, and transvaginal tape that are approved by the FDA to treat urinary incontinence, there are very often complications. We recommend trying non-surgical treatments before even considering surgery because of those risks.

Mesh is especially risky.  As the mesh ages inside the body, it can cause chronic pain, nerve damage, scarring, and infection. 36 Some women report being unable to have sex or even sit without pain.  Several surgical mesh implants have been withdrawn from the market because of lawsuits filed by women who have been seriously injured.  Men undergoing hernia repair for incontinence and other reasons have similar problems. While some surgeons prefer to use mesh and claim it improves the success rate, the mesh itself caused serious complications in 6% of mesh patients in a recent study. These included bowel perforation, wounds that would not heal, hematomas, seromas, and bowel obstruction. 37 Most of these complications required re-operation.


The Bottom Line

While stress incontinence may feel embarrassing or demoralizing, remember that it is a medical condition that affects millions of women, and it can be lessened or cured without invasive surgery. Since Kegel exercises are the simplest, safest, and most effective treatment, it should be tried first, but must be done regularly. In fact, Kegel exercises alone, when performed correctly, daily, and for at least six weeks, are extremely likely to reduce symptoms. In the meantime, you can help reduce your symptoms by scheduling regular bathroom breaks, wearing an absorbent liner, or losing weight if you need to.

All articles on our website have been reviewed and approved by Dr. Diana Zuckerman and other senior staff.

By Katie Thomas. The New York Times

January 31, 2017

President Trump promised some of the nation’s top drug company executives in a meeting at the White House on Tuesday that he would slash regulations at the Food and Drug Administration and make it easier for them to manufacture products in the United States.

He also described as “fantastic” the person he planned to nominate for commissioner of the Food and Drug Administration, someone he said would streamline the agency and get drug approval decisions faster.

“You can’t get approval for the plant, and you can’t get approval for the drug; other than that, you’re doing fantastic,” Mr. Trump said at the meeting, to laughter from top executives of companies like Merck, Johnson & Johnson, Eli Lilly and Novartis.

But even as he struck a cordial tone with them, he said that lowering drug costs would remain a focus and that he would discuss the issue further during the nonpublic portion of the meeting, away from the news media. “The U.S. drug companies have produced extraordinary results for our country, but the pricing has been astronomical,” Mr. Trump said. “We have to get prices down for a lot of reasons.” […]

“Streamlining drug approvals sounds good, but the agency has already weakened approval standards and patients are paying the price — hugely expensive drugs that don’t even work,” said Diana Zuckerman, president of the National Center for Health Research, a Washington research group that advocates strong safety and efficacy standards. She  recently studied expensive new cancer drugs and found that many did not help patients feel better or live longer.

Read the original article here.

By Carolyn Y. Johnson, The Washington Post

January 31, 2017

President Trump met with leaders of some of the world’s biggest pharmaceutical companies Tuesday and emphasized the need to lower “astronomical” drug prices, decrease regulations and bring more drug manufacturing into the United States.

Trump offered no specific policies, but mentioned increasing competition and “bidding wars” as a way to bring down prices. In the past, he has lashed out at the pharmaceutical industry for “getting away with murder” and threatened to use the government’s bargaining power to force down drug prices for programs like Medicare.

Most of Tuesday’s meeting was held behind closed doors, but Trump spoke to the media beforehand while surrounded by executives from a half-dozen large drug companies. He struck a less combative tone and didn’t mention government intervention directly.

“We have to get prices down for a lot of reasons. We have no choice,” Trump said, flanked by chief executives Kenneth Frazier of Merck and Robert Hugin of Celgene. “For Medicare, for Medicaid, we have to get the prices way down.” […]

In the past, pharma companies have railed against government intervention in pricing, saying those prices fund development of future, lifesaving drugs.

There are many empty positions at the FDA, said Diana Zuckerman, president of the National Center for Health Research. The recently enacted 21st Century Cures law provided for additional hiring authority.

“But with the hiring freeze, will they be able to hire anyone?” she asked.

Read the original article here.


January 28, 2017

The possible repeal of the Affordable Care Act reintroduces a fear among people with pre-existing conditions of maintaining health insurance.

Under the ACA, pre-existing conditions were not counted against applicants seeking coverage through the state’s exchange, Covered California, but that could change if ACA is repealed and replaced under the Trump administration.

“There’s a lot of talk right now as to what’s going to happen if ACA is repealed,” Nicklas said. “The feds may be letting the states decide if they want to keep it or not. California is one of the states that incorporated what’s called an exchange so rather than just going off of, they created their own nonprofit state funded exchange. A couple of other states interpreted the law how they wanted to as well.

Prior to ACA, insurers could deny coverage of individuals who were considered to be high-risk because of previously diagnosed conditions, Nicklas said.

Women typically had a more difficult time obtaining health insurance prior to the ACA, according to National Center for Health Research President Diana Zuckerman.

“Women applying for an individual health insurance policy had to answer a question if she’d ever had breast cancer, breast implants, or any other breast condition,” Zuckerman said. “When they first developed the test for the breast cancer gene mutation (BRCA1 and BRCA2 genes) a big problem was women who wanted to get tested where afraid they wouldn’t be able to get health insurance if they tested positive for the genes.”

Most health insurance companies also treated individual patients with pre-existing conditions differently than those that bought health insurance through large employers. This, according to Zuckerman, was what made it difficult for people to leave jobs where they had insurance.

Zuckerman said that prior to the ACA, a large majority of health insurance companies would either not sell insurance to people with pre-existing conditions, sell them insurance policies but not cover the pre-existing conditions, or sell them extremely expensive policies. Going without insurance would not be ideal due to the high cost of most medical procedures.

“The cost of treatments would be thousands of dollars that the vast majority of people do not have. People in those situations are just one hospitalization away from bankruptcy,” Zuckerman said. “None of the suggestions being made or proposed by Congress or the Trump team would be considered better by anybody with a pre-existing condition.”

Read the original article here.

By Roni Caryn Rabin, New York Times

January 29, 2017

After learning she had a high genetic risk for breast cancer, Dane’e McCree, like a growing number of women, decided to have her breasts removed. Her doctor assured her that reconstructive surgery would spare her nipples and leave her with natural-looking breasts.

It did. But while Ms. McCree’s rebuilt chest may resemble natural breasts, it is now completely numb. Her nipples lack any feeling. She cannot sense the slightest touch of her breasts, perceive warmth or cold, feel an itch if she has a rash or pain if she bangs into a door.

And no one warned her.

“I can’t even feel it when my kids hug me,” said Ms. McCree, 31, a store manager in Grand Junction, Colo., who is raising two daughters on her own.

Plastic surgeons performed more than 106,000 breast reconstructions in 2015, up 35 percent from 2000. And they have embraced cutting-edge techniques to improve the appearance of reconstructed breasts and give them a more natural “look and feel” — using a woman’s belly fat to create the new breast, sparing the nipple, minimizing scarring with creative incisions and offering enhancements like larger, firmer lifted breasts.

Read the rest of the article here.

Stephanie Fox-Rawlings, Ph.D.

January 25, 2017

Thank you for the opportunity to speak today. My name is Dr. Stephanie Fox-Rawlings from the National Center for Health Research. Our center analyzes scientific and medical data to provide objective health information to patients, providers and policy makers.

Development of new antibiotics will only help if they actually improve the health of patients with resistant infections. This is not possible when new drugs are not as safe and effective as antibiotics already on the market or when they are not studied on patients with resistant bacteria.

The FDA often relies on non-inferiority trials for antibiotics, which can result in drugs that are somewhat less effective than an approved version. After several rounds of comparing new drugs to somewhat older drugs that were slightly less effective than previously approved drugs, we can end up with new antibiotics that are much less effective than the best available. Furthermore, if they are chemically similar, the new drug adds to rather than reduces the problem of antibiotic resistance.

Unfortunately, the 21st Century Cures Act could easily increase the number of antibiotics that do not benefit patients.  This is already a problem. FDA’s efforts to speed up drug approvals have led to relatively high rates of antibiotics later removed from the market because they were not effective. The smaller clinical trials for limited populations recommended in Cures could make this worse because this increases the risk that results are due to chance.

Because researchers lack tools to quickly diagnose patients with resistant bacteria, these small studies include patients who do NOT have resistant bacteria.  And once approved, the new drugs are often promoted and prescribed for a wider patient population than included in trials. This can expose patients to unnecessary risks, lower effectiveness, and generate resistant bacteria.

Prevention and control requires methods to make sure antibiotics work on resistant bacteria and are limited to those cases. This includes development of rapid testing options for researchers to determine which antibiotics are most effective and for healthcare and veterinary practitioners to ensure that antibiotics are targeted for approved uses.


By Diana Zuckerman, The BMJ

January 24, 2017

Future-Nasty-Woman-Jan-2017-225x300Did the Women’s March on Washington, across the US, and in major cities around the world have anything to do with medical care? Yes and no.

I’ve lived and worked in Washington, DC for more than 30 years, and I’ve seen a lot of demonstrations, and participated in a fair number as well. But I’ve never seen a demonstration quite like the Women’s March that was held the day after the inauguration of Donald Trump.

Much of the media attention before the march was on the celebrities that were planning to attend. And they were there—bold faced names like Scarlett Johansen and Katy Perry, Senators and Congresswomen, and numerous celebrities whose names I am too old to recognise. But because the permit was for an area that was much too small for the crowd that showed up, the vast majority of the approximately half million women, men, and children never saw or heard most—perhaps any—of the 4-hour program.

That’Trust-Yourself-sign-Jan-2017-300x225s why, compared to other demonstrations, the Women’s March had no one clear message imposed by the organisers. Instead it took on a life of its own, based on the signs and sentiments of those attending.  There were relatively few signs regarding the Affordable Care Act. Perhaps because it seemed so unusual, I noticed quite a few with drawings of the uterus—though the message was a popular feminist theme: “keep your laws off my body.” Abortion rights were not the major issue of the day, despite some media reports that anti-abortion female Trump voters were upset because they felt excluded from a women’s march. It seemed logical to me that Trump voters—even those who considered themselves feminists—were not going to feel welcome, because the bigger, broader message was aimed at those longing to “make America great again” like it was in the good old days:  We won’t go back!


For some deSign-says-it-all-225x300monstrators, they were referring to not being willing to go back to days when women had limited reproductive rights. Others focused on not going back to a time when women were assumed to be less worthy than men and therefore merely the rare tokens in the halls of power, including the US President’s cabinet and inner circle. (Unfortunately, the new Administration has already gone back to those days in their most important Cabinet nominations.)

But what seemed to be the even stronger message was the refusal to go back to the days when sexual assault was generally dismissed as “locker room talk” rather than a public health epidemic that needed to be prevented.  The sea of bright pink “pussyhats” at the various marches—hats that many women told me they had made and given away for free to anyone who wanted them—held the key to the underlying message. There was considerable controversy before the marches about whether the pussyhats were a good joke or a terrible strategic blunder that took attention away from serious issues. But on the day of the marches, the pink hats were everywhere and the message was clear: we’re not going to forgive or forget the vulgar boast that Donald Trump made during a private conversation with a live mic, and we can’t let him or anyone else think it was acceptable just because he was elected anyway.  In other words: Women are worthy of respect and we are going to demand that respect.

The other clear message, repeated all day in what became the most popular chant at the Washington march was, “This is what democracy looks like.”  The subtext: We are peacefully demonstrating to let elected officials know that millions of Americans reject the direction they are going in, especially regarding issues of most importance to women. Those include access to affordable health care, access to safe reproductive health care, laws to protect women from sexual assault and harassment, laws to prevent violence against women and to help victims of violence, voting rights, and protections against discrimination on the basis of sex, race, or age.

Women’s marches across the US on 21 January all seemed to be much more substantial and dynamic than expected. Marches in other major cities around the world were too. I’m sure the messages varied, as they did in Washington. Here, the signs people carried had a range of messages, many hilarious, clever, or moving.  I’ve attached a small sample. A few borrowed slogans from the election, such as “Love Trumps Hate” and babies with signs announcing “Future Nasty Woman.” Some focused on traditional feminist policy issues such as reproductive rights and equal pay for equal work. But the underlying theme as we marched at the White House and the Capitol was: Women aren’t going to let elected officials take away their rights, their health, their well-being, or their self-respect.  We didn’t vote to turn back the clock, and we will fight every effort you make to do so.

Read the original post here.



By Christina Jewett and Marisa Taylor, Kaiser Health News

January 23, 2017

As Cabinet nominee Tom Price faces a Senate confirmation hearing Tuesday, a newly released trove of documents sheds further light on how he interacted as a congressman with the Centers for Medicare and Medicaid, the massive agency he may soon oversee.

Letters provided by CMS after an open records request show that the Health and Human Services pick has repeatedly stepped up in favor of drug firms, device manufacturers and higher physician payments, leading some experts to question whether he would be a reliable advocate for the public’s health.

The documents reveal additional instances where Price, a doctor from Georgia, set aside his priority of budget discipline in favor of special medical interests, including by sending a letter endorsing a medical procedure that Medicare later decided not to pay for, pointing to weak evidence that it helped patients get better.

“He’s clearly shown in this case and in other ways allegiance to the corporate interest, but not to the patient interest,” said Diana Zuckerman, president of the National Center for Health Research, a nonpartisan think tank that has studied medical device safety[…]

Read the full article here.


Tracy Rupp, PharmD, MPH, RD and Mingxin Chen, MHS, National Center for Health Research

Although people all over the world can develop cancer, cancer patients are more likely to survive in areas of the world that receive the most sun.38  Since our skin makes vitamin D when exposed to sun, researchers wondered if vitamin D protects against cancer.  New research suggests that vitamin D may help women diagnosed with breast cancer to survive the disease.

The evidence for the role of vitamin D in breast cancer

In November 2016, a study published in a major cancer journal looked at the association between vitamin D levels and survival in 1666 women with newly diagnosed invasive breast cancer in California. Among the participants, women with the highest vitamin D levels in their blood (the top one-third among the women in the study) were 28% less likely to die from all causes as compared to women with the lowest vitamin D levels (bottom one-third) in their blood. The association between vitamin D and survival was even stronger in premenopausal women: those with the highest vitamin D levels were 55% less likely to die from all causes and 63% less likely to die from breast cancer, as compared to premenopausal women with the lowest vitamin D levels.39

These results are similar to a study published in 2014, which also found that women with higher levels of vitamin D were more likely to survive breast cancer. This study used meta-analysis to pool the results from 5 previously published studies of the relationship between vitamin D levels and mortality from breast cancer. The study found that among 4443 breast-cancer patients, women with the highest vitamin D levels (about 30 ng/mL) were about half as likely to die from breast cancer as those with the lowest levels (less than 20 ng/mL).40

Since both studies found that women with higher vitamin D levels were more likely to survive the disease, we wonder: could the chances of improving survival really be so simple? Not necessarily. These two studies can’t tell us which came first: breast cancer or low vitamin D levels. For example, it’s possible that breast cancer causes vitamin D levels to drop. That’s one of the reasons it would be premature to recommend more vitamin D for women diagnosed with breast cancer.

The evidence for the role of vitamin D in melanoma

A study published in 2016 found that low levels of vitamin D may result in worse outcomes for patients diagnosed with the type of skin cancer called melanoma.41 In this study, melanoma patients who had vitamin D levels less than 20 ng/mL were more likely to have larger tumors and more advanced disease than melanoma patients with higher levels of vitamin D. The researchers also examined inflammation and found that low vitamin D levels predicted poor outcomes for patients regardless of their levels of inflammation.

This result may seem very surprising, since sunlight exposure increases vitamin D and also increases the risk of developing skin cancer. A study is ongoing in Belgium to see whether vitamin D supplements will reduce the chances of skin cancer returning or worsening.42 While it’s too early to recommend widespread vitamin D supplements for skin cancer, it’s reasonable to check vitamin D levels in patients with melanoma or who have been treated for melanoma. If their vitamin D levels are low, a supplement is an easy way to try to bring levels into the normal range.

What is vitamin D?

Vitamin D helps the body use calcium and phosphorus to make strong bones and teeth. Our bodies make vitamin D when our skin is exposed to direct sunlight. We can also benefit from the vitamin D that is added to milk and cereals.

How much vitamin D is recommended for healthy people?

Approximately one-third of children and adults in the U.S. (over 1 year of age) do not get enough vitamin D.43 The Institute of Medicine recommends the following daily amount of vitamin D for average healthy adults:44

  • For those between 1 and 70 years of age, including women who are pregnant or lactating, the recommended dietary allowance (RDA) is 600 IU per day.
  • For those 71 years or older, the recommendation is 800 IU per day.

Experts agree that just 15 minutes of sun at mid-day in the summer is enough. Of course, this varies based on how much skin is exposed (darker skinned people may need more time), the time of the day (mid-day is best for vitamin D), altitude (the higher the altitude you are at the more vitamin D your body can make). It is also more difficult to get enough make enough vitamin D from the sun during the winter. If you live anywhere north of Los Angeles, then you really can’t get much vitamin D from November to March when the sun is very low in the sky. Thus, we have to rely on the vitamin D we were able to store up during the summer or the vitamin D we can take in through our diets and supplements.

How much vitamin D is too much?

Given the possible link to reducing cancer, you might wonder if you should take vitamin D supplements even though the results of these studies are not conclusive. It is important to remember that too much of any nutrient, including vitamin D, can be unhealthy. The safe maximum of vitamin D for adults and children older than 8 years of age is about 4000 IU per day.45

Dietary supplements are more likely than foods to provide too much vitamin D.  Although too much sun exposure is dangerous because of skin cancer, it will not cause vitamin D toxicity.

January 17, 2017

Office of Pollution Prevention and Toxics (OPPT)
Environmental Protection Agency
1200 Pennsylvania Ave., NW
Washington, DC 20460-0001

National Center for Health Research’s Public Comment on
New Chemicals Review Program Under the Amended Toxic Substances Control Act; Notice of Public Meeting and Opportunity for Public Comment

[Docket No. EPA-HQ-OPPT-2016-0658]

The National Center for Health Research is a nonprofit think tank that conducts, analyzes, and scrutinizes research, policies, and programs on a range of issues related to health.  We do not accept funding from companies that make products that are the subject of our work.

We strongly support efforts of the Environmental Protection Agency (EPA) to improve chemical review, as required by the new Frank H. Lautenberg Chemical Safety for the 21st Century Act. The new law requires evaluation of risks before new chemicals go onto the market, instead of only after people have been harmed. The new law and the EPA’s improved chemical evaluation program can greatly reduce the risks that many chemicals pose to the health of consumers, workers, and the U.S. public.

The EPA’s efforts to implement the law are moving in the right direction. The EPA must continue to improve efforts to thoroughly evaluate new chemicals and oppose industry’s unnecessary and inappropriate attempts to weaken those evaluations.  We understand that, industry often opposes regulations in general, and especially when those regulations would delay in getting their products to market.  However, the health of children and adults in America is more important and must take precedence, as the law mandates.

The law requires more information about new chemicals to allow EPA reviewers to better determine risks associated with their production, use, and disposal. Unfortunately, many of the applications were incomplete and for that reason, it is taking reviewers longer to evaluate the submitted materials.  Those delays are not the fault of the law or of the EPA reviewers, they were due to shortcomings of the submitted materials.

Much of this additional information is basic information about the chemical, its manufacture, use, and exposure. Delays will decrease as industry adjusts to the new requirements of the law, and as EPA staff gain experience in conducting reviews. The EPA seeks to review applications and respond within the same 90 day period that it used before the law. But the law clearly allows the agency more time if it is needed, so that chemicals are not allowed to be manufactured due to expiration of the review period.

When evaluating new chemicals, the EPA needs to focus on risks to vulnerable populations. Vulnerable populations included both workers and users who may be exposed to higher levels of the chemicals, as well as fetuses and children who are likely to be more sensitive to small exposures.

The EPA’s risk assessments must be based on good science. Even related chemicals, such as analogs or chemicals with similar structures, can have dramatically different exposure and health effects.  It would be inappropriate to rely on shortcuts instead of adequate and appropriate testing, because to do so would put children and adults at risk.

The law was designed to protect workers, consumers who use chemicals, and people who live where chemicals are released into the environment.  As a think tank focused on public health, we strongly agree that chemicals should be carefully evaluated before they are sold, as well as afterwards. People who work around new chemicals should be informed of the risks and the appropriate protections. Thorough evaluations of new chemicals by the EPA can reduce the risks of many new chemicals, saving lives and improving the health of people who live and work in the United States.

Thank you for the opportunity to comment on the implementation of chemical safety reform.

We joined 233 other organizations in signing this letter to Senators opposing the nomination of Scott Pruitt to head the EPA:

February 15, 2017

Dear Senator:

On behalf of our millions of members and activists, we urge you to stand up for clean air, clean water, healthy communities and a safe climate by insisting that the Environmental Protection Agency must be headed by an individual who is qualified for the office and dedicated to these values.

President-elect Trump’s nominee to head the EPA, Oklahoma Attorney General Scott Pruitt, has actively worked against the mission of the agency he has been nominated to lead and he should be rejected by the Senate.

Mr. Pruitt has repeatedly sued the EPA to block clean air and clean water standards that will protect the health and well-being of millions of Americans.

For example, he has sued the EPA to overturn standards to curb mercury and other toxic air pollutants that will prevent up to 11,000 premature deaths and 130,000 asthma attacks per year. He has sued to void standards to reduce soot and smog pollution projected to prevent up to 15,000 non-fatal heart attacks, 34,000 premature deaths, and 400,000 asthma attacks every year. These lawsuits are bad news for all Americans, but especially the more than 24 million Americans with asthma.

Mr. Pruitt denies the overwhelming scientific consensus that climate change is real and is driven by human-made air pollution. He sued unsuccessfully to overturn the EPA’s scientific endangerment determination that carbon dioxide and other heat-trapping air pollutants are harmful. And he has sued to block the EPA from setting any limits on carbon pollution from power plants, the nation’s largest polluter.

Mr. Pruitt has repeatedly argued that EPA should have little or no role in protecting Americans’ health and well-being from air and water pollution. Thus, he apparently rejects fundamental provisions of the Clean Air Act that require the EPA to set national public health standards–standards that guarantee protection to all Americans, regardless of where they live, and protect states from a “race to the bottom” in which they are pressured to compete for industry by offering lax health and environmental standards.

Mr. Pruitt has sued the EPA to overturn clean water safeguards for more than half the nation’s waterways, including streams that feed into the drinking water supplies of 117 million Americans. He even sued to block limits on water pollution into the Chesapeake Bay, which has no known connection to Oklahoma.

There is a long bipartisan history in this country of supporting clean air, clean water and a healthy environment. The American public did not vote for more air and water pollution, for more pesticides in our foods or for more toxic chemicals in toys. The American people did not vote to put the EPA in the hands of someone who has recklessly worked against its mission to protect Americans’ health and the natural environment.

Scott Pruitt’s views and actions run counter to the EPA’s critical mission to protect our health and the environment. This makes him unfit to administer the laws he would be entrusted to enforce. The Senate should reject his nomination.

Air Alliance Houston
Alaska Community Action on Toxics
Alaska Wilderness League
Alliance for Citizenship
Alliance for Democracy
Alliance for Energy Democracy
Alliance of Nurses for Healthy Environments
American Rivers
Apostolic Faith Center
Appalachian Mountain Club
Appalachian Mountain Advocates
Appalachian Voices
Ashurst Bar/Smith Community Organization
Battlement Concerned Citizens
Benicians for a Safe and Healthy Community
Biomonitoring Resource Center at Commonweal
Bold Alliance
Boston Climate Action Network
Breast Cancer Action
Breast Cancer Fund
California Bicycle Coalition
California Coastkeeper Alliance
California Environmental Justice Alliance
California Environmental Justice Coalition
California Kids IAQ
California Safe Schools
Californians Against Waste
Californians for Pesticide Reform
Cancer Prevention and Treatment Fund
Center for Biological Diversity
Center for Environmental Health
Center for Food Safety
Center for International Environmental Law
Center for Science in the Public Interest
Central Valley Air Quality Coalition (CVAQ)
Chesapeake Climate Action Network
Chesapeake Physicians for Social Responsibility
Citizens Action Coalition of IN
Citizens Against Ruining the Environment
Citizens’ Environmental Coalition
Clean Air Task Force
Clean and Healthy New York
Clean Water Action
Clean Wisconsin
Climate Action Alliance of the Valley
Climate Law & Policy Project
Climatemama Action
Coalition For A Safe Environment
Coalition for Clean Air
Coming Clean
Comite Civico del Valle
Community Dreams
Concerned Residents of Portland, NY + People Like Us (CropPlus)
Conservation Colorado
Conservation Voters New Mexico & CVNM Education Fund
Defenders of Wildlife
Del Amo Action Committee
Downwinders at Risk
Earth Action, Inc.
Eco-Justice Collaborative
Ecology Center
Ecology Center (Michigan)
Elders Climate Action
Emerald Cities Collaborative
eNRG – Energizing Renewable Growth in Holston Valley
Environment America
Environment Arizona
Environment California
Environment Colorado
Environment Connecticut
Environment Florida
Environment Georgia
Environment Illinois
Environment Iowa
Environment Maine
Environment Maryland
Environment Massachusetts
Environment Michigan
Environment Minnesota
Environment Missouri
Environment Montana
Environment Nevada
Environment New Hampshire
Environment New Jersey
Environment New Mexico
Environment New York
Environment North Carolina
Environment Ohio
Environment Oregon
Environment Rhode Island
Environment Texas
Environment Virginia
Environment Washington
Environmental Advocates of New York
Environmental Defense Fund
Environmental Health Strategy Center
Environmental Integrity Project
Environmental Law and Policy Center
Environmental Protection Information Center (EPIC)
Environmental Working Group
Farmworker Association of Florida
Farmworker Justice
Food Empowerment Project
French Broad Riverkeeper
Friends of the Earth
Grand Canyon Trust
Green America
Green For All
Greenpeace USA
Heal the Bay
Health Care Without Harm
Healthy Schools Network
Hispanic Federation
Humboldt Baykeeper
Institute for Agriculture and Trade Policy
Interfaith Power & Light
Iowa Citizens for Community Improvement
Iowa Interfaith Power & Light
IVAN Statewide Network
La Union Hace La Fuerza
Labadie Environmental Organization (LEO)
Latino Decisions
LDA Minnesota
LDA Utah Chapter
League of Conservation Voters
Learning Disabilities Association of Georgia
Learning Disabilities Association of Illinois
Learning Disabilities Association of Maine
Learning Disabilities Association of New Jersey
Learning Disabilities Association of Texas
Let’s Talk Climate
Louisiana Bucket Brigade
Made Safe
Maine Audubon
Maine Wilderness Guides Organization
Martinez Environmental Group
Mass Audubon
Missouri Coalition for the Environment
Moms Clean Air Force
Montana Conservation Voters
Montana Environmental Information Center
Mossville Environmental Action Now
National Audubon Society
National Parks Conservation Association
Natural Resources Defense Council
NC Interfaith Power & Light
Neighbors for Clean Air
Nevada Conservation League
New Virginia Majority
NextGen Climate
North Carolina Council of Churches
North Carolina League of Conservation Voters
Northcoast Environmental Center
Northern Plains Resource Council
Ocean Futures Society
Ohio Citizen Action
Ohio Environmental Council
Ohio Valley Environmental Coalition
Oil Change International
Oregon Aviation Watch
Oregon Environmental Council
Our Roots Multicultural Center
Parents for Nontoxic Alternatives
Partnership for Policy Integrity
People’s Action
Pesticide Action Network
Physicians for Social Responsibility
Potomac Riverkeeper Network
Poverty & Race Research Action Council
Prevent Harm
Public Citizen
Public Lab
Renew Missouri
RESTORE: The North Woods
Rochester People’s Climate Coalition
Safe Alternatives for our Forest Environment
Safe Climate Campaign
Safer Chemicals Healthy Families
San Pedro & Peninsula Homeowners Coalition
Save The Bay, Oakland, CA
Sierra Club
South Portland Air Quality
Southern Maine Conservation Collaborative
St. Philomena Social Justice Ministry
Students for a Just & Stable Future
Sunflower Alliance
Sustainable Silicon Valley
Texas Campaign for the Environment
Texas League of Conservation Voters
The Alaska Center
The Center for Media and Democracy
The Climate Reality Project
The Climate Trust
The Environmental Justice Center at Chestnut Hill United Church
The Greenlining Institute
The Moving Forward Network
The Trustees
Toxic Free NC
Tri County Watchdogs
Union of Concerned Scientists
Utah Moms for Clean Air
Utah Physicians for a Healthy Environment
Ventura Coastkeeper
Virginia Organizing
Voices for Progress
WaterWatch of Oregon
WE ACT for Environmental Justice
West Oakland Environmental Indicators Project
West Virginia Highlands Conservancy
Western Colorado Congress
Western Organization of Resource Councils
Wholly H2O
Wilmington Improvement Network
Wisconsin League of Conservation Voters
Wisconsin Environment
Women’s Voices for the Earth
Young Evangelicals for Climate Action
Young Fresnans for the Environment

Number of organizations updated from 173 to 234 on February 15, 2017.

In April 26, an FDA advisory committee voted 7-6 that the exon-skipping drug eteplirsen for Duchenne muscular dystrophy (DMD) failed to meet the standards needed for accelerated approval. It was widely assumed that the FDA would tell the drug’s developer, Sarepta Therapeutics, to try again with better data. That, of course, did not happen. In this follow-up, we report on how it eventually did turn out for the drug and for the DMD community.

To the surprise of many, the FDA approved eteplirsen in September with the trade name Exondys 51.

While patients and families applauded the decision, believing their efforts in collaborating with industry and the agency had paid off, critics in the medical and research community questioned whether the drug really worked, and whether the FDA had made the right call. Documents later revealed internal FDA friction, and even though the agency’s boss backed the approval, he also called for a key study supporting the drug to be retracted.

In addition, two recent events — passage of the 21st Century Cures Act, a major health bill meant to spur innovation and speed the delivery of new drugs, and the surprise election of Donald Trump as president — have sparked concerns that the FDA might inch closer to deregulation for the sake of innovation under the new administration.

Perhaps more than in previous White House transitions, confusion and uncertainty cloud the FDA’s future.

Yet while consumer groups express alarm, some clinicians and policy experts believe a dramatic reversal in the FDA’s core mission is unlikely. MedPage Today spoke with key stakeholders to gauge the importance of eteplirsen’s approval: what it means for patients and the future of the FDA’s review process.

In approving eteplirsen, the FDA had overruled its own advisory committee. The seven members voting against approval did not believe Sarepta had shown adequate evidence that eteplirsen triggered production of the protein dystrophin at a level that was “reasonably likely to predict clinical benefit.” (DMD is caused by a genetic deficiency in dystrophin.)

Moreover, several of the FDA’s senior staff members also saw evidence that patient benefit was inadequate, as documents detailing a months-long dispute between those staff members and Janet Woodcock, MD, director of the Center for Drug Evaluation and Research (CDER), showed. She overruled their conclusions and FDA Commissioner Robert Califf, MD, ultimately sided with Woodcock. Curiously, however, Califf also called for the retraction of a 2013 study that aimed to demonstrate that eteplirsen produces adequate levels of dystrophin, which he called “misleading.”

Insurers also appear to need more convincing that the drug is effective. The investment firm Jefferies found that three of five national payers and eight of 15 regional managed care organizations “denied or restricted coverage for the drug,” according to Gena Wang, PhD, CFA, an analyst for Jefferies. Wang told Endpoints News, a biopharma newsletter, the response was “in line with our expectation of pushback from private payers.”

Unlike private insurers, public payers such as Medicaid do not have the option to omit FDA approved drugs from their formularies.

The ripple effect of the eteplirsen decision could prove damaging to the healthcare system, Diana Zuckerman, PhD, president of the National Center for Health Research, told MedPage Today.“Many drug companies will be submitting applications that they wouldn’t have dreamed of submitting [before].”

Zuckerman said Woodcock’s decision stemmed from sympathy for the patients.

“She approved a drug not realizing that by approving the drug based on evidence that was so obviously inadequate many health insurance companies would just refuse to pay for it.”

Without insurance coverage, families have to pay $300,000 a year for the drug. “The company failed in its responsibility, the FDA failed in its responsibilities, and the patients are paying the price,” Zuckerman said.

If the agency continues to move in this direction, insurers will spend more money to perform their own analyses of product data. Money that could have been better spent on coverage, she added[…]

Read the full article here.

Comments on Guidance for Industry for
Low Sexual Interest, Desire, and/or Arousal in Women: Developing Drugs for Treatment

Docket No. FDA-2016-D-2817


23 December, 2016

US Department of Health and Human Services
Center for Drug Evaluation and Research (CDER)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852

The National Center for Health Research is a nonprofit think tank that analyzes and reviews research on a range of health issues, with a focus on determining safe and effective treatments for diverse patient groups.

Low sexual interest, desire, and/or arousal are problems for many women in the United States, and there are numerous possible causes for this condition. Flibanserin was approved by the FDA in August 2015, despite substantial concerns about the efficacy and safety of the drug. In fact, some of our concerns regarding the research data for flibanserin are addressed in the proposed guidance, such as the reliance on patients’ monthly reports, which raised questions of accuracy compared to daily diaries, and safety studies conducted primarily on men rather than the target population (pre-menopausal women).

We support the FDA’s efforts to provide a guidance to stimulate stronger evidence of efficacy and safety. However, we have several concerns about the draft guidance.

We agree that it is important to study subgroups that represent the target populations for whom the drug is intended for use. The guidance suggests inclusion of both premenopausal and postmenopausal women and recommends subgroup analysis according to the cause of menopause, hormonal contraceptive use for premenopausal women, hormone therapy use for postmenopausal women, age and race. However, there are other key subgroups that were not included. For example, women with hormone disorders such as Polycystic Ovarian Syndrome and women who are severely overweight or underweight may differ in etiologies and treatment responses. These differences may affect the generalizability of the efficacy and safety data from the clinical trials. Therefore, the guidance should also recommend subgroup analysis based on the presence of hormone disorders and BMI. Furthermore, the guidance should emphasize the importance of providing sufficient and reliable data of safety and efficacy for each subgroup separately, rather than comparing the subgroups. It does not really matter if one group benefits more than another; what matters is whether the benefits outweigh the risks for each specific subgroup. Approval should be specifically indicated only for those subgroups where benefits are most likely to outweigh the risks.

Other common drugs, such as over-the-counter medications or alcohol, can interact with the drug under study, and these interactions should be assessed. These potential interactions can be identified by pharmacology or because the drugs are commonly used together. For instance, flibanserin was found to interact with alcohol to cause hypotension and temporary loss of consciousness caused by a drop in blood pressure 46.

While the guidance’s suggestion that trials lasting at least 24 weeks may be sufficient to establish efficacy, a much longer trial is needed to establish long-term safety. This is especially true if the drug under study is intended to be used indefinitely, such as flibanserin. The guidance instructs sponsors to follow the International Council of Harmonisation (ICH) guideline information, which suggests that, in order to observe the frequency of adverse events over this time period, a larger cohort size is necessary 47. However, if the drug shows indications of rare or slow-developing adverse events, it will be important to also increase the length of trial, to evaluate the clinical significance of these potential adverse events and to conduct a risk/benefit analysis based on the findings. For instance, in the case of flibanserin, there were some indications in animal studies that showed a possible increase in malignant mammary tumors. Although it was considered to be an unlikely adverse event, there was enough concern for it to be included in the label. When signals like this occur, studies longer than 24 weeks should be required.

In conclusion, there is a need to find treatments for low sexual interest, desire, and/or arousal in women, as those conditions affect women’s quality of life. However, the scientific standards should be solid, and consistent with long-term use, if that is the intent. If a sponsor does not want to conduct a large enough study to carefully evaluate safety and efficacy for all the known subgroups, the FDA should approve the drug only for those subpopulations that have been found to have benefits that outweigh the risks.

Ethan Pierce, Tap into Morristown
December 19, 2016

Safety Advocates Call the Decision “Well Timed” with the State Assembly Voting Today to Ban the Dangerous Product in New Jersey

TRENTON, NJ – On the eve of a critical vote in the state Assembly today to ban the sale of supplemental baby mattresses in New Jersey, Target announced it is the latest national retailer to remove the dangerous product from its line.

In making this important decision, Target joins a number of retailers who have stopped selling supplemental mattresses because of proven hazards to babies. Toys R Us, Sears, Kmart, Buy Buy Baby and Wayfair have already removed these products in their stores and online. Meanwhile, other major retailers, such as Walmart and Amazon, have, to date, keep selling the product – a known suffocation hazard.

Today, at 1 p.m., the Assembly is set to vote on the legislation (A-1139), which “prohibits the sale of unsafe supplemental mattresses designed for children products.” The bill had moved through the Assembly Consumer Affairs Committee, which unanimously voted for the legislation to be moved to the floor, for a full Assembly vote.

Target made its decision, following repeated requests from Keeping Babies Safe (KBS), a non-profit organization that has been calling for state legislation to remove the product from the market in New Jersey. Simultaneously, KBS has a petition docketed with the U.S. Consumer Products Safety Commission, seeking a nationwide ban. Public comment for the petition was 99 percent in favor of the ban, with mattress manufacturers as the primary opponent.

The Assembly bill is sponsored by Assembly members Jamel Holley, Nancy Munoz and Angela McKnight. Holley, the driving force behind the legislation and a KBS “Legislator of the Year,” lauded the Target decision and urged his colleagues to pass the bill[…]

There are a number of leading consumer and public health organizations closely following the Assembly decision, with the Consumer Federation of America, Consumers Union, Kids In Danger, National Center for Health Research, and Public Citizen all calling for New Jersey to pass a law. The American Academy of Pediatrics, representing 64,000 primary care pediatricians, pediatric medical sub-specialists and pediatric surgical specialists, is also calling for the ban.

Supplemental mattresses are mattresses sold individually, can be bought for use with play yards, and are advertised as safe. According to U.S. Consumer Product Safety Commission data from 2000 through 2013, at least 15 children died while sleeping on supplemental mattresses. These deaths involved a child being wedged between gaps created when the supplemental mattress was added to the play yard or portable crib.

The voluntary standard, ASTM F406-13, acknowledges this risk with a warning label telling parents not to use these mattresses, and instructing consumers to only use the original mattress pad contained in the play yard package.  Still, these supplemental mattresses, seemingly in contradiction to this voluntary standard and the warning label, continue to be sold[…]

The bill bans the sale of unsafe supplemental mattresses intended to be used by children in products, such as non-full size cribs, portable cribs, play pens, and play yards[…]

“The evidence is clear:  These supplemental mattresses can kill children,” says Dr. Diana Zuckerman, Ph.D., President of the National Center for Health Research.  “They should be banned. As someone who was born and raised in New Jersey, I’m proud to see my home state on the forefront of this important issue.” 

The voluntary standard (ASTM F406-13) for play yards provides that mattresses in children’s products meet certain standard consumer safety specifications, such as setting forth the allowable thickness of a mattress, selling a product with the mattress included, only using a mattress provided by the manufacturer, and including a warning about the risk of infant suffocation.

Despite these requirements and standards, supplemental mattresses continue to be marketed to consumers for use in certain cribs, play pens, and play yards and present a risk to babies of injury and death.

Read the full article here.

Diana Zuckerman, PhD, American Journal of Public Health   
December 16, 2016

The public health community welcomed the Obama Administration with enthusiasm. The George W. Bush Administration had been criticized for having “made decisions about important public health issues based solely on political considerations, not scientific ones.”1 Even President Bush’s former surgeon general, Richard Carmona, told a Congressional Committee that he was muzzled on a wide range of topics, including stem cells, sex education, and secondhand smoke.1 The Obama Administration reversed that bias on a wide range of health issues, but one federal agency has been a clear disappointment to many of us who have watched it closely: the Food and Drug Administration (FDA).

The Obama Administration has been a strong voice on many public health issues, such as tobacco control, healthful eating, childhood obesity, campus sexual assaults, and reproductive health. The Affordable Care Act (ACA), despite some disappointments, is still viewed as a major accomplishment.

But while other federal agencies strengthened their scientific credibility, the FDA caved in to political pressure by approving new medical products based on lower standards of scientific evidence. Given the antiregulatory policies of previous Republican Administration, will the Trump Administration also align with industry against public health? Or will it take a more populist position, protecting patients against a system that seems rigged to protect corporate profits?

Food and Drug Administration Promises

Shortly after being named FDA Commissioner, Margaret Hamburg and her deputy Joshua Sharfstein wrote an unprecedented editorial, “The FDA as a Public Health Agency.”2 They addressed the dilemma that the agency is accused of having only two speeds for approval decisions: “too fast and too slow.” They admitted that because new products are approved on the basis of relatively small studies, safety problems are more likely to emerge later, when the products are widely used. But, they pointed out the other side of the debate: “people with life-threatening diseases have no time to wait.” They promised a “public health approach [that] recognizes that the potential good of a new medical product or policy must be balanced against the potential harm.”2(p2494)

The editorial also promised the FDA would be transparent, providing the data on which it bases its regulatory decisions and explaining its decision-making process to the public. They agreed to attack public health problems by collaborating with other federal agencies.

Despite their well-thought-out analysis, several major public health problems have worsened in the last eight years that are linked to the FDA. Pundits predict these trends will worsen under the Trump Administration, but since the Trump campaign expressed concern about the costs of medical treatment without specifically supporting the views of Big Pharma, it may be possible to reverse rather than worsen the lowering of standards at the FDA.

Faster and More Expensive Cures

Congress has repeatedly criticized FDA approval as too slow, holding hearings and considering legislation focused on that message. Despite that pressure, FDA has the authority and responsibility to enforce the law requiring medical products be scientifically proven to be safe and effective. Instead, they increasingly ignored the importance of replication as a key principle in science, frequently approving treatments based on one clinical trial rather than two. Contrary to the Obama Administration’s commitment to cure cancer, the FDA approved many cancer drugs based on shorter-term studies of smaller numbers of patients, usually allowing companies to study outcomes measured by biomarkers rather than meaningful clinical outcomes such as survival or fewer days in the hospital.3 The FDA required postmarket studies to shore up that weaker scientific evidence, but only a small minority reported a significant clinical benefit.3 The problem of unproven treatments is not unique to cancer drugs; once a drug or medical device is on the market, it stays there for many years even when its safety and benefits are not confirmed.4,5 This contributes to skyrocketing health care and insurance costs, as patients pay for expensive new treatments that are ineffective or inferior to older, less expensive treatments.

Unproven Medical Devices

The Obama Administration made a promising start in 2008 when they selected a new director to oversee the FDA Center for Medical Devices and Radiologic Health. The Center had never received the resources or attention it deserved, and in 2008, fewer than five percent of medical devices, including the minority of implants, were required to be tested in clinical trials. The FDA sought advice from the Institute of Medicine (IOM), which issued a report in 2011 referring to the major FDA pathway for getting devices on the market, called the 510(k) process, as “fatally flawed” because it did not require evidence of safety or effectiveness.

Paradoxically, the scathing IOM report dramatically halted FDA reforms of device standards rather than inspiring a public health–oriented change of standards. The IOM report concluded that the defective 510(k) pathway could not be fixed. One possible solution could have been to move more devices to the more stringent approval process, requiring clinical trial evidence of safety and effectiveness for all life-saving and life-sustaining implanted devices, such as cardiac implants, spinal implants, and joint replacements.

Instead, the FDA took the opposite approach, changing the definitions to justify not requiring evidence of safety or effectiveness for life-saving devices. For example, the FDA had previously claimed that many spinal implants were eligible for 510(k) reviews because they were “moderate-risk” rather than “high-risk.” The FDA now categorizes both moderate-risk and high-risk devices as appropriate for the no-clinical-trials-required standards of a 510(k) review. The more stringent review that requires one clinical trial, which FDA used for fewer than five percent of medical devices in 2008, is used for even fewer devices today. And despite promises of transparency, no scientific evidence for most devices is available to the public.6

Opioid Abuse

The FDA has been strongly criticized for approving opioid drugs that have been widely abused, resulting in an epidemic of abuse and fatalities. The FDA is clearly not the major culprit, but has the epidemic been exacerbated by FDA decisions? The FDA has been criticized for approving opioids that claimed to have abuse-deterrent properties, even when patients could easily find ways around those deterrents. And, although the FDA is not supposed to interfere with the practice of medicine, the law encourages the FDA to use risk-mitigation strategies (called REMS) to reduce misuse of drugs that could be harmful. The FDA spent years developing REMS that require physicians to be trained on how to reduce inappropriate opioid prescribing, but those REMS were never proven effective.7 It has become increasingly obvious that those REMS aren’t working well. It wasn’t until 2016, after several US senators cited the opioid epidemic as a reason to reject the nomination of Robert Califf as FDA Commissioner, that the FDA announced changes in its opioid policies. None involve working with other federal agencies that are also involved in trying to reduce opioid abuse.

Health Disparities

Under the Obama Administration, health disparities have been a major public health concern. But, like the administrations before them, the strategy has been to improve access to medical care, with no attention to the FDA’s failure to require diversity in clinical trials or statistical analyses to determine if new drugs or medical devices are safe and effective for major demographic subgroups. A recent analysis by the National Center for Health Research found that most drugs or devices are not adequately studied on people of color or patients older than 65 years; this means that treatment safety and efficacy is usually unknown for most Medicare patients and many Americans ( Although women are almost always included in clinical trials, almost half the studies did not analyze data to determine if the drugs or devices were safe and effective for women (

Issues Have Gotten Worse

Health disparities, the opioid epidemic, and the skyrocketing costs of prescription medical treatments that are undermining the ACA, Medicare, and Medicaid, are three major public health issues. Those issues have gotten worse, not better, in the last years of the Obama Administration’s FDA. Harmonization with other federal programs has not improved.

The FDA will need to dramatically change course in the next administration to address those public health problems. And while strengthening patient safeguards may not seem a likely priority for a Republican Administration, candidate Donald Trump promised to do things differently. Unfortunately, his only words about FDA focused on the need for “new and innovative” treatments, rather than safe, effective, or affordable ones. Since he apparently isn’t beholden to pharmaceutical and medtech company contributions, can he be persuaded to follow through on his populist promises by helping patients get what they really want—treatments that work and don’t destroy their quality of life? Those are the criteria that previously made FDA approval the gold standard for the world.

Read original article here.


The 21st Century Cures Act has been lauded as a bipartisan success. It’s actually the result of a long war on drug regulation.

Christmas came early for the pharmaceutical industry this year. Last week, the Senate followed the House in passing the 21st Century Cures Act. Though this bill has been lauded by liberals for providing much-needed funds for medical research, its real impact will be elsewhere. Whereas drug approval traditionally required the demonstration of real clinical benefit in a randomized clinical trial, under the Act drug firms will increasingly be able to rely on flimsier forms of evidence for approval of their therapies (incremental steps in this direction, it is worth noting, have already occurred). The Act, by reconfiguring the drug regulatory process, lowers the standards for drug approval—a blessing for drug makers, but an ill omen for public health.

In the Senate, a grand total of five senators—including Bernie Sanders and Elizabeth Warren—voted against it. The media, meanwhile, has for the most part done a poor job dissecting its actual contents. As a result, few now realize how detrimental the act is likely to be for drug safety, or appreciate the mix of conservative ideology and pharmaceutical industry greed underlying the longstanding campaign that brought it to fruition.

The thinking behind the 21st Century Cures Act—and likeminded proposals—goes something like this: In the twenty-first century, the pharmaceutical industry—driven by the profit-motive—continues to do a fine job innovating new therapies. Far too often, however, they are being held back by risk-adverse, slow-moving FDA bureaucrats with outdated standards for approval. “Modernize” the FDA—release the cures! Yet if the law did nothing other than weaken FDA standards, it may not have passed: Liberals understandably embraced the act’s new NIH funding, its mental health provisions, and its support for state anti-opioid programs. For Democrats, it also represented the sort of bipartisan “victory” that shows that all is not gridlock in Washington, after all.                

Yet this thinking is flawed on multiple levels: “We need to remember,” as former editor-in-chief of the New England Journal of Medicine Marcia Angell wrote in her 2004 pharmaceutical exposé, The Truth About the Drug Companies, “that much of what we think we know about the pharmaceutical industry is mythology spun by the industry’s immense public relations apparatus.” First among these myths is the notion that the status quo of private sector drug research and development is the best of all worlds. On the contrary, as Angell put it, “me-too” drugs—lucrative, duplicative agents that do not improve on existing therapies—are in fact the “main business of the pharmaceutical industry.” We can’t rely on the profit motive to bring forth new cures, when it’s just as easy for companies to make big profits by redesigning or tweaking drugs that already exist.

Second, the notion of a slow-moving, risk-adverse FDA is wrong: If anything, the agency’s drugs review process is sometimes too hasty, while its standards of evidence for approval are frequently too lax. Consider, for instance, two recent studies of new cancer drugs. The first—published a year ago in JAMA Internal Medicine by Chul Kim and Vinay Prasad—looked at cancer drugs approved by the FDA on the basis of “surrogate endpoints” between 2008 and 2012. “Endpoints” is a term for outcomes: Hard clinical endpoints refer to outcomes such as survival, where the benefit to the patient is unambiguous. Surrogate endpoints, however, refer to metrics like the change in the size of a tumor on a CT scan. Though a shrinking tumor logically sounds like a good outcome, it is only meaningful if it actually translates into an improvement that an individual actually experiences, like a longer life or a better life. Often, however, that’s not the case: New therapies can change numbers without improving our actual health. This is what Kim and Prasad found: Of the 36 drugs approved on the basis of surrogate endpoints, at least half had no demonstrated benefit.

Perhaps they had other benefits? Or perhaps not. In late November, Tracy Rupp and Diana Zuckerman in the same journal examined these 18 drugs, and found that not only did they not improve survival, but only one had evidence that it improved quality of life (the others lacked data or had no effect, negative effects, or mixed effects). Despite this lack of benefit for either the quantity or quality of life, they note, the FDA withdrew approval for only one drug. Those drugs that either didn’t improve or actually worsened quality of life continue to be sold at an average price of $87,922 per year. Not a bad return for a basically useless drug.

How has this state of affairs come about? At least in part because, as scholar Aaron Kesselheim and colleagues describe in a 2015 study in the British Medical Journal, a total of five new “designations” and one new pathway (“accelerated approval”) have been created since 1983 to lubricate the drug approval process. As they find in their study, as of 2014 some two thirds of drugs are now being reviewed through one or more of these expedited programs, which sometimes allow them be approved more quickly, in some instances with skimpier evidence.

The 21st Century Cures Act will only take us further down this road. Indeed, as Trudy Lieberman has written at Health News Review, the bill is best seen as the “culmination of a 20-year drive by conservative think tanks and the drug industry that began during the Clinton Administration to ‘modernize’ the FDA.” PhRMA—the industry’s primary lobbying group—alone spent $24.7 million on Cures Act-related lobbying, according to data assembled by the Center for Responsive Politics and reported by Kaiser Health News. No less important, however, are the industry’s generous campaign contributions, which have helped construct a compliant and conducive political climate in Washington over the years.

The act reverses many of the protections that stemmed from the 1962 Kefauver–Harris Amendments, signed by John F. Kennedy, which bolstered the Food and Drug Administration’s (FDA) regulatory powers: These reforms meant the FDA could require proof not just that a drug was safe, but that it actually worked, prior to approval.

Read full article here.

In honor of the Giving Season, we are sharing our new public service announcement (PSA) featuring a devoted mother and talented actress, Elisabeth Rohm.

Click here to watch!

cyb6cm5xaaan6ei-jpg-largeYou’ve seen Elisabeth in Law & Order, The Last Ship, and with Jennifer Lawrence in Joy and American Hustle. And she has just joined the cast of CW’s Jane the Virgin.  We hope you will join Elisabeth Rohm by supporting The Cancer Prevention and Treatment Fund with a donation today.

We need your support now more than ever because of the very real threats from Congress. They want to reverse the ban on cancer-causing chemicals in our air, water, and our homes and neighborhoods.  And they are trying to lower safety standards on cancer medications.

Your donation makes a difference, because 98 cents of every dollar goes directly to programs and services, making us one of America’s Best Charities. The Cancer Prevention and Treatment Fund thanks you for your support on Giving Tuesday, and will continue to work tirelessly in the fight against cancer.

Thank you in advance for considering us worthy of your support!

By Peter Sullivan, the Hill

December 8, 2016

The possibility that President-elect Donald Trump could nominate Jim O’Neill, a Silicon Valley investor with no medical background and controversial views, as head of the Food and Drug Administration (FDA) is setting off alarm bells among some healthcare experts.
[…] The most attention has fallen on O’Neill’s comments in a 2014 speech, where he called for changing the FDA’s mission so that it no longer considers whether drugs are effective when deciding whether to approve them. Instead, O’Neill said the agency should only consider whether drugs are safe.
[…] Diana Zuckerman, president of the National Center for Health Research, said that O’Neill’s idea of having the FDA no longer consider whether a drug is effective would cause chaos because insurance companies would no longer be able to use FDA approval to decide which drugs they would cover, and possibly could have to start making those determinations on their own.

“It would throw the entire U.S. healthcare system into turmoil,” she said.

To read the full story, click here

By Teresa Carr, Consumer Reports

December 7, 2016

Congress has passed the most expensive and far-reaching health reform bill since the Affordable Care Act in 2010.

The 21st Century Cures Act, which garnered widespread, bipartisan support in both the House and Senate, is expected to be signed into law by President Obama soon.

The bill signifies an investment of billions of dollars over the next decade to fight cancer, prevent and treat brain disorders, and harness enormous amounts of data to develop individualized treatments based on a person’s environment, genes, and lifestyle.

But the bill also lowers the bar for the kind of scientific evidence that companies must provide to gain the Food and Drug Administration’s (FDA) approval for their products. It would mean, for instance, that in some circumstances the FDA could rely in part on individual patient experiences with a drug or device, instead of evidence from large-scale, randomized controlled clinical trials.


Faster Drug Approvals, But Lower Standards

The Cures Act loosens requirements for how drugs are studied and approved that have been in place since 1962.

Here’s how it usually works: A company submits evidence from studies done under controlled conditions, comparing patients who received the treatment with those who didn’t. The studies typically have to show that people who were given the new drug lived longer or felt better than those who didn’t get it.

This kind of research can be expensive and time-consuming for drug companies to collect, says Diana Zuckerman, Ph.D., president of the National Center for Health Research, a nonprofit think tank focused on health research. Cancer drugs, for example, can take several years to show that a drug improves survival.

The Cures Act calls on the FDA to approve some drugs more quickly, based on less thorough testing. The problem with this, says Zuckerman, is that “getting drugs to market faster doesn’t help consumers at all if they turn out not to work or causes them harm.”

To read the full article click here

Testimony of Dr. Jay G. Ronquillo
FDA Public Workshop on The Role of Hospitals in Modernizing Evidence Generation for Device Evaluation

December 5, 2016

Thank you very much for the opportunity to speak today.  My name is Dr. Jay Ronquillo and I am speaking on behalf of the National Center for Health Research.  I am a physician who trained at Massachusetts General Hospital, have two engineering degrees from Cornell, a Master of Public Health from Harvard, and a master’s in biomedical informatics from Harvard Medical School.  These are the perspectives I bring with me today.  Our research center analyzes scientific and medical data and provides objective health information to patients, providers, and policy makers.  We do not accept funding from the drug or medical device industry and I have no conflicts of interest.

Hospitals are expected to play a key role in evidence generation and adverse event reporting for the National Evaluation System for Health Technology.  While the collection and analysis of real-world data will be important for post-market surveillance of medical devices, the quality of information being collected and used for decision-making must be focused on making patient safety a priority.

Software is a critical component of many medical devices, from implantable cardiac pacemakers, to drug infusion pumps, to health IT like electronic health records and clinical decision support.  However, software-related errors for medical devices are complex.  They can be hard to identify and clearly separate from other types of problems, such as human error or mechanical failure.  Software vulnerabilities also represent a growing cybersecurity concern that could directly affect individual patients and even populations.  As a result, accurate risk assessment and adverse event reporting will require clear guidance regarding how all types of software malfunctions are measured, monitored, and could impact patient care.  We currently do not have that guidance today.

Similarly, real-world evidence collected from new data sources, such as electronic health records (or EHRs), is expected to be central to the National Evaluation System for Health Technology.  However, EHRs in their current state are not very usable or user-friendly, making it difficult for hospital staff to interact with data in a structured format without hindering patient care.  For example, the FDA medical device database has reported several thousand EHRs in oncology and anesthesiology that were recalled due to software flaws over the last five years.  These defects reportedly caused incorrect drug dosage calculations or displayed medical data for the wrong patient.  Unless there are robust regulatory safeguards for improving the design, development, and implementation of EHRs, poor system usability and interoperability will limit the quality of surveillance data being collected as well as any conclusions or decisions based on that data.

In summary, we recognize the potential value of a National Evaluation System for Health Technology that leverages real-world evidence from hospitals.  However, the complexity of software errors, along with the limitations of current EHRs could limit the quality of evidence being generated and used for post-market surveillance of medical devices.  We recommend clear guidelines on the regulation of health IT, and stronger safeguards to ensure that all medical devices, along with their software and data, remain safe and effective for patients.

Thank you again for the opportunity to speak today and for consideration of our views.

By Gabe Alpert, Barrons

December 3, 2016

To get drugs to market faster, the Food and Drug Administration has increasingly allowed drug trials to use “surrogate endpoints,” such as a lab test that might predict an eventual clinical benefit. After the drugs reach market, companies must then perform studies to ensure the drugs actually do provide the intended clinical benefit. If not, the FDA is supposed to rescind approval.

FDA follow-through hasn’t happened, says a study in an American Medical Association journal by Diana Zuckerman and Tracy Rupp, both of the National Center for Health Research, a Washington, D.C.–based think tank.

To read the full article, continue here

Katherine Ellen Foley, Quartz

December 1, 2016

Late on Nov. 30, the US House of Representatives voted in sweeping favor across both sides of the aisle (392-26) on a $6.8 billion medical research bill. It’s expected to pass in the Senate, and it has support from the Obama administration.

The 21st Century Cures Act is great for medical research[…]

All of this medical research spending, though, came with a regulatory compromise. Tucked in the folds of the bill were a number of new laws that allow the US Food and Drug Administration (FDA) to speed up the approval process for a range of treatments.

For example, the Cures Act allows for the expedited approval of new uses of drugs that had been approved previously for other conditions with just anecdotal case studies providing evidence that they work, instead of the usual randomized clinical trials. On the one hand, this means that treatments could reach patients more quickly, and save more lives. But on the other, it means that patients could be exposed to therapies whose risks aren’t completely understood.

There’s also a section of the Act that “expedite[s] the development and availability of treatments for serious or life-threatening bacterial or fungal infections in patients with unmet needs.” That sounds great in theory, but in practice, these drugs might be approved for use in specific patient populations without ever being tested in those people.

According to NPR, some 1,445 lobbyists from 400 organizations worked to sway lawmakers on this bill. Over 1,300 were from groups in favor of the bill, including deep-pocketed pharmaceutical companies in favor of the expedited approval process.“It really is a David and Goliath issue of where the money is,” Diana Zuckerman, the president of the nonprofit National Center for Health Research (which did not lobby for the bill), told NPR.

The Act will go to the Senate next week, where it is expected to pass. Notably, though, Democratic senators Elizabeth Warren from Massachusetts and Bernie Sanders from Vermont have vocally opposed it because of the softened regulations. On Nov. 28, Warren called the bill “extortion,” implying the benefits to the patients with additional medical research would be greatly outweighed by the risks of diminished regulation. The same week, Sanders said in a statement, “This is a bad bill which should not be passed in its current form. It’s time for Congress to stand up to the world’s biggest pharmaceutical companies, not give them more handouts.”

Find original posting here.

Diana Zuckerman, PHD Spectrum News

December 1, 2016

Why would anyone vote against “cures,” especially “21st century cures?” That question is the key to understanding how the U.S. House of Representatives, whose members usually can’t agree on anything, overwhelmingly passed a 996-page health bill yesterday — just a few days after the bill, the 21st Century Cures Act, was written behind closed doors.

Here’s why many health policy and consumer advocacy groups — including the National Center for Health Research, where I work — strongly oppose the bill and are asking senators not to pass the bill next week: On the bright side, the bill promises more than $6 billion dollars over the next few years for medical research and to fight the opioid epidemic. On the other hand, the ‘promise’ of that money does not include anything resembling a guarantee that the money will be provided for that purpose.

If the bill passes, those 996 pages of mostly unintelligible legislative language will influence important issues that affect all of us. Most importantly, the bill instructs the U.S. Food and Drug Administration (FDA) to help drug and device companies get their products on the market more quickly. Unfortunately, it does that by loosening and lowering the very scientific standards that have made FDA approval the gold standard for countries around the world.

A perfect storm of lobbying:

More than 1,450 lobbyists pushed to get this bill passed, including many patient groups and others funded by the companies that make prescription drugs and medical devices. Universities and medical schools also lobbied for the bill because they want more funding for medical research.

But physician groups concerned about patient safety, such as the National Physicians Alliance, the American Medical Women’s Association and the American Medical Student Association, oppose the bill. Dozens of nonprofit groups such as the Consumer Reports Safe Patient Project and National Consumers League also strongly oppose the bill. In one surprising twist, some of the same HIV/AIDS activists who have criticized the FDA in years past for being too rigid are lobbying against the bill because it doesn’t do enough to make sure new treatments are safe and effective.

None of these groups oppose all aspects of the bill. They just want Congress to take the time to fix it. That would mean waiting until next year instead of rushing through a bill that nobody has time to read.

The FDA’s job is to review new medical products to make sure they are safe and effective. ‘Safe’ doesn’t offer a 100 percent guarantee: Any treatment can harm some people; if the benefits are substantial enough, the FDA may even approve a treatment that can be lethal.

But on balance, the treatment’s benefits must outweigh the risks for most people. The company that makes a treatment provides all of the studies and information about it to the FDA, and scientists there review that information. For prescription drugs, that review usually entails the FDA scientists scrutinizing data from clinical trials that compare people taking the new drug with those taking a placebo or a different treatment, such as behavioral therapy.

Lower standards:

Traditionally, the goal of clinical trials is to see if a treatment improves the outcome for the participants. Depending on the condition being treated, better can mean living longer (for cancer, for example) or losing a few pounds (for a weight-loss drug) or being more able to enjoy life (for many psychotropic drugs and devices).

However, the new bill wants the FDA to make it easier for companies to prove a ‘benefit,’ by relying on ‘surrogate markers’ of effectiveness. Surrogate markers don’t measure benefits directly, but rather a secondary trait that is believed to predict the benefits. For diabetes, for example, blood glucose level is the surrogate endpoint, but the treatment’s goal is to avoid amputation or extend life. Better glucose levels don’t always predict those health outcomes.

Cancer drugs are a perfect example. Last December, researchers reported that the FDA approved 36 of 54 new cancer drugs more quickly by basing approval on surrogate markers such as tumor shrinkage, rather than on survival benefit1. Years after approval, only 5 of the 36 drugs had been proven to help people live longer, 18 definitely did not, and the manufacturers of the other 13 had not made the results of their studies publicly available (a sign that the drugs probably don’t work).

Our center looked at those same 18 ineffective cancer drugs and found that only 1 improves quality of life, 15 have no proven impact on quality of life and 2 makes quality of life worse. The most expensive of these drugs, costing $170,000 per person, is one of these last two.

Spiraling costs:

The standards for medical devices, including implants and devices that send electromagnetic pulses to the brain, are even lower than for cancer drugs. Device companies are rarely required to conduct clinical trials to prove their product is safe or effective. More than 90 percent of the time, all they need to do is explain to the FDA that their device is similar to another device already on the market.

Even when device studies are required, the companies rarely use a placebo group as a comparison to the new device. Wishful thinking can make almost any new treatment seem effective at first. That’s why so many people pay so much for treatments that don’t seem to do any good at all.

The new bill would make that situation worse, by pressuring the FDA to make it even easier for new, unproven treatments to be sold in the United States. People might end up paying billions of dollars for a wide range of treatments that were approved based on shorter studies of fewer participants and surrogate endpoints. That would increase the already-spiraling cost of insurance coverage and challenge the financial stability of Medicaid and Medicare even more than is the case today.

Don’t be distracted by the false promises of funds for medical research. The reality is that there is too much in this bill that would dismantle the structures that help physicians make informed decisions and keep us all safe.

Read original post here.

Julia Boccagno, CBS News

November 30, 2016

The House passed legislation intended to accelerate the “discovery, development and delivery” of treatments for patients with unmet medical needs on Wednesday evening after spending more than two years in political limbo.

Next it heads to the Senate for a vote. If passed, the 21st Century Cures Act would allocate more funding to the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) that will help them move new drugs and devices through the approvals process more efficiently, says Paul Melmeyer‎, associate director of Public Policy at National Organization for Rare Disorders. […]

What does the bill do?

First introduced by Rep. Fred Upton, R-Michigan, in May 2015, the Cures Act aims to expedite the process of approving and introducing potentially life-saving drug therapies into the market for consumer use, principally by increasing funding to the FDA and NIH. Proponents say that the $500 million dollars allocated to the FDA over the next decade would allow the federal agency to update and expand electronic health record systems to ensure that the new drugs and devices can be delivered to the right patients. […]

In July 2015, the House approved the bill, but the Senate rejected it, sending the bill back to the drawing board. Since then, the bill has been reworked to include $4.8 million dollars to the NIH, which would help advance President Obama’s Precision Medicine Initiative, bolster Vice President Biden’s “Cancer Moonshot” research, and fund Alzheimer’s research.

In a statement following the vote, the White House applauded the House for making “advances in health,” and urged the Senate to do the same. “The bill is not perfect,” the statement read, but noted that the legislation’s investment in mental health and in combating the opioid epidemic surpasses the bill’s faults.

What do critics say?

Dr. Diana Zuckerman of the National Center For Health Research described the Cures Act as a “misnomer to start with.” She cited two issues with the bill: it lowers the standards for medical products, drugs and devices — and the money outlined in the bill is promised, not guaranteed.

Both concerns could have severe implications, she explained, by saturating the market with ineffective FDA-approved drugs and increasing overall medical costs.

“We’re doing the research showing there are a lot of drugs already on the market that don’t work,” Zuckerman said, “And it’s contributing billions of dollars to the cost of Medicare, billions of dollars to the cost of health insurance, and thousands of dollars per patient.” 

She continued: “When insurance companies pay money for drugs that don’t work, all of us have more expensive premiums and more expensive co-pays.”

Those concerns haven’t escaped prominent politicians like Vermont Sen. Bernie Sanders and Massachusetts Sen. Elizabeth Warren. Prior to the vote Wednesday morning, Sanders spoke about the “greed of the pharmaceutical industry,” urging Congress to stand up to corporations instead of giving them “handouts” and “giveaways.”

On Monday, Warren blasted the bill, saying she knows the difference between “compromise and extortion.” And one of her chief complaints echoed the concerns of Zuckerman: Warren described the $4.8 billion allocated to the NIH as a “fig leaf” compared to what was initially requested, $8.75 billion. 

“And most of that fig leaf isn’t even real,” Warren said. “Most of the money won’t really be there unless future Congresses pass future bills in future years to spend those dollars.”

An “effective lobbying campaign”

The 21st Century Cures Act is one of the most lobbied pieces of legislation in recent history. According to the Center for Responsive Politics, more than 1,455 lobbyists representing 400 companies have made their case for, or against, the Cures Act. That equates to three lobbyists for every lawmaker on Capitol Hill.

Zuckerman described the Cures Act as an “effective lobbying campaign,” referring to the idea of exploiting patients’ vulnerability and appealing to people’s empathetic tendencies.

“It’s really unfortunate how desperate patients, and desperate parents of patients with rare diseases, have been used to lobby for a bill thinking that it’s actually going to benefit them when it isn’t,” she said. 

“Anybody who knows anything about NIH research will tell you that the vast majority of research that NIH does is what’s called ‘basic research.’ It takes many, many years to go from basic research at NIH to a product that can be tested on a human being, and even more years before it’s proven to be safe and effective for patients.” […]

Zuckerman, who spent a number of years on Capitol Hill pushing for heath reform and advocating for safe treatments, said that her opposition to the bill doesn’t equate to an apathy for patients and their illnesses. 

“We need to speak not just for the patients who are desperate for treatments, we need to speak on behalf of the patients who have tried treatments that harmed them, and in some cases made their final weeks and months painful and miserable,” she said. 

Read the full story here.

Richard Harris, NPR and WBUR

November 29, 2016 -Updated December 1

The House of Representatives voted overwhelmingly on Wednesday to approve the 21st Century Cures Act, a sprawling bill to fund medical research and revamp how drugs and medical devices are approved by the Food and Drug Administration.

The bill passed on a vote of 392-26. The Senate could vote on the bill early next week. It has garnered support from a wide variety of advocacy groups, industry lobbyists and the Obama administration. One notable dissident is Sen. Elizabeth Warren, D-Mass., who said her colleagues have “let Big Pharma hijack the Cures bill.” […]

Advocates for biomedical research are happy to see billions of dollars in new funding for the NIH and associated projects. (The money will come, in part, from portions of the Affordable Care Act that are on the chopping block following the November election.)

The National Institutes of Health budget has slipped about 20 percent over the past decade in terms of buying power. The 21st Century Cures Act does little to offset that decline, but it does include billions of research dollars for some new high-profile projects, such as Vice President Biden’s push to accomplish 10 years of cancer research in half that time. […]

The bill also promises faster action on potential new drugs and devices. And this is where some of the concerns come in. […]

“There’s no doubt that patients would like to get treatments faster to the market, but they need to be assured those treatments are safe and effective for each of those marketed uses,” Carome says.

The new law would allow a drug approved for one purpose to be used for a related disease without going through the entire approval process, he said. Carome worries that measure could put patients at risk.

The Food and Drug Administration has been working faster on drug approvals, even without the new legislation. Drugs can be approved based on inferences that they work, but without proof of a long-term benefit. Researchers use a “surrogate endpoint,” such as tumor’s response to a drug, even though that response may not lead to a longer or healthier life.

The faster approach doesn’t always work out as planned. Diana Zuckerman, who heads the National Center for Health Research, points to a study that found 18 of the 36 cancer drugs recently approved on the basis of surrogate endpoints turned out not to help people live longer.

“What’s happening is we are flooding the market with medical products that don’t work very well, or we don’t know whether they work,” Zuckerman says.

She has a new study (slated for publication in JAMA Internal Medicine, but not yet in print) showing that often these new cancer drugs don’t even improve the quality of life for these cancer patients, despite the drugs’ soaring price tags.

Zuckerman say she’s concerned that the 21st Century Cures act will make it even easier for drug companies to use this express lane for approval. […]

Read the full story here and here.

Thomas Burton, Wall Street Journal

November 30, 2016

Negotiators still need to hammer out agreement in some areas, including GOP proposals for limiting FDA regulation

U.S. Senate and House negotiators are in final stages of talks toward legislation that would boost funding for the National Institutes of Health, provide states with $1 billion for opioid-addiction treatment and improve access to mental-health treatment.

Continue here

Peter Sullivan, The Hill

November 28, 2016

Consumer groups are cautioning against approval of a medical innovation bill moving toward passage in the House and Senate, warning that it could endanger patients by lowering standards for the approval of new drugs.

Groups like Public Citizen and the National Center for Health Research say the bill, known as 21st Century Cures, would unacceptably lower safety standards at the Food and Drug Administration. 

Proponents of the measure, which has been the subject of months of bipartisan negotiations, say it will speed up the FDA’s approval of life-saving new drugs and devices while ensuring that regulatory standards are still strong enough to ensure safety.

The House is slated to vote on the bill Wednesday, and the Senate is likely to follow soon. A new bill was released Friday night, though it is still undergoing tweaks.

The measure, which is being combined with a less-controversial mental health bill, is a priority for Republican leadership before Congress leaves for the year.

The consumer groups argue that Democrats are being won over by $4.8 billion in new funding over the next decade for research at the National Institutes of Health. Without this sweetener, they argue provisions loosening FDA regulations would never pass muster on the left. […]

However, the consumer groups opposed to the bill point to provisions that would allow the use of more “real world evidence” of a drug’s effects, which is a lower standard than more rigorous randomized clinical trials.

The groups, which have long been opposed to the measure, also warn against provisions allowing for companies to submit summaries of data about a drug, rather than the full data set, when seeking to expand the areas for which a drug is approved.

The bill would also create a faster approval process for “breakthrough” medical devices, which the groups warn would lead the FDA to rush the process.

Diana Zuckerman, president of the National Center for Health Research, a think tank, said overall the FDA provisions in the bill “make it much easier to get FDA approval for things that may not work and may not be safe.”

Furthermore, there are concerns about such a long bill — almost 1,000 pages — being passed in the lame-duck session of Congress after the elections.

Versions of the bill have been debated for more than a year, but the final language was released just on Friday night.

“Congress should not rush to pass this nearly 1,000 page bill before there is time to thoroughly review it and understand the public health consequences,” Public Citizen said in a statement.

The group did note that some provisions it had objected to were removed in the latest version of the bill.

While most of the attention is on concerns on the Democratic side, Sen. Chuck Grassley (R-Iowa) said Monday he would object to passage of the bill unless a provision is removed that loosens reporting requirements for doctors who receive gifts from drug companies.

To read the full article, click here. 

Sydney Lupkin, Kaiser Health News

November 28, 2016

The 21st Century Cures Act set for a House vote Wednesday is one of the most-lobbied health care bills in recent history, with nearly three lobbyists working for its passage or defeat for every member on Capitol Hill.

More than 1,455 lobbyists representing 400 companies, universities and other organizations pushed for or against an earlier House version of a Cures bill this congressional cycle, according to federal disclosure forms compiled by the Center for Responsive Politics. A compromise version was released over the holiday weekend.

The bill includes funding for enhanced cancer and Alzheimer’s research and would boost funding for the National Institutes of Health to $4.8 billion. The bill would speed up the drug and device approval process at the Food and Drug Administration.

Other than major appropriations bills, a transportation spending bill and an energy infrastructure funding bill, the Cures Act garnered more lobbying activity than any of the more than 11,000 bills proposed in the 114th Congress, an analysis of the CRP data shows. It’s also the second-most lobbied health care bill since 2011, following only the Medicare Access and CHIP Reauthorization Act of 2015, which, among other things, overhauled Medicare payments to health providers. […]

Senate Majority Leader Mitch McConnell identified the legislation as a priority after a 2016 election that has cast doubt on the future of the Affordable Care Act. Republican President-elect Donald Trump has vowed to eliminate “red tape” at the FDA but hasn’t specifically commented on the Cures Act. […]

The Pharmaceutical Researchers and Manufacturers of America, or PhRMA, the main trade group for brand-name drugmakers, applauded the House bill’s passage. The group’s lobbying reports naming the bill accounted for $24.7 million in spending by the group, which spent $30.3 million overall. […]

Several nonprofit patient advocacy and research groups have opposed the bill, citing concerns about endangering patients with simplified drug and device approvals.

Beyond the pharmaceutical industry, the bill’s supporters include universities, medical schools and groups representing them, as well as patient groups funded by drug and device companies, said Diana Zuckerman of the nonprofit National Center for Health Research, which has not lobbied the bill but has launched a campaign to convince Congress to “fix” it.

“It really is a David and Goliath issue of where the money is,” Zuckerman said. […]

Hospitals and medical schools, which oppose rising drug costs, supported the bill because the NIH funding could propel grants to medical and research institutions, Zuckerman said. […]


See the full story here. 

Mingxin Chen, MHS

It seems like common sense: patients treated in better hospitals should live longer.  But do they?

A study of the survival rates of heart attack patients treated between 1994 and 2012 suggests that they do.48 The researchers looked at short-term survival but also followed the patients for 17 years. Because the risk of dying from different diseases at different times varies, the study took all those important factors into account. “High-performing” hospitals are defined as those with lower mortality rates. Likewise, “low-performing” hospitals are associated with higher mortality rates. The researchers made sure that any differences they found in life expectancies were due to hospital performance, not factors such as whether the sickest patients go to hospitals affiliated with medical schools rather than community hospitals, for example.  They did this by statistically controlling for the possible impact of patient characteristics such as age, gender, race, and medical history.

Among hospitals with similar types of patients, the results show patient treated at high-performing hospitals lived between 9 and 13 months longer on average compared to patients treated at low-performing hospitals.

This study suggests that hospitals that focus on improving the quality of care and short-term health care outcomes will also be the most likely to help patients live longer. Patients sometimes just want to go to the most conveniently located hospital or the one that they are familiar with. This study shows that choosing hospitals with the best track record of patient care may be much more important.

Richard Harris, NPR

November 28, 2016

Patients and their advocates are getting an ever-larger voice in how medical research is carried out. They participate in the design of experiments and have a greater say in what outcomes they care about most — and it’s not always simply living longer. […]

There are now hundreds of patient groups, focusing on everything from arthritis to  xeroderma pigmentosum. And patient participation in medical research is no longer an informal matter. The concept is written into federal laws, including one that funds the Food and Drug Administration. Since 2013, the FDA has held more than 20 meetings in which patients are invited to lay out what actually matters to them. […]

“I think the fact that they’re holding the events — it’s well-organized and well-planned — indicates that they’re taking it very seriously,” says Kevin Longino, who heads the National Kidney Foundation. Companies seem to develop drugs without regard to what patients actually value most, Longino says, and “I think patients have very little influence with drug companies.”

The FDA historically hasn’t considered its work from the patient’s point of view. It generally starts with a company pitching a new drug and is supposed to answer the narrower question: Is the product safe and effective? Now that the FDA knows what patients care about, it can suggest that drug companies measure those outcomes in the course of testing new products.

The FDA has already demonstrated that it’s paying more attention to what patients want. Earlier this year, after hearing impassioned pleas from parents and their scientific allies, the agency temporarily approved a drug for Duchenne muscular dystrophy, even though the science in hand didn’t provide proof positive that the drug was effective.

Patients considered that a victory. And the company could start selling its drug for $300,000 per patient per year.

“The bad news about what’s happening is that so much of patient input is involving patients who are working with or who are recruited by drug companies,” cautions Diana Zuckerman, who runs a consumer group called the National Center for Health Research.

These companies pour millions of dollars into disease advocacy organizations and provide information to patients. So the patient voices are sometimes inadvertently channeling commercial interests, Zuckerman says.

With that system, patients also hear more about potential benefits than risks, she says. Clearly patients should have a voice in how big a risk they are willing to take, she says, but within limits.

“I think it’s safe to say we need a sweet spot, and it hasn’t been found. And the FDA has not done a good job of finding that sweet spot,” Zuckerman says. […]

To get around this cozy relationship between drug companies and patient groups, Zuckerman runs training sessions to teach people to serve as the voice of the patient, independent of advocacy groups. […]


See the full article here.

Do you think that health insurance companies should be able to refuse coverage for people with pre-existing medical conditions such as cancer or heart disease, or complications from a defective implant?  That is what will happen if Congress repeals the Affordable Care Act without replacing it with something at least as good.  If you want the Affordable Care Act to stay in place until it is replaced, please take 5 minutes to make this call!

When you call a Congressional office, start by saying who you are.  If you are a constituent, say so.  If you aren’t, don’t say so.  If you are a member of an organization or community that the Congressman or Senator might care about, say so.

Call the Capitol Switchboard at 202 225-3121 and ask for the Congressional office you’re calling (e.g. Senator Jones’ office).  Ask to speak to the Health L.A. (legislative assistant).

Hi, my name is [FIRST LAST NAME], and I am a [constituent (and/or member of ORGANIZATION)]. I’m calling to urge your boss to oppose repealing the Affordable Care Act unless it is replaced at exactly the same time with something better.

Luke Winkie, Motherboard by Vice

November 15, 2016


Probuphine isn’t a drug as much as it’s a novel way to administer treatment. In a 15-minute in-office visit, a doctor will insert four white, tube-like flexible implants into a patient’s upper arm. Those implants are each the size of a matchstick and will drip-feed Buprenorphine directly into the bloodstream and up to the brain. The drug will then fasten onto the same neural receptors triggered by heroin or prescription narcotic painkiller use, effectively curbing one’s desire to get high. The implants will be replaced every six months. Think of it like a microscopic IV with no physical traces. The convenience is the primary perk—Probuphine patients don’t have to worry about remembering their pills or refilling their prescriptions. […]

Probuphine can only be prescribed to those who’ve already successfully been on traditional Buprenorphine treatment in order to make sure patients aren’t rushed to mismatched treatment. But the promise is still intriguing. Probuphine was featured in a double-blind, double-dummy phase III clinical trial earlier this year. The trials were overseen by Dr. Richard Rosenthal, a psychiatrist at New York’s Mt. Sinai Hospital, who told CBS Evening News that what excites him most about Probuphine is that it’s “a new weapon in our armamentarium to fight drug addiction.”

“The risk of relapse is reduced,” he added, “because you’re not going to miss a dose.”

Braeburn Pharmaceuticals, the company that manufactures Probuphine, reported “significant superiority for the implant versus the oral formulation” during the trials. The Food and Drug Administration approved the drug in May.

Behshad Sheldon, CEO of Braeburn Pharmaceuticals, told me implant-based medicine could be applied to any chronic condition that requires daily maintenance.

“We have heard of people who use Probuphine to protect them from their future selves,” Sheldon said. “They might think, ‘Right now I’m really determined to not use, but in three days I might slip up. It’s easier if I had to make the choice to take the medicine to not take it.’ There are people who use this medication who like that idea.”

Anyone who’s ever been on long-term medication knows how difficult it can be to stay disciplined between side effects, scheduling, and the loose, wax-and-wane bell curve of the therapeutic duration. But what if, someday, the very concept of a daily prescription becomes a thing of the past? […]

Sheldon said that there are already implant products for people with schizophrenia, depression, and bipolar disorder. According to Sheldon this is where the industry is headed.

“I do think this is the wave of the future,” Sheldon said. “A lot pharma companies are looking [at implants] for things even beyond mental health like long-term infections. There’s even one looking at an implant for diabetes.”

But others aren’t as convinced about the treatment’s efficacy and safety. Diana Zuckerman is a former post-doctoral fellow at Yale Medical School and currently serves as the president of the Washington, DC-based think tank The National Center of Health Research, which recently noted gaps in Probuphine’s trials.

“I think the big issue is the transition,” said Zuckerman, who was not involved with the trials. “People are going to go from taking pills to using this implant. The implant doesn’t work immediately. It takes a while for it to work and settle into the level that it’s supposed to be. And during that time the person is still going to be taking pills, and that becomes a vulnerable time. How many pills should that person take before the implant is really working, and should they continue to take pills after it’s working?“

According to Zuckerman, medical companies rarely have the motivation to do research once a treatment has been approved by the FDA. At the end of the day, her main concern is that a subdermal implant like Probuphine treats opioid addicts with more opioids.

“Obviously we’ve got a huge problem in this country, and we’ve got to find a way, and I think step number one is to prevent people from becoming addicted by having doctors not prescribe [opioids] so loosely,” Zuckerman said. “But step number two is finding out a better way to help people once they’re addicted. We don’t seem to have a good handle on that.”

The National Institute on Drug Abuse estimates that some 2.1 million Americans are dependent on prescription opioids like Percocet and Oxycontin. The Center for Disease Control has called it an epidemic. […]

It can be easy to categorize the US opioid epidemic in a seedy, faraway place where good morals and prudence will always conquer. But opioid dependency finds everyday people. Probuphine is making the disease easier for some of those addicts to live with, but treatment, which costs an estimated $6000 per six-month session, is only one part of recovery. […]

To read the full story, click here. 

John Fauber, Kristina Fiore, and Matt Wynn, MedPage Today

November 16, 2016

Beginning in 2000, the FDA approved four drugs to treat premenstrual dysphoric disorder, a more severe form of PMS.

The hitch: The condition didn’t yet exist.

The FDA approvals started less than 2 years after a private meeting of six FDA officials with four executives from Eli Lilly. The drug company’s patent on the antidepressant Prozac (fluoxetine) was about to expire and officials had a new market in mind. […]

At the time, there was strong disagreement about the condition, which had been listed as being in need of further study by the American Psychiatric Association in the Diagnostic and Statistical Manual of Mental Disorders (DSM). […]

Same Drug, New Name

For the PMDD version of the antidepressant, Eli Lilly changed its green-and-yellow capsule to pink-and-purple, and gave the drug a more feminine-sounding name: Sarafem.

Sarafem won FDA approval and then it approved three drugs from other companies: the antidepressants Zoloft (sertraline) in 2002 and Paxil (paroxetine) in 2003, followed by the birth control pill Yaz (drospirenone/ethinyl estradiol) in 2006.

But those approvals all pre-dated the 2013 decision of the American Psychiatric Association to recognize PMDD as a distinct psychiatric condition — a determination based on the recommendation of a panel on which nearly 70% of the members had drug company ties.

By then, marketing had created demand: For instance, annual sales of Yaz peaked at $782 million in 2009, though sales dropped after that year following reports linking drugs in its class with dangerous blood clots.

The market was built on a disputed condition, inflated estimates of how many women have it, and drugs that can carry severe risks, such as suicidal thinking in the case of antidepressants. […]

Marketing or Medicine?

Like other everyday conditions that in recent years have morphed into mainstream disorders treated by expensive and often dangerous drugs, a MedPage Today/Milwaukee Journal Sentinel investigation into PMDD found evidence of drug company influence — and money — at virtually every step along the way.

What stands out about PMDD is that so much of the effort began long before it was recognized as a psychiatric disorder. […]

Dangerous Drugs

All antidepressants used to treat PMDD — Sarafem, Zoloft, and Paxil — carry the FDA’s most stringent black box warning for increased risk of suicidal thinking.

The drugs also carry a substantially increased risk of causing lack of energy, sleepiness, and decreased libido.

While studies showed antidepressants worked in about 60% of women with PMDD, one clinical trial found basic calcium supplements were effective in 55% of women with premenstrual symptoms.

No rigorous studies have been done that show the long-term effectiveness of the antidepressants.

In practice, they seem to wear off after about 6 months, according to Amanda LaFleur, executive director of the National Association for Premenstrual Dysphoric Disorder.

Meanwhile, she said, for many women the only other approved drug, Yaz, only made symptoms worse.

In two clinical trials, Yaz reduced PMDD symptoms, but not a lot more than a placebo.

Women who took Yaz showed a 37.5-point improvement on a symptom scale that had a maximum of 126 points, but those who get a placebo had a 30-point improvement.

And the drug’s effectiveness beyond three menstrual cycles is not known.

LaFleur said she started her association in 2013 to help women sift through all of their options.

For her part, she suffered from severe premenstrual symptoms since the age of 14, but found the drugs to be ineffective. Two years ago, at age 35, she took a more extreme route — removal of her ovaries.

“Now, I have no symptoms whatsoever,” she said.

Treating PMDD with birth control pills, such as Yaz, that include the drug drospirenone is especially concerning, said Diana Zuckerman, PhD, president of of the National Center for Health Research, a nonprofit think tank that analyzes health research.

The drug’s label warns that women over 35 who smoke should not use Yaz, because of the risk of clots and the increased likelihood of a serious cardiovascular event.

“Safer drugs are available to effectively treat PMDD,” Zuckerman said. “How bad is your PMDD that you are willing to put your life at risk?”

To read the full story, click here. 

Jim Spencer and Joe Carlson, Star Tribune

November 10, 2016

First, health policy expert Diana Zuckerman displayed huge photos of the disfigured, bruised faces of two babies who had been implanted with Medtronic’s Infuse Bone Graft product in their skulls.

Then Zuckerman, who is president of the National Center for Health Research, put up on the same projector screen the language of the Food and Drug Administration’s 2015 warning that the product had never been approved for use in children and could be dangerous for them. The warning does not include the commercial name of the Medtronic product, nor does it include a ban on using it in kids.

“That’s a pretty calm and un-disconcerting warning, especially when they don’t even name the products,” Zuckerman said Thursday during a presentation on the second day of a two-day FDA hearing. The agency called the meetings to gather fresh perspective on a growing conflict it faces in trying to regulate whether companies can use First Amendment rights to promote uses of devices and drugs that have not been studied. […]

The perspectives of injured patients drove home the stakes in an ongoing legal and ethical debate over what companies can say and do when they sell products for uses not described on the FDA-approved labels.

Doctors are legally allowed to use devices and drugs in unapproved ways that they think will benefit patients. The question for the FDA is how far manufacturers should be allowed to go in explaining products for applications the FDA has neither approved nor cleared, a practice known as off-label use.

Zuckerman said the Infuse Bone Graft is “contraindicated” for children. But the FDA’s 2015 warning didn’t ban the product in kids because some children have such significant bone defects or such rare bone disorders that they would be willing to accept the risks.

Although both kids and adults are susceptible to risks from Infuse like excess bone growth, kids are more vulnerable because their bones are still growing and they have less space to absorb unexpected swelling.

Medtronic has repeatedly denied allegations in lawsuits that it promotes Infuse for off-label uses. […]

The parents of Hailey Reuter, whose injury photo was one of the two Zuckerman displayed at the FDA hearing Thursday, have said in a lawsuit that no one informed them Infuse was going to be used in what they called an “experimental” surgery on their 5-month-old daughter at a children’s hospital in Cincinnati.

“Most consumers have no idea when they are given a drug or device off-label,” Minnesotan Kim Witczak told the FDA panel Thursday. Witczak became a patient advocate after her husband killed himself in 2003 after being prescribed the antidepressant Zoloft for insomnia, which she blames for causing his death.

Witczak was among roughly 30 witnesses Thursday, most of whom said companies don’t need more leeway to spread information about product applications on which the FDA has not ruled. […]

Steven Francesco, who said his son died from careless off-label prescribing, believes the answer is a strong commitment to much more pediatric research.

“Seventy to 90 percent of medications prescribed to children is off-label,” said Francesco, a former pharmaceutical executive. “Where you have no data, you have the Wild West.”

For the full article, click here.

Carolyn Y. Johnson, Washington Post

November 14, 2016

A single sentence in President-elect Donald Trump’s health-care platform sends a strong hint to the drug and medical device industry that they may have an easier time getting their products on the market under his administration.

“Reform the Food and Drug Administration, to put greater focus on the need of patients for new and innovative medical products,” his health plan states.

On the face of it, the bullet point may seem almost bland, but efforts to integrate patients’ preferences and encourage innovation often result in proposals aimed at speeding up the process for getting new medicines on the market by easing regulations. Critics argue that such efforts can erode standards that are in place to protect patients from drugs that don’t work and might even be harmful.

“The language … is industry code for deregulation and reducing of safety standards,” said Robert Weissman, president of Public Citizen, a consumer watchdog. “Of course, the general deregulatory rhetoric from candidate Trump is a worry for us, but as applied to FDA, it would be very troubling.”

No one is sure about the precise direction of policy under the Trump administration. But the idea of faster approval of medicines and devices has been popular, meaning this may be one of Trump’s health-plan goals to gain support from both sides of the aisle. The drug industry, which had been preparing to defend its business model and pricing under a possible Hillary Clinton presidency, may now see an opportunity instead to streamline the drug-approval process, which companies have complained can be onerous, bureaucratic and a barrier to competition.[…]

But it may be unwise to read too much into the sentence, given Trump’s unpredictability — and the lack of certainty about who will define his health policy.

“I think the honest answer is nobody knows” what to expect, said Diana Zuckerman, president of the National Center for Health Research. “Some members of Congress owe pharma a favor; we don’t know the Trump campaign is in that position, and they might not be — and that might give them a certain amount of flexibility. The Trump campaign is nothing if not iconoclastic.” […]

For the full article, click here. 

Shannon Firth and Joyce Frieden, MedPageToday

November 9, 2016

WASHINGTON — With the Republicans winning the White House and retaining control of both houses of Congress on Tuesday, healthcare scholars predict big changes in some healthcare policies, although perhaps not as much as feared.

MedPage Today spoke with several policy specialists experts who shared their views of what changes a Trump administration, coupled with a Republican Senate and House, could mean for the healthcare system, including the Affordable Care Act (ACA). […]

Offering a more liberal perspective, Diana Zuckerman, PhD, president of the National Center for Health Research, said,”I’m not sure what will happen to the Affordable Care Act.” She noted that full repeal of Obamacare would be hard with so many people now relying on it, many of whom live in red states.

“A year from now, we could be in a very different situation politically,” she said, acknowledging that the election showed a very divided electorate. “Every president wants to say they have a mandate … it is harder to make very dramatic changes when you’ve got less than half of the vote and when you have a party that is very truly as divided as the Republican party.” […]

For the full article, click here.

Elisabeth Rohm filming

Elisabeth Rohm during the filming of our public service announcement.

It can be easy to forget that actors and actresses are real people with hopes and concerns just like ours. They seem to live in a totally different world on a totally different planet – planet Hollywood to be exact. But they are mothers, fathers, sons, daughters – and they all care about their health and the health of their friends and loved ones.

We were thrilled when Elisabeth Rohm enthusiastically agreed to film a public service announcement for the Cancer Prevention and Treatment Fund. She is particularly interested in our unique work to prevent cancer and keeping cancer-causing chemicals out of children’s products as well as our food, homes, and neighborhoods. As a devoted mother, she shares our concerns that her daughter might be exposed to these chemicals on playgrounds and in toys, soda cans, and even pizza! She has been so excited to be involved and donate her time to our cause, and we are so excited to have her! While she is a dedicated mother and down-to-earth human being, she is also in a unique position as a TV and film actress. She’s been in TV shows such as Law and Order, Hawaii Five-O, and The Last Ship, and in many films, including starring alongside Jennifer Lawrence in American Hustle and Joy. To see her using that kind of fame and recognition for a good cause is truly inspiring.

Elisabeth Rohm on red carpet

Elisabeth Rohm on the red carpet for American Hustle with Bradley Cooper and Amy Adams.

We wrapped up the filming of our video on November 1st, and we are currently in post-production (as they say in Hollywood). We look forward to sharing it with you soon and letting you hear from Elisabeth herself about why she is supporting the Cancer Prevention and Treatment Fund.

Elisabeth Rohm with filmmaker and producer

Elisabeth Rohm with friend and filmmaker Gigi Gaston (right) and producer Anastasia Roussel (left) at the filming of our video.

Testimony of Samantha Watters, MPH

FDA Risk Communication Advisory Committee – Public Forum to Discuss Strategic Plan for Risk Communication and Health Literacy

Thank you for this opportunity to speak today.  My name is Samantha Watters and I’m Director of Communications and Outreach for the National Center for Health Research. Our Center conducts and scrutinizes medical research to determine what is known and not known about specific treatment and prevention strategies.  We do not accept funding from companies that make medical products, so we can be unbiased while focusing on patient-centered and public health perspectives.

We then translate that complicated information into plain language so that patients, consumers, media, and policy makers will understand it.

My background is an unusual one with degrees in Biochemistry, English, and Public Health with a focus in Health Communication. I’ve also written health communication materials at the NIH.  I am well aware that great scientists don’t necessarily know how to communicate that science to the public.  That is our Center’s challenge, as well as the FDA’s challenge.

After reviewing the draft Strategic Plan and numerous current FDA documents, we have several suggestions.

  1. FDA needs more staff who are extensively trained in plain language communication –going beyond the standard computer-based training course that everyone has to take and no one reads. FDA materials do not seem to reflect best practices in health communications.  I don’t know why that is, but there is a lot of room for improvement.  I will say that the FDA’s Office of Women’s Health seems to do a better job of communicating to patients, and other staff at FDA could possibly learn from them and from colleagues at the NIH.
  2. You mention consistency in branding, formatting, and communications coming from FDA, with an understanding of all your potential communication channels. This is good, but while consistency is important, part of tailoring your message to your target audience means varying format, as well as method of dissemination. Many of your current educational materials are not easy to find online or readily accessible, and those that are easy to find are not always easy to read. A lot of people don’t like to read at all, or don’t read well. That’s why it is important to communicate risk information at the 5th grade or 8th grade level. The FDA has not been doing that.
  3. FDA communications to patients need more graphics and fewer words. Just adding a graphic doesn’t necessarily help if it is still full of words, too busy or confusing, or not colorful or engaging. This is also going to change based on the age of your audience, since the size and color of a font, both on paper and on a screen, is crucial for the aging population. There is also a tendency to include a lot of information because, of course, there is so much to know. However, the more focused your piece can be, the better. The more complicated it is, the less likely someone will be to read it at all, let alone to retain what they’ve read.
  1. A major problem for FDA communication is how it has become increasingly promotional, rather than providing objective information about the products that the FDA regulates. Academic researchers have been studying FDA press releases and other materials, and concluded that FDA press releases often are used for promotional purposes and that doctors and patients misunderstand the content.  The underlying message for many FDA press releases is that the FDA has done something wonderful by approving this new product, and the company has done something wonderful by making it.  Information about risks or restrictions are downplayed.  For example, when the FDA approves a drug through the “breakthrough” pathway, the use of the term “breakthrough” in the press release makes the media and the public think this is the best drug available.  The FDA does not adequately convey all the unknowns about breakthrough drugs, which are often approved based on smaller, shorter term studies of surrogate endpoints, rather than clinically relevant benefits, such as living longer or spending fewer days in the hospital.

Sadly, when drugs or devices are found to have life-threatening side effects, the FDA is not likely to send out a press release to warn doctors or patients in a way that gets their attention.  For example, if a product has a brand name, it should be prominently used in those warnings so that patients will know what you are talking about. An example of this would be Infuse bone cement. It is approved for use in adults 18 and older, but contraindicated for children. When it was used in spinal surgery with children, several experienced cranial swelling so severe that their faces became terribly engorged, requiring additional surgery.  Here are photos of 2 toddlers.  And in the next 2 slides are the language that FDA provided years later, to finally warn doctors and patients.  You can see how inadequate that warning is.  Effectively conveying the actual severity of the risk can help prevent tragic off label uses like this.

  1. Similarly, FDA needs to do a better job of preventing misleading information and inadequate explanations of risk in direct-to-consumer ads. Requiring companies to list risks doesn’t necessarily tell patients what they need to know, since most patients will not have the health literacy to understand what a lot of those side effects and risks mean. For example, commercials that start the list of warnings with “don’t take this drug if you are allergic to it” is a surefire way to get viewers to tune out, since it is obvious.  This is true when listing side effects on a drug ad, in a press release, or on labels required for all prescription drugs and devices. The way risk information is rattled off in TV commercials, for example, is clearly done because it is required, not because the company wants patients to pay attention and understand those risks.

Thank you so much for the opportunity to share our Center’s views with you today.


The National Lung Cancer Screening Trial (NLST) found a reduction in lung cancer mortality in high-risk patients aged 55 to 74 who were screened with low-dose computed tomography (LDCT) rather than chest x-ray. This reduction was 20% after 6.5 years and 16% after 7.5 years, with a 6.7% reduction in all-cause mortality after 6.5 years. The US Preventive Services Task Force recommended LDCT screening for people aged 55 to 80 who currently smoke or had quit within the last 15 years and who have a history of at least 30 pack-years of smoking. Advocates are urging Medicare to cover the cost of screening for that same targeted population, but the Medicare Evidence Development and Coverage Advisory Committee recommended against coverage, expressing their lack of confidence that the benefits would outweigh the risks for the Medicare patients likely to be screened. Data that support and refute the advocacy position regarding Medicare coverage for LDCT screening are presented. The likely benefits and risks for Medicare patients are discussed in the context of NLST data, other research findings, comparisons between NLST patients and Medicare patients of the same age and smoking histories, and Medicare policies.

See the journal article here.

Amy Martyn, ConsumerAffairs

October 27, 2016

Makers of medical devices face such little scrutiny from the Food and Drug Administration that even a 2011 Institute of Medicine report, commissioned by none other than the Food and Drug Administration, described the agency’s medical device evaluation process as “fatally flawed.”

Even worse, the FDA has reportedly allowed device-makers to flout the few regulations that they are supposed to follow.

Federal law requires pharmaceutical companies to report any injuries possibly related to medical devices within 30 days of learning about the so-called “adverse event.” But a Minneapolis Star-Tribune report, published last April, details how Medtronic, the world’s largest medical device company, waited years before telling the FDA about more than 1,000 adverse events related to one of its medical implants, Infuse. […]

In a lengthy statement, the FDA tells ConsumerAffairs that it had granted an exemption to Medtronic and defended the company’s actions.

“FDA’s allowance of a summary report in certain circumstances, under the relevant regulation, is both appropriate and in the best interests of the public health,” the statement says in part. “Such summary reporting can create practical efficiencies by reducing data entry and FDA staff review time of information that is already well-understood about a particular device.”

Asked to comment, Medtronic referred ConsumerAffairs to a statement the company published online. 

Multiple companies

A follow-up report published this month details how the FDA similarly accepted late adverse event reports from multiple companies, not just Medtronic, without penalizing the companies.
“When patients have been horribly harmed by medical devices, they’ve notified the FDA. But nothing changes,” Dr. Diana Zuckerman, President of the Center for Health Research, tells ConsumerAffairs. “And, the FDA has not penalized companies that failed to report serious complications to the FDA, as required by law. The FDA’s track record could hardly be worse.”

Zuckerman’s complaints aren’t new. In 2014, three years after the FDA’s Institute of Medicine panel called its regulatory process for devices flawed, Zuckerman lead a separate study claiming that there is scant public research to back up the safety of many FDA-approved medical devices. The agency has repeatedly contested such critical findings.

Criticism invited

But recent actions by the FDA now suggest the agency may finally be taking some of the criticisms of its device regulation to heart. On October 21, the FDA launched a new online program to encourage anyone, from patients to doctors, to report misconduct by medical device-makers.

“The webpage is not in response to any recent news articles,” FDA spokesman Stephanie Caccomo tells ConsumerAffairs via email. “The webpage was developed to provide the public with more information on allegations of regulatory misconduct related to medical devices and provide clear instructions for reporting to the FDA.” For public health watchdogs like Zuckerman, whether the FDA’s new program will have teeth remains to be seen.

The FDA’s new site, “Reporting Allegations of Regulatory Misconduct,”  specifically singles out medical devices and instructs people to report anonymously if they wish to do so. “Anyone may file a complaint reporting an allegation of regulatory misconduct,” the FDA says, with instructions on how to submit complaints via email or hard mail. […]

For watchdogs like Zuckerman, the FDA’s new site soliciting allegations of abuse in the medical device industry is an encouraging step, but only on paper for now.  Though the new policy “sounds great,” she says, “will it make a difference? Will the FDA finally stop treating device companies like their favorite customers and remember that patients and consumers are their most important customers? …More importantly, will FDA finally decide that they will no longer allow device companies to ignore patient safety?”

For the full article, click here. 

Contraception Journal

To the Editor:

The excellent study by Gariepy et al. provides important information about the comparative effectiveness of the Essure device (hysteroscopic sterilization) compared to two different laparoscopic sterilization techniques (silicone rubber band compared to bipolar coagulation).49

According to the Food and Drug Administration (FDA), Essure sterilization is a nonsurgical procedure that is 99.8% effective at permanently preventing pregnancy over 5 years, when used correctly.50 That statistic is apparently based on effectiveness when proper placement and occlusion is achieved. However, the study of Gariepy et al. indicates that if one takes into account the women who do not return for the mandatory hysterosalpingogram, women undergoing an Essure procedure will have a 10% pregnancy rate over 10 years, which is 3-4 times as high as the laparoscopic rates.1

Between 2002, when Essure was approved by the FDA and December 2014, approximately 1500 reports of adverse reactions were reported to the FDA, including pregnancy, hemorrhage, pelvic pain, and menstrual irregularities.2 More than 14,000 women have reported problems with Essure on a Facebook page (!/groups/Essureproblems/) created by U.S. patients to share information and support each other, and our Center has surveyed more than 900 of them. Although not a random sample of women with Essure problems, it is important to note that our survey found consistent patterns of reported complications. The most common include chronic pain in the pelvic area (26%), autoimmune symptoms such as fatigue(27%), hair loss (20%), depression (14%), heavy bleeding (14%), and allergic symptoms (8%). Fifteen percent required a hysterectomy to remove the device.

Gariepy et al. reported that there were more than 650,000 Essure sterilization procedures worldwide, based on industry statistics for Essure kits sold.51 However, physicians and patients have reported to us that doctors often use at least 2 kits per patient because of difficulties in correctly threading the Essure devices in the Fallopian tubes. In addition, “kits sold” includes those not yet implanted. An accurate estimate of the number of women in the US with Essure implants is essential for interpreting the implications of pregnancies and complications reported by US women to the FDA and in the media; a smaller denominator makes the adverse outcomes reported by thousands of women more worrisome to physicians and patients.

There are clear advantages to a sterilization procedure that does not always require hospitalization or general anesthesia, but more require hospitalization or general anesthesia, but more research is needed to determine the rate of long-term complications and effectiveness. Bayer is making changes to Essure, and it is essential that the older and newer versions of the device be carefully studied in long-term research to determine safety and effectiveness. Despite the advantage of a quick recovery, we expect that patients would be much less willing to undergo the Essure procedure if they were told they would have a 10% chance of pregnancy within the first 10 years, as well as the risk for chronic pain and autoimmune symptoms.

Diana Zuckerman 1
Lauren Abla Doamekpor *
National Center for Health Research, 1001 Connecticut
Avenue Suite 1100, Washington, DC 20036, USA

* Corresponding author. Tel: +1-202-223-4000
E-mail address:


To see the original letter, click here.

Anna Muzzucco, PhD, and Diana Zuckerman, PhD, National Center for Health Research

The excellent article by O’Connell et al.52 describes the remarkable utility of silicone bracelets as “chemical exposure monitors,” which can act as a personal data-gathering device to document an individual’s chemical exposures. The authors demonstrate the absorptive capacity of silicone for a diverse array of chemical entities, including pesticides, phthalates, polycyclic aromatic hydrocarbons (PAHs), and numerous other industrial and consumer product chemicals.

An interesting implication of O’Connell et al. is that millions of women and men worldwide already have silicone biomonitoring devices in their bodies,53 since silicone is often used for implanted medical devices. Silicone breast implants, like silicone bracelets, have been shown to absorb chemicals from the surrounding tissue milieu that can be analyzed after explantation.54 Similar to the findings described in O’Connell et al., the authors of the implant study detected chemicals in silicone breast implants in amounts similar to those found in human tissues, suggesting that silicone equilibrates with surrounding tissue and can represent steady-state body burden of such substances. The authors found pesticides, phthalates, flame retardants, and other chemicals. The results were so striking that the authors suggested using removed silicone implants as biomonitoring devices to gain valuable epidemiological information about chemical exposures among these women.

While silicone is used in many implants because it is considered relatively biocompatible and chemically inert, silicone interacts with human tissue and actively absorbs chemicals from the body. In fact, O’Connell et al. notes that silicone absorbs chemicals similar to the way human cells do. An important question is what are the potential implications of silicone retention of these chemicals within the implant? Could the implant act as an additional reservoir for lipophilic chemicals comparable to the function of adipose tissue of the breast?55 While these studies suggest that silicone behaves similarly to human tissue for the chemical entities examined, it is not known if silicone may have a greater or lesser affinity for some compounds based on their properties.

Numerous health problems have been linked to silicone breast implants, including anaplastic large cell lymphomas (ALCL), a rare and potentially fatal cancer that can develop within the scar capsule around the implant.56 Several hypotheses have been suggested for implant-related ALCL, implicating an immunological mechanism likely attributable to tissue response to silicone.57,58 The significance of long-term accumulation of toxins within silicone implants and their potential contribution to these pathologies is unknown, but with approximately 5 million women with breast implants worldwide, this question deserves further study. The accumulation may be especially relevant when the implant leaks, which has been documented as inevitable as the implant ages, but is often not immediately detected.2,59 Although less attention has been paid to the health implications of other silicone implants, such as testicular implants, gastric lap bands, pectoral implants, cheek implants, and calf implants, the same health questions arise. However, silicone gel, which is primarily used in breast implants, may represent a unique situation when the gel leaks and possibly migrates to secondary sites.

We do not yet fully understand the complexity of the interactions between silicone implants and human tissue, but new evidence regarding implant-related ALCL and the results of O’Connell’s study raises questions about the implications for autoimmune and connective tissue diseases among women and men with silicone implants. Given the potential risks of leaking silicone gel suffused with chemicals, clinicians and patients should also be reminded that the FDA recommends that women with silicone gel breast implants undergo breast coil MRI monitoring of their implants three years after implantation every two years subsequently to check for rupture and leakage.2

The authors declare no competing financial interest.

See the original article here.

Sarah Romano

If you or a family member is involved in a contact sport like football, soccer, wrestling, or basketball, chances are you know something about concussions. Maybe you know someone who has had one, or maybe you’ve had one yourself. But, did you know that even one concussion may have serious, short-term and long-term consequences for your brain and can increase the chances that you will have another concussion in the future?

Concussions are defined as a brain injury that causes a change in mental functioning. It’s more than “being dazed” and less than a coma. Sports and bicycle accidents account for most concussions in children between 5 and 14.60 A concussion does not necessarily cause loss of consciousness, but if it does, unconsciousness may last only a few seconds and not be noticed. Other symptoms, which may not be noticed right away, are:

  • Vacant stare (dazed, befuddled facial expression)
  • Delayed responses (slow to answer questions or follow instructions)
  • Inattention (easily distracted or unable to follow conversations)
  • Disorientation (walking in the wrong direction, unaware of time, date, place)
  • Slurred or incoherent speech (making disjointed or incomprehensible statements)
  • Lack of coordination (stumbling, inability to walk a straight line)
  • Inappropriate emotions (appearing distraught, crying for no apparent reason)
  • Memory problems (repeatedly asking a question that has already been answered or showing memory deficits on formal tests of mental status)
  • Loss of consciousness (paralytic coma, unresponsiveness to stimuli)61

A person who suffers a concussion may not even realize that his or her brain has been injured, especially after a mild concussion. Still, just one concussion puts the child or adult at a greater risk for a second. For instance, one study of high school and college football players found that students who suffered a concussion were almost six times more likely to suffer a second concussion in the five years after the initial injury.62 The second concussion will probably come with symptoms that last longer than the first.63 It is always important to wait until all concussion symptoms are gone before resuming sports or doing anything physically demanding. The National Institutes of Health (NIH) recommends that children with concussion symptoms should “avoid sports, hard play at recess, being overly active, and physical education class.” 64

Certain concussion symptoms indicate that the person will need more time to recover. Anyone who has more than 4 separate symptoms, a prolonged headache, or the presence of fatigue or “fogginess” should take a few extra days after symptoms go away before they return to play after their concussion.65

What if someone suffers a concussion but doesn’t realize it? At this point it’s important for adults to ask the right questions. Children and teens who have suffered a blow to the head are much more likely to report concussion when asked about symptoms in everyday language rather than complicated, medical terms that they may not understand completely.66

Are there long-term effects from getting a concussion? In one study a group of male college athletes who had suffered a concussion more than six months before the start of the study did not differ significantly in neurological tests compared to college athletes who had never had a concussion.4 However, a study of retired professional football players suggested that those players who suffered more than three separate concussions during their career were at a significantly greater risk of having depressive episodes later in life than retired players with no history of concussion.67 And, another study of retired NFL players found that players who had suffered more than three concussions during their careers were at greater risk of developing early Alzheimer’s Disease.8

Concussions and head impacts also cause instant changes. A 2016 study of heading soccer balls showed immediate loss of brain function and lowered memory performance. 68 Fortunately, brain performance resumed normal levels after 24-hours, but the authors point out that they did not evaluate the effects of repeated heading over a period of months or years.

Ultimately the decision about how long to wait after a concussion before resuming practice and playing in games should be made in consultation with a medical professional. Every injury and every player is different. A 2015 study in the journal of Pediatrics found that adolescents who rested 5 days after having a concussion reported more daily symptoms and slower recovery than those who rested the usual 1-2 days. The researchers advised resting for 1-2 days and then gradually starting to resume physical activity.69

All articles on our website have been approved by Dr. Diana Zuckerman and other senior staff.

Testimony of Dr. Stephanie Fox-Rawlings
FDA meeting of the Bone, Reproductive and Urologic Drugs Advisory Committee

October 19, 2016

Thank you for the opportunity to speak today. My name is Dr. Stephanie Fox-Rawlings. I am a Senior Fellow at the National Center for Health Research. Our research center analyzes scientific and medical data to provide objective health information to patients, providers and policy makers. We do not accept funding from drug companies so I have no conflicts of interest.

Nocturia symptoms are caused by a wide range of underlying conditions. It is not surprising that SER120 does not create a clinically meaningful improvement when averaged across all of these conditions.  SER120 may be effective for a subset of people with specific underlying conditions or other characteristics, but the sponsor has not identified that group of patients or their underlying conditions.  To justify FDA approval, a drug should have a clinically meaningful improvement over placebo for the patients to whom it would be prescribed. A general indication for any patients with nocturia would not be appropriate because the drug clearly does not work well for a general population of nocturia patients.

You probably share my concern that the sponsor’s studies excluded patients with diseases/treatments that could reduce the safety of SER120.  And yet, those same patients would consider the drug if it were approved for all adults with nocturia.

There are other safety concerns as well. The studies did not measure possible effects on the underlying conditions. Further studies should determine that treatment with desmopressin does not worsen any of the conditions that cause nocturia or that co-occur with it. In addition, about 11% of patients experienced mild hyponatremia and 1% experienced severe hyponatremia, which requires careful monitoring. For approval, the benefits of SER120 need to outweigh the risks for most patients, but to achieve that we need data on which patients are most likely to be harmed.  And, that information needs to be widely available and mentioned in any advertising or promotional materials.

Patients’ age is also a concern.  Although most patients with nocturia are over 50 years old, there are also many patients under 50, and the safety and effectiveness of the drug could be very different for younger adults.  This may be especially true for premenopausal women.

Only a small number of premenopausal women were studied and they were not analyzed as separate subgroups, so it is impossible to know if the drug is appropriate for premenopausal women. Since 78% of patients were white, the risks and benefits may differ for other racial groups.

There are versions of desmopressin on the market already. It is not clear that this version has a better risk-benefit profile. Whether or not it is better, if approved, SER120 will be much more expensive.   While cost is not FDA’s concern, the skyrocketing cost of older pharmaceuticals that are repurposed is a clear threat to Medicare, the affordability of health insurance, and to public health.  For that reason, this Advisory Committee should make sure that it only approves a drug for an appropriate indication, and that the indication includes the ages and types of patients most likely to benefit.

Unfortunately, information in labels has little impact on prescribing behavior and DTC (direct to consumer) ads tend to minimize those details.

In conclusion, do not recommend this as the first drug approved for nocturia symptoms, unless there is a clinically meaningful benefit and sufficient safety profile for a clearly indicated population. An overly broad indication does not help patients and could harm them.

Thank you.

Dan Diamond, Politico

October 21, 2016

AMA OPENS UP ON OBAMACARE. The president’s hour-long speech on Thursday — his most extensive comments on his signature health law in months — was wonky, feisty and overtly political.

“The Affordable Care Act has done what it was designed to do,” and improved the health system, President Barack Obama said during a speech at Miami Dade College where he repeatedly attacked Republicans. “Why is there still such a fuss? Well, part of the problem is a Democratic president named Barack Obama passed the law. That’s just the truth.”

With open enrollment looming, the speech gave the president a high-profile platform to defend the ACA and frame its legacy. A big part of that pitch was the law’s role in coverage expansion: Obama seized on the nation’s historically low uninsured rate and stressed that Medicaid expansion needed to continue after he leaves office, Pro’s Paul Demko reports. He also reiterated his support for a government-run insurance plan that could bolster competition and called for additional subsidies to make coverage more affordable — ideas that have encountered strong resistance from Republicans.

But the president acknowledged some of the law’s setbacks, particularly the emerging problems related to Obamacare marketplaces. In recent weeks, even Democrats like Bill Clinton and Minnesota Gov. Mark Dayton have begun warning about the challenges to affordability posed by the cost of some plans on the exchanges. “Just because a lot of the Republican criticism has proven to be false and politically motivated doesn’t mean there aren’t some legitimate concerns about how the law is working now,” Obama said.


Bigger problems looming? Many attendees also expressed concern about whether FDA has enough resources to support the biosimilar program. “A major goal is that this should make medications more affordable, and we are concerned that sometimes this isn’t happening,” said Diana Zuckerman of the National Center for Health Research. “We are concerned about any kind of backlog and how slow this process has been so far.”

Zuckerman also expressed concern that user fees don’t include any money to monitor the safety of biosimilars once they’re on the market.


Click here for the full article.

Jim Spencer, Star Tribune

October 25, 2016

WASHINGTON – The U.S. Food and Drug Administration has set up a new webpage where the public can report allegations of regulatory misconduct against medical device makers and marketers.

Among the examples of things that might be reported through the webpage are charges that manufacturers or marketers improperly promoted non-FDA-approved uses of products or that companies failed to tell the FDA about “device-related safety concerns” as federal regulations require.


Besides prohibited off-label promotion and failure to report potential product-related problems, the FDA said the webpage will be a place to expose devices or manufacturing processes that do not meet government standards, to reveal efforts to hide information from the FDA, or to point out regulatory issues with imported medical devices.

Charges will be assigned tracking numbers, assessed by FDA’s Center for Devices and Radiological Health, and given priority based on the potential risk to patients, the FDA spokeswoman told the Star Tribune. Penalties could include inspections of device facilities, warning letters or recalls.

Sen. Amy Klobuchar of Minnesota applauded the new webpage’s “potential to strengthen oversight and safety of medical devices.”

“All patients deserve to know the risks and side effects of a medical procedure and a medical device in order to make informed decisions about their care,” she said.


“This is a good idea, but the question is: what resources will the Center [for Devices and Radiological Health] devote to this to ensure that the allegations of misconduct are followed up and dealt with accordingly?” asked Diana Zuckerman, president of the National Center for Health Research.


For the full article, continue here.

Natalie Rosseau, Kristine Chin, and Simon Essig Aberg
National Center for Health Research

When babies start to get teeth, it can be painful–and no parent wants to see their baby suffer! But teething gels have risks as well as benefits.

Many parents reach for Orajel or Baby Orajel, which are widely available teething remedies which contain benzocaine in the form of gel to soothe sore gums.

What many parents don’t realize is that using benzocaine comes with a serious risk. Those risks aren’t just for babies – even adults can be harmed when they get treated with benzocaine after a dental procedure or intubation. 70

The most serious risk is a rare but serious condition, methemoglobinemia. It’s just as scary as it sounds—it results in low levels of oxygen in the blood and can result in death. The Food and Drug Administration (FDA) has identified 29 cases of methemoglobinemia caused by benzocaine gel, 15 of those cases were in infants (under two years of age).71

When considering any medical treatment for children, parents should always ask the question: “do the risks outweigh the benefits?” Don’t assume that if it is sold in your local drug store or online it must be safe. In the case of benzocaine gel, the serious risks – although rare – are very serious.  Allergic reactions are also possible, which can cause swelling of the tongue, lips or throat.

What Else to Do Help with Teething

Instead of using Orajel or products containing benzocaine, the American Society of Pediatrics advises parents to give their child a teething ring that has been stored in the fridge for some quick relief. Instead of using a drug like benzocaine as an anesthetic, cold items such as refrigerated pacifiers, frozen bananas, and wet washcloths act to relieve pain. Another safe alternative is to simply massage your child’s gums.72

What Symptoms to Watch For

Here’s what to look for if you’re worried that you or your child may be suffering from methemoglobinemia:

  • Pale, gray, or blue-colored skin, lips and nail beds
  • Trouble breathing
  • Confusion
  • Tiredness
  • Headache
  • Lightheadedness
  • Racing heartbeat

If you notice any of these symptoms after using Orajel or any product containing benzocaine, go see a physician as soon as possible.73

Benzocaine: It’s Not Just for Babies!

Even though most of the cases of methemoglobinemia occur in children, adults can also experience the disorder in rare cases. Before using products like Orajel, make sure to talk to your physician. If you have heart disease, are a smoker, or have breathing problems, you are particularly at risk of being harmed by the drug.

The FDA suggests that you should:

  • Use benzocaine gels and liquids only at the recommended dosage and only when needed.
  • Read the label to see if benzocaine is an active ingredient when buying OTC products.
  • Put any products with benzocaine out of the reach of children.
  • Talk to your doctor if you have any concerns.

This article was written in 2016.

All articles on our website have been approved by Dr. Diana Zuckerman and other senior staff.

Karin Price Mueller,

October 20, 2016

Parents want the best for their babies. They try to make sure they’re comfortable. And make sure they’re safe.

Critics say those two goals collide when it comes to adding extra padding — what are called “supplemental mattresses” — to play pens, port-a-cribs, play yards and similar sleep spaces.

Last year, Bamboozled profiled the fight of one mom whose four-and-a-half month old son suffocated while sleeping in a soft-sided play yard. […]

The problem, she said, is that supplemental mattresses aren’t always the right fit. Even ones that advertise they are suitable to go with suitable sleeping spaces can leave a gap between the mattress and the side of the play yard, Davis said.

That leaves enough of a space for a baby to suffocate, Davis and other advocates say.

There are warning labels now, but, Davis said, it’s not enough because the mattresses are still readily available, and they’re often marketed as a suitable addition to play yards.

Now legislators in New Jersey are considering a measure to ban the mattresses, thanks in large part to Davis’ non-profit advocacy group Keeping Babies Safe (KBS).

Davis said the bill will do what KBS has been advocating for all along: to prohibit the sale of supplemental mattresses in New Jersey. […]

Sen. M. Teresa Ruiz, who introduced the bill in the Senate, called the mattresses “a clear and present danger to infants.”

She said cited a report from the Consumer Product Safety Commission (CPSC), which said the products have caused at least 15 deaths over the past 16 years. Still, Ruiz said, federal officials have failed to ban their sale.

CPSC is considering a ban, but the issue isn’t expected on its agenda until 2017.

“These are tragedies that can and should be prevented. The warning labels are not enough, especially when the mattress manufacturers and retailers promote their sale to unsuspecting parents,” Ruiz said. “A host of consumer safety and public health organizations have called for stronger action and I agree with them. We need to act to put an end to this risk with a complete ban on their sale in New Jersey.”

The other groups Ruiz cites as supporters include the Consumer Federation of America, Consumers Union, Kids In Danger, National Center for Health Research, Public Citizen, U.S. PIRG and the American Academy of Pediatrics (AAP). […]


Read the full article here. 

October 13, 2016
Thomas J. Nasca, M.D., M.A.C.P.
Chief Executive Officer
Accreditation Council for Graduate Medical Education
515 North State Street, Suite 2000
Chicago, IL 60654

Re: The Accreditation Council for Graduate Medical Education’s (ACGME’s) Common Program Requirements for Resident Duty Hours in the Learning and Working Environment

Dear Dr. Nasca:

The National Center for Health Research strongly supports maintaining the cap of 16 consecutive hours for shift work for first-year residents. The data on the negative impact of sleep deprivation makes it very clear that the 16-hour cap should be expanded to all medical residents. The caps will help to minimize errors in patient care due to fatigue and compromised concentration and judgment, and will improve the quality of residents’ learning experience. As you know, resident duty hours (RDH) became an issue in 1984 when a college student (Libby Zion) in New York died from a medical error that was determined “to be related to resident fatigue and poor resident supervision.” 74 The current RDH requirements are based on the findings of the 2010 ACGME Task Force on Quality Care and Professionalism. In September 2015, ACGME established a task force to develop recommendations for revising the RDH requirements. We understand that ACGME will publicly release a draft proposal for revision of its resident duty-hour limits and solicit comments from stakeholders in the coming months. However, we are commenting now because we are concerned that numerous physician organizations have advocated lifting the 16-hour cap to allow interns to work for 24 or more hours in a row without sleep.

Previous studies found residents scheduled for 24-hour shifts suffer 61% more needlestick and other sharp injuries after 20 hours on duty, and their risk of automobile accidents doubles when driving home after 24 consecutive hours of work 75. Studies also indicate these excessive consecutive work hours are harmful to patients, with a “greater than 2-fold increase in preventable adverse events.” 76 In addition, a recent study found that “patients taken care of by housestaff working more than 80 hours per week had increased length of stay and number of ICU transfers.” 77

Most healthy adults require a minimum of 5 hours of sleep per 24 hours and ideally 7.5-8.5 hours. When working 24-hour shifts, acute and chronic fatigue impairs residents’ executive and cognitive functions including response time, mood, motivation and initiative 78.

Respondents to an orthopaedic patients’ survey stated that they “felt that resident fatigue was harmful to patient care and that DHRs [duty-hour requirements] were beneficial for both residents and patients.” 79 Although there are conflicting studies on whether DHRs have accomplished their goal of improving patient safety 6, this is more likely to be because 16 consecutive hours are still too long, not because 24 consecutive hours are not a problem. We therefore urge the ACGME to make patients’ lives a priority by keeping the first-year residents’ hours capped at 16 hours of continuous duty and to also apply the cap to all other residents.

We are aware that concerns have been raised about “decreased [resident] experience in patient care, and inexperience in handoffs leading to errors in patient care.” 4 Many newly trained general surgeons are perceived to be underprepared to independently practice general surgery. 3 However, there is no scientific evidence that removing the 16-hour cap will improve that situation.

Diana Zuckerman, PhD
National Center for Health Research

Comments of the National Center for Health Research on
GDUFA II Commitment Letter
October 21, 2016
[Docket No. FDA-2012-N-0882]

Thank you for the opportunity to speak today. I am Paul Brown, Government Relations Manager for the National Center for Health Research. Our Center is a think tank that conducts and scrutinizes research on the safety and effectiveness of medical products.

Our Center does not accept funding from pharmaceutical companies, so we have no financial interests in the medical products and policies we examine.

We respect the FDA and we’re committed to ensuring that it has the resources it needs to make sure that our medical products safe and effective. Given the inadequate appropriations provided to the FDA, we strongly support increasing GDUFA and other user fees to improve FDA’s resources in order to enable the agency to fulfill its public health mission.

Our Center has attended in-person or by phone all of the GDUFA II stakeholder meetings. We agree with GDUFA’s intent, which is to provide additional revenues so that FDA can hire more staff and improve the generic drug review process that will lead to increased access to generic drugs.

We also support the commitment letter’s streamlining of the user fee structure, which will provide a more stable source of funding, and give small companies a financial break on the user fees.

We support the GDUFA II Commitment Letter. It increases user fees by nearly $1 billion over 5 fiscal years from 2018 to 2022. GDUFA I users fees totaled $299 million a year; the fees go up to $493.6 million a year for GDUFA II. The increased fees are needed because the number of Abbreviated New Drug Applications (ANDAs) was under-estimated in GDUFA I. FDA has hired additional staff to meet the increased demand and estimates that it will spend $430 million in the final year of GDUFA I. However, we are concerned that if applications go up to more than estimated in GDUFA II, then even the increased user fee amounts will not be enough to cover the needed FDA staff.

We are also concerned that the fees may not have increased enough to offset the increased workload that the performance goals required FDA to meet. The performance goals are resource- intensive and include tight timelines for the review of original ANDAs and ANDA amendments (both standard and priority submissions), PASs (Prior Approval Supplements) and PAS amendments. They also include short timelines regarding the review of Drug Master Files, controlled correspondence, pre-submission meetings, safety determination letters, teleconferences, and many other deadlines.80

Some of the performance goals in the commitment letter seem unrealistic. For example, the commitment letter states that FDA will meet 90% of most of the performance goals by certain dates, including 6 months for priority major ANDA amendments. That sounds like a New Year’s resolution: “I will lose 90% of my body fat by the end of June.” It’s a nice goal but is it doable? As public health advocates, we need more information on how much time and staff it currently takes the FDA to review and act on these applications. Then we can better estimate how much additional staff and funding it will take to reach these goals. 2

FDA monitors adverse events reports for generic drugs. It is one way that FDA evaluates the safety of drugs after approval. This is important. It is not unusual for side effects from drugs to show up years after the drug has been on the market—when the generic drug is dominating the market or the only drug on the market. That is why GDUFA fees should be used for enhanced safety reviews.

Conflicts of interest
FDA’s primary mission is to protect the public health. GDUFA and other user fees should fund an independent review of how the program has affected overall public health. Have user fees changed FDA’s priorities? Is FDA now treating industry as a customer that it needs to please, instead of acting as a regulator to ensure the public health? Some ethics researchers suggest that user fees are harming, not helping, the FDA’s public health mission.81,82

Process Issues
Let’s start with the positive. We greatly appreciated the GDUFA II Fee Structure Summary. It was concise and easy to read.

But the commitment letter itself needs improvement. The current letter uses too much jargon. Too many abbreviations are not defined, making the commitment letter a chore to read.

Regarding the meetings themselves, FDA needs to improve its outreach efforts. It was disappointing that few consumer, patient, and public health advocates attended the GDUFA stakeholder update meetings. For GDUFA III, we recommend that you hold some of the meetings in Washington, DC at the Hubert H. Humphrey Building. It is difficult for some advocates to attend meetings at FDA’s White Oak campus, which is not easily accessible by Metro.

We strongly support increasing GDUFA and other user fees to improve FDA’s resources in order to enable the agency to fulfill its public health mission. We strongly support the increased resources for hiring review staff. However, we have not seen enough data in the commitment letter to convince us that the GDUFA user fees are adequate to cover the increased workload, especially since the proposed performance goals call for tight deadlines and numerous time-consuming meetings between FDA and industry.

Patients and consumers and the groups and public health advocates should have a greater role in the discussion of how user fees are spent. We do not pay the fees, but we do pay for the medications. We pay directly when we use our own money to buy the products, pay for insurance and federal health programs, or pay for the health problems that may result when the medicines don’t work well. And, as taxpayers we pay for the appropriations that are still supporting a major proportion of FDA resources, including the salaries of FDA staff and officials. We should be at the table or at least in the room when negotiations take place for user fees.

Once again, thank you for the opportunity to speak.

Courtney Nguyen
October 2016

Trying to decide which health insurance policy to choose?

Healthcare websites can be confusing but now there’s an interactive tool to help you choose an insurance policy [1].  It’s a quick and easy way to find out the cost of any healthcare plan for you or you or your family.

Check it out!

You will only need to provide four pieces of information: age, zip code, household size, and household income.  Once you do that, you’ll see a graph showing the estimated costs to you.  There are four to five different kinds of health insurance: bronze, silver, gold, platinum, and catastrophic.  Each bar in the graph tells you how much you would pay for your health insurance every month (premium) and the most you would have to pay for covered services in a plan year (out-of-pocket maximum).

Note: Catastrophic insurance is the least expensive. But it is only for people who are willing to pay for the typical medical expenses when they go to the doctor and depend on insurance only for major medical expenses, such as going to the hospital. Wondering what would happen if you don’t pay for insurance? This tool can even show you how much having no coverage could cost you.


Health Insurance Plan Costs

This graph is an example of what a 45-year-old person would see if he or she has a family size of 4 people and an average annual income level of around $52,600 and lives in Madison, Wisconsin [2]. Of course, the graph will look different for each person based on their own income, location, and family size.

Unfortunately, if you live in a state with state-based Marketplace insurances, you will be sent to their own state insurance websites instead of getting your own personal chart.  Below is a list of those states:

California Colorado Connecticut District of Columbia Idaho
Kentucky Maryland Massachusetts Minnesota New York
Rhode Island Vermont Washington

This tool looks into all available health insurance options, such as Medicaid, the Children’s Health Insurance Program (CHIP), and Qualified Health Plans, to calculate the lowest cost for each plan.  It even calculates all possible discounts for each income level.

Some states lack certain types of health insurance plans so this tool will only give information about the plans available for every zip code.  Also, each state has different rules about age limits for specific plans.  The catastrophic plan, for instance, will not usually accept anyone over 30 years of age.  

For specific questions, contact a healthcare insurance representative.

If you prefer speaking to someone on the phone, call 1-800-318-2596.

But if you prefer speaking to someone face-to-face, visit to find local assistance.


Here are some useful links:


[1] Developed by Social Interest Solutions, a nonprofit based in northern California

[2] based on the Census AC1

Division of Dockets Management (HFA-305)
Food and Drug Administration
5630 Fishers Lane, Room 1061
Rockville, MD 20852

October 11, 2016

Comments of National Center for Health Research
on the draft guidance documents
“Compounded Drug Products That Are Essentially Copies of a Commercially Available Drug Product Under Section 503A of the Federal Food, Drug, and Cosmetic Act”
[Docket No. FDA-2016-D-1309]
“Compounded Drug Products That Are Essentially Copies of Approved Drug Product Under Section 503B of the Federal Food, Drug, and Cosmetic Act”
[Docket No. FDA-2016-D-1267]

The National Center for Health Research, National Physicians Alliance, and Jacobs Institute of Women’s Health support the above draft guidance documents (Docket No. FDA-2016-D-1309 and Docket No. 2016-D-1267), which would place tighter restrictions on the compounding of drugs.

The 503A draft guidance places restrictions on pharmacy compounding by licensed pharmacists and physicians for drug products that are essentially copies of commercially available drugs. The 503B draft guidance places restrictions on pharmacy compounding by outsourcing facilities for drug products that are essentially copies of FDA approved drugs.

The 503A draft guidance states that if the compounded drug has the same active pharmaceutical ingredients (API), the same or similar dosage strength, the same route of administration, and the same characteristics of two or more commercially available drugs, then it is essentially a copy of a commercially available drug—and it cannot be compounded.83  This is a necessary safety precaution because commercial drugs are subject to FDA approval, labeling, and Current Good Manufacturing Practices (CGMP) requirements, which compounded drugs are not. Therefore, compounded drugs should not replace essentially identical FDA-approved drugs.

The 503B draft guidance states that a compounded drug cannot be a copy of one or more approved FDA-approved drugs, or a drug that has not been withdrawn from the market because the drug (or its components) has been found to be unsafe or not effective. Also, it states that if the compounded drug has bulk components of one or more approved drugs, it is essentially a copy of an approved drug.84

The draft guidance documents address compounded drug safety issues that were brought to light during the 2012 fungal meningitis tragedies caused by contaminated compounded drugs that led to more than 60 deaths and hundreds of infections.

The 503A draft guidance describes the conditions that licensed pharmacists and physicians must meet for a compounded drug to qualify for the exemptions under section 503A of the Federal Food, Drug, and Cosmetic Act (FD&C). It states that the pharmacist and physician compounders cannot compound “regularly or in inordinate amounts any drug products that are essentially copies of a commercially available drug product.”

We strongly agree with the FDA that section “503A is not intended to provide a means for compounders to produce compounded drugs exempt from the Act’s requirements that are essentially copies of commercially available drug products.”1 We strongly agree with the 503B draft guidance that compounded drugs from outsourcing facilities should only be distributed to health care facilities or dispensed to patients to fulfill specific patient needs that cannot be met by an FDA-approved drug.

Compounded drugs are an important option for patients who have special needs such as patients with allergies to a pill’s dye, or a senior who cannot swallow a pill, or a child who needs a lower dosage drug than those commercially available. However, compounded drugs are not FDA-approved, which means they have not been subject to FDA’s “premarket review for safety, effectiveness, and quality.”1 That is why we strongly agree with the FDA that “compounded drugs pose a higher risk to patients than FDA-approved drugs,” and that compounded drugs should be subject to severe restrictions for patients who cannot safely use FDA-approved drugs.

Restrictions are needed to:
• Protect patients from less safe and less effective medications. Pharmacists and physicians should not compound drugs for patients who could use commercially available drugs, and outsourcing compounders should not be allowed to sell copies of FDA approved drugs.
• Protect the “integrity and effectiveness” of the FDA approval processes for new and generic drugs. Drug makers would be less likely to invest in new drugs, if a compounder could make substitutes that would not have to demonstrate safety and effectiveness and other FDA requirements.
• Protect FDA’s drug monograph process for over-the-counter (OTC) medications. Restrictions in the 503A draft guidance prevent licensed pharmacist and physician compounders from making drugs without having to comply with monograph standards or Current Good Manufacturing Practices (CGMP) requirements.

The 503A draft guidance spells out when a compounded drug is exempt from being a copy of a commercially available product. It notes that when a drug has been discontinued and is no longer marketed then it should not be considered a “commercially available drug product” and so can be compounded.

Drugs compounded for “an identified individual patient, which produces for that patient a significant difference, as determined by the prescribing practitioner” are exempt from being a copy of a commercial drug.1 And, a compounded drug made to address a shortage need would also be exempt from the copy provision.

The 503B draft guidance lists several exemptions when compounded drugs are not “essentially a copy of an approved drug.” The list includes when a prescriber makes a determination of clinical difference; when an outsourcing facility compounds a drug that differs on its active ingredients, route of administration, dosage strength and form, and excipients; and when the drug appears on FDA’s drug shortage list at the time of compounding, distribution, and dispensing.2

Clear definitions
We are pleased that the 503A draft guidance defines “regularly or inordinate amounts.” We had criticized a 2014 compounding draft guidance for not specifically defining “inordinate amounts.”85 We strongly support FDA’s proposed safe harbor definition of “regularly or inordinate amounts” as meaning when a compounder fills four or fewer prescriptions for the compounded drug in a month.

We strongly support the draft guidance documents because they place tight restrictions on pharmacy compounding under sections 503A and 503B of the FD&C Act to prevent compounding drug products that are essentially copies of commercially available drug products and for those that are copies of approved drugs. Although compounded drugs are needed in certain circumstances (patients with allergies, seniors who need a liquid form of a pill, or children who need a lower dosage amount), these drugs are not FDA approved and are not subject to FDA safety standards (such as performing clinical trials), or labeling requirements. That is why compounded drugs need to be tightly restricted.
National Center for Health Research
National Physicians Alliance
Jacobs Institute of Women’s Health

Kristina Fiore, John Fauber, and Matt Wynn, Medpage Today and Milwaukee Journal Sentinel

October 15, 2016

In 2001, an automated telephone survey paid for by a drug company asked adults a simple, uncomfortable question: How often do you go?

The results produced a striking number: Nearly 17% of adults in the United States — some 33 million people — were declared to have overactive bladder disorder.

And a massive new market for drug sales was born.

Last year, sales of drugs to manage overactive bladder, once simply known as incontinence, reached nearly $3 billion — even though experts in the field say the condition is best managed without drugs at all. […]

While overactive bladder is not a life-threatening condition, the drugs used to treat it have been included in more than 12,000 reports of problems to the U.S. Food and Drug Administration since 2013. That includes nearly 200 deaths and more than 700 hospitalizations. […]

The drugs themselves work moderately better than a placebo, studies show, but not more effectively than non-drug treatments that pose no risk. Such behavioral therapies include bladder training, pelvic muscle exercises, weight loss and fluid management. […]

There are now more than a dozen overactive bladder drugs and treatments on the market, ranging from Botox injections and prescription patches to Myrbetriq, which uses an advertising campaign featuring a cartoon bladder. […]

‘Creating a Disease’

For decades, bladder problems were identified as incontinence or “detrusor instability.” The detrusor is the bladder muscle, which can squeeze too often — or without warning — and lead to feelings of having to go.

The conditions were generally considered a consequence of people getting older.

What followed was an example of illness inflation — an effort driven by drug companies to create or expand the definition of conditions that are part of everyday life and to create guidelines that call for treatment with drugs that are expensive and often dangerous. […]

Starting around 2008, after conducting a “comprehensive evaluation of post-marketing reports,” the FDA began requiring drug companies that make such drugs to add a label warning about such side effects.

Yet the Journal Sentinel/MedPage Today review of FDA records found reports of problems related to the drugs are continuing — since 2013, they were cited in nearly 200 cases, with patients noting confusion, a “confusional state” or feeling abnormal.

The analysis included only cases reported by medical professionals and drug companies. Since drug companies are the only ones that are required to file reports they receive, the real total could be much higher.

Another side effect of many of the drugs linked to confusion is dry mouth. In clinical trials, it was reported in between 20% and 30% of patients, depending on the drug.

Dry mouth can lead to tooth decay, but it also makes people want to drink more — which can be a problem if a person has bladder issues.

Diana Zuckerman, president of the National Center for Health Research noted there is another factor arguing against the use of drugs to treat overactive bladder.

While incontinence episodes are reduced moderately, they are not eliminated. That means protective pads likely still are needed.

“All of these drugs have the potential for serious risk,” she said. “So what’s the actual benefit? To weigh that against risks that can be serious, it’s pretty unimpressive.”

Read the full article here and here.

Natalie Grover, Reuters

October 7, 2016

Health insurer Anthem Inc said it will not cover Sarepta Therapeutics Inc’s drug to treat the rare condition of Duchenne muscular dystrophy (DMD), calling it “investigational and not medically necessary.”

The U.S. Food & Drug Administration approved the drug last month under pressure from patient advocates, even though an outside panel of experts and the agency’s own reviewers had questioned its efficacy.

Sarepta has priced the drug, called Exondys 51, at about $300,000 per patient per year. […]

UnitedHealth Group Inc., the largest U.S. health insurer, does plan to cover the drug, a spokesman said on Friday. Aetna Inc plans to conduct a full clinical review to determine its coverage policy, spokesman T.J. Crawford said on Friday.

Anthem, the second-largest U.S. health insurer, said on Thursday on its website that clinical benefit, including improved motor function, had not been demonstrated by Exondys 51.

“Exondys 51 failed to show it improves health outcomes, and therefore it is not a covered benefit for our members,” Anthem spokeswoman Leslie Porras said in an emailed statement on Friday.[…]

The FDA gave it accelerated approval based on data believed to predict a clinical benefit. Sarepta has to prove that benefit in a subsequent clinical trial, the outcome of which is expected to take at least a few years.

To keep health insurance affordable, companies need to ensure that they are paying for safe and effective treatments, said Diana Zuckerman, president of non-profit organization National Center for Health Research.

“When FDA fails to ensure those standards, then ‘FDA approval’ is no longer a gold standard that insurance companies can rely on,” she said. […]

Read the full article here.

To see the NCHR president’s official statement, click here.

Dr. Diana Zuckerman, PhD
October 7, 2016

Anthem’s decision to not pay for Exondys 51 for Duchenne’s Muscular Dystrophy is not surprising, since there is no solid scientific evidence that the drug works.  This is exactly the concern we had when the FDA approved Exondys 51, over-ruling FDA scientists who agreed that the drug did not meet the requirements of proof that it is effective.  FDA knows that health insurance companies do not pay for experimental treatments, which is one of the reasons why FDA is not doing patients any favors when they approve a drug or device that is not proven to work. We continue to strongly urge Sarepta to do the studies needed to support their claims that patients benefit from Exondys 51, and in the meanwhile Sarepta should ensure that patients in those studies receive the treatment for free.

In order to keep health insurance affordable, insurance companies need to ensure that they are paying for safe and effective treatments.  When FDA fails to ensure those standards, then “FDA approval” is no longer a gold standard that insurance companies can rely on.  Had FDA delayed an approval decision until the company proved that the product is effective (or not), patients could have continued to get the drug for free in clinical trials.  Now that the drug is approved and costs about $300,000/year, who will be able to afford it?    This is a tragic situation for patients and their families, and Sarepta and the FDA share responsibility for it.

This is a major issue of importance to all patients and physicians, not because of this one drug, but because FDA approved a drug despite its own scientists saying the drug doesn’t meet FDA standards for approval.  FDA should listen to patients, using that information to understand what patients need as well as what their positive and negative experiences when using a medical product.  But the bottom line is that FDA should not approve a medical product unless it is proven to be safe and effective based on solid scientific evidence.  Listening to objective, knowledgeable scientists and listening to patients should go together, not oppose each other.

To see Anthem ‘s statement on Exondys 51, see (

Ed Silverman, STAT News

October 7, 2016

One of the nation’s largest health insurers has decided not to cover a controversial Duchenne muscular dystrophy drug after raising doubts about clinical trial data that regulators relied on to approve the medicine last month.

In a bulletin issued to clients on Friday, Anthem reviewed the results from various studies and concluded that Exondys 51, which is sold by Sarepta Therapeutics, is “not medically necessary” and that “the clinical benefit … has not been demonstrated.” Duchenne is a rare disease that confines boys to wheelchairs and condemns them to an early death.

The Anthem decision is significant because the insurer is one of the largest in the country, covering nearly 38 million lives. Not only does Anthem have a significant presence serving individuals and small employers, the insurer is also a major player serving large employers that operate in different states.


The decision is not only a setback for Sarepta, but also amounts to a rebuke of the US Food and Drug Administration, which approved the drug last month after unusually protracted bickering among high-ranking agency staff.

For months, FDA officials quarreled behind the scenes over study results. […] Consequently, Anthem pointed to the uncertainty over these findings to justify its decision.


To some, the Anthem decision is not a surprise.

“The FDA is not doing patients any favors when they approve a drug or device that is not proven to work,” said Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank. “In order to keep health insurance affordable, companies need to ensure that they are paying for safe and effective treatments. When FDA fails to ensure those standards, then FDA approval is no longer a gold standard that insurance companies can rely on.”


Read the full article here.

To see the NCHR president’s official statement, click here.

By Ed Silverman, STAT

September 30, 2016

Despite objections from his own regulators, President Barack Obama Friday signed into law a bill that will briefly extend a voucher program that rewards drug makers for rare pediatric medicines.

As a result, the program will run through Dec. 31 while Congress attempts to further extend the effort for another few years. […]

“This is important because if he hadn’t signed this extension, there would have been a gap in the program,” said Nancy Goodman, who is executive director of Kids v Cancer, a patient advocacy group. “And we need to maintain incentives for companies to develop these types of drugs.”

At issue is the pediatric review program, which was created in 2012 and awards a voucher to a drug maker that wins approval of a treatment for a rare pediatric disease, an area of drug development that was seen as neglected at the time.

The vouchers have gained notice in the pharmaceutical industry because they are valuable – companies can later redeem them when seeking approval from the US Food and Drug Administration for another medicine to treat any illness. […]

Moreover, the newly signed law appears to expand the potential for awarding vouchers. How so? The language may widen the patient population for which a drug may be used by broadening the definition of a rare pediatric disease to include symptoms that emerge any time before 18 years of age.

The language reflected an effort to modify the approach taken by the FDA for viewing certain diseases, according to Paul Melmeyer, associate director of public policy with the National Organization for Rare Disorders. This could also become a boon to drug makers. As the FDA Law Blog noted, “diseases that are extremely severe in childhood but tend to be less severe in adulthood may qualify” for vouchers.

Nonetheless, the voucher program is not universally embraced.

[…] the FDA strongly objects to the program.

FDA officials told the GAO they have not seen any evidence the program has encouraged increased development of drugs for rare pediatric diseases. They also maintained the program hinders their ability to set priorities because agency staff must provide priority reviews of new drugs that would not otherwise qualify.

Nonetheless, the push for an extension is also a timing issue. “It’s just before the election, so it’s not surprising for Congress or the White House” to support the extension, said Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank.

Read the original article here.

By Shayla Love, STAT News

September 28, 2016


Here are some excerpts of the conversation, edited for clarity.

Let me start off by asking: What do you think needs to be improved in the FDA approval process?

Zuckerman: I am increasingly concerned when the standards and criteria for what’s safe and what’s effective is moving more from the pre-market stage, before approval decisions are made, to the post-market stage. More drugs and devices are being approved on a basis of preliminary data, smaller samples, shorter time frames, and sometimes lacking control groups, as what recently happened with Duchenne muscular dystrophy. When that happens, it has a chilling effect on those who are trying to develop treatments and cures. Why would a company spend all of its energy working to do the best possible research if they can get an approval based on a shorter-term study, less definitive data, as long as they encourage patient groups to advocate and lobby for them?

What about the notion that patients and parents living with the disease are really the only ones who can understand what that’s like, and they should be in a position to assess the benefit and risk?

Zuckerman: I think patient perspectives absolutely should be factored in. And they should be factored in at every level. It’s not just important for patients who are wanting a treatment, it’s also equally important for the patients who get harmed. There are some folks in this room who have been harmed by unsafe medical products. They feel like FDA doesn’t listen to them. It’s really important to listen to patients, both the patients who can talk about the benefits of the drugs, or devices, but also the patients who can talk about the risks and the complications.


Going forward, why wouldn’t we be concerned that other companies won’t be emboldened to try and put an application in, and then force the issue? What we saw with this Duchenne episode is that when you have an effective pressure campaign, that can have an effect. And I’m not saying that’s necessarily a bad thing, that introduces a very human element into the discussion, and it can provide additional information.


Zuckerman: I want to get into the specifics of why this particular decision concerned us so much. The scientists all said this drug isn’t proven to work, we don’t know if it works, and therefore it doesn’t meet the legal standards that FDA is supposed to use to make a drug approval decision.

The company said they didn’t have a control group because it would be unethical to have a control group. That is a very frightening statement. If you think that it is not ethical to have a control group to study a drug that you don’t know whether it works or not, then you will never be able to find out if the drug works. You have to have a control group, particularly if you have a small sample like that.

Another big issue is the company announced the same day of the approval that this drug is going to cost $300,000 a year. This is a drug that has to be taken every year for the rest of these boy’s lives. It’s not a cure, it’s management. Now, these patients who have been getting this drug, presumably, for free as part of a clinical trial will somehow have to come up with $300,000 a year to continue to get the drug. I don’t know if insurance companies are going to pay for it, considering that the data show there’s not evidence that it works.


To see the original article, click here

By Katie Thomas, New York Times

September 27, 2016

Public anger over the cost of medical products has burned hot for a year, coursing through social media, popping up on the presidential campaign, and erupting in a series of congressional hearings, including one last week over the rising price of the allergy treatment EpiPen.

But one set of voices has been oddly muted — the nation’s biggest patient advocacy groups. The groups wield multimillion-dollar budgets and influence on Capitol Hill, but they have been largely absent in the public debate over pricing.

To those who have closely followed the drug world, the reason is simple: Many of the groups receive millions of dollars a year in donations from companies behind the drugs used by their members. When they prod drug companies, it is generally for better — not less expensive — treatments.

But critics say that by avoiding the debate over cost, they are failing in their patient-advocacy duties.. […]

Over the last year, pharmaceutical companies have increased prices on medications as varied as breakthrough hepatitis C drugs and little-known generics that have been around for decades. The higher prices have hit American pocketbooks harder than usual, as insurers have increasingly shifted costs to patients.

And for patient groups, loudly addressing the issue can be perilous, as Cyndi Zagieboylo, the chief executive of the National Multiple Sclerosis Society, recently discovered. […]

But as soon as Ms. Zagieboylo started discussing a plan — a modest proposal that involved bringing together drug makers, insurers and others to find solutions — she said she encountered resistance. Other patient groups would not join her, and she said she was told by members of Congress, as well as some of the pharmaceutical companies that donate to her group, to tread carefully.

“We were warned, you know, in a number of ways, just sort of to be careful about this,” Ms. Zagieboylo said. “A couple of pharmaceutical companies mentioned, ‘Boy, we support you, why are you doing this to us?’”

That influence is what makes patient groups so attractive to the drug industry. Some of the largest groups can call on millions of dedicated and highly motivated members and help drug companies by signing up participants for clinical trials, running financial assistance programs and even lobbying government officials for drug approvals or favorable legislation.

“It’s much more compelling when a parent reaches out to their congressman and says, ‘Please contact the F.D.A., because my child is dying,’” said Diana Zuckerman, the president of the National Center for Health Research, a nonprofit that does not accept money from industry.

But she said patient groups were less likely to take positions that might undermine a drug company’s business. “I’ve found almost none that are focused on the public health issues of affordable health care, affordable insurance, a sustainable system,” she said. […]

To read the original full article, click here.

By Natalie Rosseau, Kristine Chin, and Simon Essig Aberg

National Center for Health Research

Representative MarkFitzpatrick (R-PA) and Representative Louise Slaughter (D-NY) introduced the Medical Device Guardians Act during the summer of 2016. The bill would require doctors to report serious problems with medical devices to the Food and Drug Administration (FDA). Currently, doctors are asked to voluntarily report adverse events.  If the bill passes, doctors would join hospitals and manufacturers in having a legal obligation to report problems that occur with devices.  The law protects physicians (and encourages their reporting) by stating that such reports could not be held against a physician in a malpractice suit.

This bill was inspired by the stage 4 uterine cancer of Dr. Amy Reed, a constituent of Rep. Fitzpatrick, as well as the deaths of numerous women, including three in Rochester, NY.  All underwent gynecologic procedures to remove what they thought were benign uterine fibroids using power morcellators.  These devices have high-speed blades that pulverize tissue, and inadvertently spread undetected cancer in the patients, changing what was an early-stage cancer that could be easily removed to a potentially deadly stage 4 (metastatic) cancer. The dangerous use of power morcellators continued for years because doctors did not report that the devices were spreading cancer.  The law would increase reporting by physicians, so that dangerous devices would be identified and removed from the market more quickly.

The National Center for Health Research President, Dr. Diana Zuckerman, asks, “even after doctors knew patients had been killed by power morcellators, why were those deaths not reported to the FDA and made available to all physicians?  Were physicians concerned that they would be held responsible for not realizing that the fibroids contained cancer?”  NCHR supports this bill because there is clear scientific evidence that the vast majority of adverse events from medical products are not reported by physicians, and the percentage may be especially low for devices.  “Since most devices are allowed to be without any scientific evidence of safety,” Zuckerman points out, “requiring adverse events to be reported by physicians is especially important.”

By Natalie Rosseau, Kristine Chin, and Simon Essig Aberg

National Center for Health Research

In June 2016, Representative Mark Fitzpatrick (R-PA) and Representative Louise Slaughter (D-NY) introduced the bipartisan bill, Ariel Grace’s Law.  NCHR president Dr. Diana Zuckerman explains that the NCHR believes the bill is needed because “it provides an important incentive for manufacturers to do a better job of testing the safety of their implants and other devices.  The incentive would be their desire to avoid law suits by patients harmed by defective high-risk medical devices.”

Under the law, the highest-risk devices (called Class III by the FDA) are required to be proven “reasonably safe” and “reasonably effective” in studies of patients (called clinical trials) before they can be sold in the U.S.  However, these studies are often small, short, and without good comparison groups of patients who did not use the device.  In a Supreme Court decision written by Justice Antonin Scalia, the court decided that patients harmed by these devices can’t sue.  “This decision makes no sense, since prescription drugs are held to much higher scientific standards, but patients harmed by them have the right to sue,” explains Zuckerman.  If Ariel Grace’s Law passes, then patients will be able to sue manufacturers whose defective Class III devices have harmed them.

The bill is strongly supported by several patient groups, such as women who have been injured by Essure, a Class III permanent birth control implant manufactured by Bayer. Thousands of women have reported that they were harmed by the device, and questions have been raised about the accuracy of the data regarding safety and effectiveness submitted by the company to the FDA that was the basis of FDA approval.  The bill is named after Ariel Grace Burrell, whose stillborn birth was caused by Essure.

The National Center for Health Research (NCHR) conducts and scrutinizes research and works to improve policies that affect the health of adults and children across the country. NCHR supports this bill, based on its research indicating that even the highest risk medical devices are often not held to stringent scientific standards of evidence of safety or effectiveness.

Sabrina Tavernise, The New York Times

September 19, 2016

The Food and Drug Administration approved the first drug to treat patients with the most common childhood form of muscular dystrophy, a vivid example of the growing power that patients and their advocates wield over the federal government’s evaluation of drugs.

The agency’s approval went against the recommendation of its experts. The main clinical trial of the drug was small, involving only 12 boys with the disease known as Duchenne muscular dystrophy, and did not have an adequate control group of boys who had the disease but did not take the drug. A group of independent experts convened by the agency this spring said there was not enough evidence that it was effective.

But the vote was close. Large and impassioned groups of patients, including boys in wheelchairs, and their advocates, weighed in. The muscular dystrophy community is well organized and has lobbied for years to win approval for the drug, getting members of Congress to write letters to the agency.

A decision on the drug had been delayed for months. The approval was so controversial that F.D.A. employees fought over it, a dispute that was taken to the agency’s commissioner, Dr. Robert M. Califf, who ultimately decided that it would stand.

The approval delighted the drug’s advocates and sent the share price of the drug’s maker, Sarepta Therapeutics, soaring. But it was taken as a deeply troubling sign among drug policy experts who believe the F.D.A. has been far too influenced by patient advocates and drug companies, and has allowed the delicate balance in drug approvals to tilt toward speedy decisions based on preliminary data and away from more conclusive evidence of effectiveness and safety.

“The agency has set a dangerous precedent,” said Diana Zuckerman, president of the National Center for Health Research in Washington. “To prove something works, you have to compare it to something else — a placebo or a treatment. They didn’t do that.” […]

The drug, eteplirsen, uses a technology called exon skipping that seeks to partly correct the genetic defect, allowing muscle cells to produce a somewhat functional form of dystrophin. The drug is applicable to only about 13 percent of Duchenne patients. Other exon-skipping drugs are being developed for patients with different mutations.

Sarepta said the average cost of eteplirsen for a patient would be about $300,000 a year. That is double the cost of most new cancer drugs, according to Dr. Zuckerman.

The drug received accelerated approval, which is reserved for medicines that treat serious diseases and address an “unmet medical need.” The agency defines that as “a condition whose treatment or diagnosis is not addressed adequately by available therapy.”

But even as it approved the drug, the agency also required the company to conduct another clinical trial to confirm the drug’s effectiveness. If the drug maker fails to prove it, the agency said it may “initiate proceedings to withdraw approval.”

During a heated public meeting in April, experts voted 7 to 3, with three abstentions, that the clinical data did not meet the F.D.A. requirements for well-controlled studies necessary for approval. The agency had urged Sarepta, which is based in Cambridge, Mass., to do a larger study with a placebo control to better determine whether the drug worked.

But the company argued that doing so would be unethical and impractical, since early hints of effectiveness meant that parents would no longer enroll their sons in a trial where they might not get the drug. Instead, Sarepta compared the data from the 12 boys in the trial to historical data from patients in Italy and Belgium who were as closely matched as possible in disease characteristics.

Now the company will have to conduct another trial. But Dr. Zuckerman argued that it is more difficult to enroll patients after a drug had been approved, because families will not want to take a chance that their son would be in a placebo group. […]

To see the original article, click here

Division of Dockets Management

Food and Drug Administration

Department of Health and Human Services

5630 Fishers Lane, Room 1061

Rockville, MD 20852


September 14, 2016


Comments of the National Center for Health Research

on the draft guidance

‘‘Factors to Consider Regarding Benefit-Risk in Medical Device Product

Availability, Compliance, and Enforcement Decisions’’

[Docket No. FDA–2016–D–1495]


The National Center for Health Research does not support the current version of the draft guidance, “Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions” because it would clearly lower FDA safety standards for Premarket Approval (PMA) medical devices, which are the highest-risk devices (the guidance does not apply to 510(k), de novo, and other device classifications).

This guidance is designed to assist medical device makers in making decisions regarding “nonconforming product or regulatory compliance issues” (recalls or market withdrawals). It is also written to help the FDA address shortage situations that may occur following an inspection where “non-compliance was observed.”  The guidance clarifies the “benefit and risk factors” FDA and industry may consider regarding “medical device quality and patient safety.”

The guidance states that FDA may consider benefit-risk factors on a “case by case basis,” and “factors may be weighted differently.”  But then it contradicts those statements by saying that FDA intends “to improve consistency and transparency” regarding the factors it considers. Case-by-case considerations do not lead to consistency; on the contrary, they tend to be subjective and as such are also labor and resource-intensive. Since they would likely be subject to bias, we oppose them.  However, if the FDA believes they are needed, industry should pay for them through generous user fees.  Such reviews should not be funded by appropriations that are already inadequate for CDRH.

The guidance also states that “product availability and other medical device compliance and enforcement decisions are generally fact-specific.”  Providing fact-specific data is the best way to provide consistency in reviewing medical devices, which is why we are concerned about some of the other sources of information that the guidance promotes. Our specific concerns are listed below:

Real World Evidence

The guidance states that benefit-risk factors will be assessed using real world evidence but it does not define real world evidence. Anecdotal information from patients and clinicians should not be used, unless the FDA specifically reaches out to gain information from patients who were harmed according to MAUDE reports, and not just to patients affiliated with industry.  Registries can provide useful information, but only if the registries are providing data on representative patients and healthcare providers.  Registries that are only used by physicians that are most actively treating specific types of patients are not able to provide data that is generalizable to the patient or provider population as a whole.

Patients’ Perspective

The guidance states that FDA and industry “can help maximize benefit and reduce risk to patients” by considering patients’ perspectives regarding regulatory non-compliance or device nonconformity.  It states that “FDA may consider…patient perspectives on what constitutes meaningful benefit, what constitutes risk and what tradeoffs patients are willing to accept.” However, the guidance does not state how FDA will obtain patients’ perspectives.  It states that FDA will consider “reliable patient preference information” but it does not define “reliable.”   The track record for FDA is that it primarily depends on the perspectives of patients who were recruited by sponsors.  Those sponsors directly or indirectly influence patients by funding their nonprofit organizations.  The FDA rarely relies on information from patients or consumers who were harmed by medical devices.  They have very different perspectives from the patients recruited and encouraged by the device companies.  Similarly, the Medical Device Innovation Consortium (MDIC) does not include small, independent patient organizations because they are not able to afford the annual fee to join.


The guidance states that “clinicians may find unanticipated ways to use the medical device and additional types of benefit.”  In other words, they will be using the devices off-label. We have heard from many patients who were harmed by off label uses and who were never informed by their physicians that a use was off-label.  FDA should ensure that such off-label uses are accompanied by a clear oral and written informed consent, informing the patient that the device is not approved for the off label indication.  In addition, the FDA should require databases that clearly provide data supporting the safety and effectiveness of these off-label uses. A standardized database is important to provide uniform, consistent, and reliable data.


The guidance states that “medical device nonconformities may directly increase risk or introduce new risks.” We agree.  However, the guidance also states that nonconformity can “include the failure of a medical device to meet its performance specifications even though the device still performs adequately.”  On what scientific evidence does FDA base this statement?  In the “likelihood of risk” section of the guidance, the FDA states that “reliable data” may not exist.

Understanding risks 

In the “patient tolerance of risk” section of the guidance, FDA notes that “patients may not understand device-related risks.”  We strongly agree.  Unfortunately, FDA increases the likelihood that the patient will receive biased information about those risks when the guidance states “a patient’s assessment of risk would be appropriately informed by information from his or her clinician” (emphasis added). Most clinicians receive information about medical devices primarily from the manufacturer, and many also receive consulting fees or other gifts from the manufacturers.  In addition, it is not unusual for a clinician to design a device that they then encourage patients to use.  For those reasons, many clinicians may not be able to provide objective or appropriate information to their patients.

Safety Standards Lowered

The guidance states that FDA should use the “outcome of a benefit-risk assessment to inform decisions related to product availability” due to a recall, safety signal, or medical device nonconformity.

The four bullets listed in this section all suggest lowering safety standards:

  • When should a correction be used instead of a removal?
  • What actionsif any should FDA take regarding nonconforming devices in shortage situations (emphasis added)?
  • When should FDA grant a variance from Quality System regulations during a PMA pre-approval inspection?
  • When can FDA “exercise enforcement discretion” and not take action when a device maker makes a significant change or modification prior to obtain a clearance?

All these questions revolve around issues that would weaken safety standards.  These are not the questions CDRH should be asking.  Where are the questions regarding how to strengthen safety standards?  The list of medical devices that could have benefited from stronger standards is long and includes power morcellators, duodenoscopes, metal-on-metal hips, Essure, and surgical mesh to name a few. And yet, this guidance document states that “If FDA’s benefit-risk assessment indicates high benefit to patients with little risk, FDA may decide to work with the manufacturers to address the underlying issues without initiating a formal compliance or enforcement action.”  Unfortunately, because CDRH requires so little data on safety and effectiveness, it lacks adequate information to make this determination on the basis of scientific evidence.  The adverse medical device reporting system (MAUDE) greatly undercounts device problems, devices are constantly being changed before required post-market studies are completed, and devices are not yet part of the Sentinel program.  This clearly puts patients at unnecessary risks.

Also, it is unacceptable that the guidance states “if FDA’s benefit-risk assessment indicates low benefit to patients with high risk, FDA would likely take formal compliance or enforcement action to address the problem” (emphasis added).  The word “likely” should be removed from that sentence.


For the above reasons, we strongly oppose this version of the draft guidance. It does not enhance FDA’s goal of “protecting and promoting the public health.” We are particularly concerned that FDA is lowering its standards to address possible “shortage situations,” and it is putting patients at risk by allowing device manufacturers to use benefit-risk factors to allow “nonconforming” devices or devices with regulatory compliance issues to remain on the market instead of being recalled.

Carolyn Y. Johnson, Washington Post

September 19, 2016

After months of advocacy and speculation, the Food and Drug Administration today granted accelerated approval to the first treatment for a rare form of muscular dystrophy. The decision pitted the passionate testimony of patients and families against an FDA advisory committee and internal reviewers who weren’t convinced the drug worked.

The approval of Sarepta Therapeutics’ drug, eteplirsen, was a huge, emotionally fraught victory for families with Duchenne muscular dystrophy — one of the most vocal and involved patient communities since the days when HIV patients pushed the agency to approve more drugs. The approval specifies that the clinical benefit of the drug “has not been established” and is contingent on a follow-up clinical trial. The process has been closely watched by parents, patient advocates and biotech investors, and has been seen as an important test case for the effort to integrate the patients’ point of view more deeply into the approval process. […]

The agency usually follows the recommendation of its advisers, whose split vote against approval was met with angry outbursts at the conclusion of a long and contentious meeting in April. The FDA missed its initial deadline to decide on whether to approve the drug and requested more data from the company, fueling a rollercoaster of rumors about whether the drug would ultimately be approved. […]

The study supporting approval was a small trial with a dozen boys who carry a gene mutation that occurs in 13 percent of the 9,000 to 12,000 boys in the U.S. with Duchenne. FDA deemed that the trial did not show improvement on a walking test, but the study did show a measurable increase in dystrophin, the protein that is missing in the disease. There was significant dispute internally at the FDA over whether that increase was likely to be a predictor of a benefit. Internal documents released as part of the approval show that, in an unusual move, the decision to approve the drug was appealed to the highest levels of the FDA — to commissioner Robert Califf. […]

But critics of the decision worry that the approval of a drug — when internal scientists and an external advisory committee have found the evidence unconvincing — could be a slippery slope.

“If this drug can be approved under those conditions, is there any drug that FDA won’t approve?” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit research organization. “This drug was based on the strong lobbying of patients and the company, and time will tell whether it will really help these boys or not, and that has always been the question.”

At a meeting in April, the agency walked a difficult high-wire act, visibly struggling to strike the balance between respecting the views of parents and boys who attributed their health to the drugs while also pointing out that the data showed scant evidence of the positive effects reported. […]


To see the original article, click here

Brett Norman, Politico Pro

September 16, 2016

Drug companies and research institutions will have to publicly report more clinical trial data, including results that show their products or experiments failed, under new policies rolled out Friday by HHS.

The new rules address the fact that researchers have routinely flouted requirements to report data to A final rule from FDA and a complementary policy from NIH clarify which trials must be reported and project a new federal commitment to crack down on violators – threatening fines of $10,000 per day or disqualification from NIH funding.

The widespread failure by institutions to report the data is “simply unacceptable,” NIH Director Francis Collins said on a call with reporters.

“This is fundamentally an ethical issue,” FDA Commissioner Robert Califf said. “This is about maintaining the trust that we have with [clinical trial] participants … that if it doesn’t benefit them, it will benefit others.”

A 2014 review of the results of 400 clinical studies found that 30 percent had not been published or shared with four years after they had been completed. A study earlier this year found that 43 percent of the trials conducted at 51 academic medical centers went unpublished two years after the trial was completed.

The new requirements, which will take effect in January, clarify that all NIH-funded studies must be published on The FDA will also require publication of studies being done on drugs and devices even if they have not yet been approved. Most studies must be reported within a year of completion, but those involving unapproved products could wait up to three years.

FDA and NIH are also expanding the range of information that must be published, including the demographic data on study participants, any adverse events and the original plan for statistical analysis. The latter is meant to combat so-called p-hacking – when the original hypothesis of an experiment fails and researchers massage the data to come up with some other positive finding that is statistically significant.

“We’ve had a problem where people do the trial and don’t like the result, so they do another analysis,” Califf said.

FDA and NIH decided not to require a lay summary of trial results that would enable the public – not trained in statistics – to more easily digest the information that is posted, angering some consumer advocates.

“That is an outrageous decision,” said Diana Zuckerman, president of the National Center for Health Research. “Without it, will not be providing useful information to patients.” The data won’t be useful to physicians either unless it is summarized, she said.

Collins and Califf said they had been uncertain how to keep “bias” from creeping into the summaries, adding that advocacy groups should be able to harness the relevant data and make it available in a way that would be more useful to patients. […]

To see the original article, click here

Anna Edney, Bloomberg

September 13, 2016

The U.S. Food and Drug Administration has a message for doctors: The money you’re taking from pharmaceutical companies may be clouding your judgment.

Research sites where Pfizer Inc. had paid doctors at least $25,000 in speaking, consulting or other fees reported sunnier results for its smoking-cessation drug Chantix, the FDA disclosed Monday. At those sites, doctors studying the drug’s possible link to suicide risk and other behavior changes reported fewer side effects than at locations where colleagues accepted lower or no payments.

The FDA’s findings — part of an agency review of Pfizer’s proposal to drop the most severe consumer warning on the drug’s label — demonstrate the federal government’s concern about the influence of consulting and speaking fees on medical decisions. President Obama’s 2010 health law requires drug makers to report such payments for posting to a public database. The law followed years of efforts by U.S. Senator Charles Grassley, an Iowa Republican, to make drugmakers publicly disclose financial ties to doctors. […]

Diana Zuckerman, president of the National Center for Health Research, part of a coalition of consumer and other groups that petitioned to keep the warning on Chantix, said the FDA staff report shows that it doesn’t trust the “integrity of the data.”

“FDA clearly seems to be saying we can’t trust the results of this study — the way it was coded, the way it was analyzed, and by the way there’s conflicts of interest,” Zuckerman said. […]

Outside experts are scheduled to meet Wednesday to advise the FDA about the necessity of the warning on Chantix. The pill’s “black box” label — the agency’s strongest — currently cites risk of “serious neuropsychiatric events” such as suicidal thoughts or behavior. Chantix generated $671 million in sales last year,according to Pfizer. […]

To see the original article, click here

Testimony of Dr. Stephanie Fox-Rawlings

FDA’s Public Hearing on Draft Guidances Relating to the Regulation of Human Cells, Tissues or Cellular or Tissue-Based Products  


September 13, 2016

Thank you for the opportunity to speak today. My name is Dr. Stephanie Fox-Rawlings. I am a Senior Fellow at the National Center for Health Research. Our research center analyzes scientific and medical data to provide objective health information to patients, providers and policy makers. We do not accept funding from the drug or medical device industry. I have no conflict of interest.

Before coming to the National Center for Health Research, I worked as a developmental neuroscientist at the Children’s National Medical Center. My project was to understand how cells respond to damage, and how neural stem cells respond to changes in their environment to promote recovery. If my work in the laboratory taught me anything, it’s that cells, and especially stem cells, are extremely dynamic. They continuously react to and are modified by their environment. Small changes can greatly affect the way that cells behave. For example, exposing cells to a different growth factor or signaling molecule or even varying the oxygen level can change the number of cells and what they become.

Cells and tissues are much more complicated than drugs and biologics. They are not a simple compound or single protein that can be easily characterized in a lab test. A cell is a living, changing organism. They can move throughout the body and can make other cells change their behavior. Stem cells can even transform into new cell types. Because of this, cells and tissues have an amazing and exciting potential to heal people and cure disease.

But just as these cells have the potential to heal, they also carry the potential to harm. That’s why cells and tissues should be properly tested and regulated before widespread use in patients.

The FDA’s guidances provide a scientifically logical distinction between which cell and tissue treatments need stricter regulation and which do not. The guidances require a cell or tissue product where cells are changed or used for a new function to be clinically tested to insure that they are safe and effective. This is reasonable, because we cannot assume that they will function in this new way in this environment or that they would not do something unexpected to cause harm.

This regulatory process is equivalent to the simpler drugs and biologics. The fact that cells and tissues are more complicated does not mean that they should be less regulated.  To the contrary, their increased complexity should warrant an increased need for testing.

FDA proposes less stringent regulation for cell and tissue treatments for rare diseases or for diseases that currently lack approved treatment options. Fortunately, the FDA already has mechanisms in place for reviewing those types of urgently needed treatments. But those mechanisms must not be weakened.

We don’t know how many people are helped or harmed by many of the cell therapies currently being marketed. How many of the clinics providing treatments have studies to back up their success rates or side effects? In some cases, the harms are sensational enough to make the news. But when treatments are harmful, there is little incentive to report them to the FDA.  And in some cases, neither patients nor physicians will realize that a complication was caused by the treatment.

Even if a treatment isn’t dangerous, an ineffective treatment harms patients because it is so expensive.  And, of course, many of these treatments offer little beyond false hope.

At worst, serious clinical side effects can occur, such as tumors and vision loss.

That’s why clinical trials are absolutely necessary. Patients should be able to make an informed decision about a treatment with information based on data and good science, not just hype and hope.

Regulations will also insure that the cells that clinics claim to use are actually the cells that are put into a patient’s body. It can insure that the chemicals used to process cells are safe for this process.

Regulation and rigorous scientific testing benefits patients now and in the future. If there are too many cases of patients harmed or too many treatments fail because some clinicians used untested treatments, the whole field could be disregarded as snake oil. Not only will patients be harmed by bad treatments, but also the failure to develop real treatments.

In conclusion, we strongly support the FDA’s regulation of cell and tissue products. The guidances are reasonable. Through regulation the FDA can protect patients and encourage innovation in the development of new treatments based on sound science. However, enforcement will be critical to stop untested and potentially harmful ‘therapies’.

Thank you for your time and consideration of our views.



Division of Dockets Management

Food and Drug Administration

Department of Health and Human Services

5630 Fishers Lane, Room 1061

Rockville, MD 20852


September 9, 2016


Comments of members of the National Center for Health Research

on the draft guidance

Updating Abbreviated New Drug Application Labeling After the Marketing Application for the Reference Listed Drug Has Been Withdrawn

[Docket No. FDA-2016-D-1673]


The National Center for Health Research, and its Cancer Prevention and Treatment Fund, generally support the draft guidance “Updating Abbreviated New Drug Application Labeling After the Marketing Application for the Reference Listed Drug Has Been Withdrawn.”  It is a small step in the right direction to address generic drug labeling safety issues.

Generic drug makers are expected “to update their labeling” after the Agency has approved changes to the labeling for the brand name drug (RLD or Reference Listed Drug).86 However, when the brand name drug is no longer sold (withdrawn from the market), we strongly agree that the labeling of the generic drug might become “inaccurate and outdated, resulting in labeling that is false and/or misleading.”¹

The draft guidance describes a process for updating labeling for generic drug applications in cases where “FDA has withdrawn approval of the new drug application (NDA) for the ANDA’s reference listed drug (RLD) for reasons other than safety or effectiveness.”¹ A major problem with the draft guidance is that it is limited to situations where the RLD has been withdrawn. A better way for the FDA to ensure all generic manufacturers update their labels when necessary is for the Agency to finalize the proposed rule, “Supplemental Applications Proposing Labeling Changes For Approved Drugs and Biological Products.”

Finalizing the proposed rule would allow generic drug makers to quickly make safety updates to their labels through changes being effected (CBE).  Through the Patient, Consumer, and Public Health Coalition, we submitted comments in support of the proposed rule in 2014 because it would give patients access to the most up-to-date product labeling information regardless of whether they choose a brand-name or generic drug.87  It is not acceptable that two years later the FDA has not yet finalized the proposed rule.  The proposed rule is more robust and would better safeguard the health and safety of patients and consumers because it would require safety changes, whereas the draft guidance is not binding.  Also, due to the lack of access to the CBE process, generic drug makers currently are not responsible for injuries or deaths caused by their products, and consumers are left without judicial recourse.

We cannot emphasize enough how important it is to quickly update safety and efficacy information on generic drug labels, since nearly 80 percent of prescriptions filled in the U.S. are for generic drugs.88 Also, once generic drugs dominate the market, the brand name drug often leaves the market, and since generic drug makers cannot currently use the CBE process, the generic drug labels may not list safety problems, and that unnecessarily puts patients at-risk.


Millions of Americans rely on generic drugs to provide the same benefits as brand-name drugs, and they deserve up-to-date information about the prescription drugs that they use. It is essential that all prescription drugs (not just brand named drugs) carry accurate safety warnings and all manufacturers remain attentive to potential health hazards.

We support this draft guidance, which is a step in the right direction for protecting the public health.  However, the guidance should be broadened to include all situations, regardless of whether the RLD has been withdrawn.  Moreover, to truly ensure that generic drug labels are updated in a timely manner, the FDA needs to finalize the proposed rule, “Supplemental Applications Proposing Labeling Changes For Approved Drugs and Biological Products.”


Brett Norman, POLITICO

September 12, 2016


THE NEXT BATTLE IN THE STEM CELL WARS A two-day meeting hosted by the FDA kicks off today to hear public comment on four draft guidances put out in 2014 outlining the agency’s approach to regulating stem cell therapies. The meeting, first scheduled for one day in April on FDA’s campus, was delayed, expanded to two days and moved to an NIH auditorium to accommodate a tidal wave of requests for participation. The FDA has proposed some limited exemption from regulation for a narrow range of stem cell treatments — part of what is outlined in the guidances. But it is generally maintaining its traditional standards of safety and efficacy for more complex applications — the use of adipose stem cells to repair retinas, for instance. Many prominent scientists, entrepreneurs and stem cell trade associations, agree with the FDA’s approach. But others want a more lax standard: grant a preliminary approval once studies show a treatment is safe — meaning it could be marketed and used by patients while data is collected to establish whether it actually works.

… That’s the thrust of Sen. Mark Kirk’s REGROW Act, which he has been pushing this year, setting off alarm bells inside FDA and among consumer advocates, who worry about unleashing a wave of unproven treatments on patients seeking cures. For years, people have traveled to unregulated clinics in other countries for stem cell treatments. A report earlier this summer found that such clinics have also proliferated in the United States, with more than 351 companies in 570 locations. REGROW advocates say a lighter touch by FDA could help weed out bad actors while enabling small companies to get in the market and advance the science. But many experts say lowering the bar for stem cell treatments could badly tarnish a promising field of research if dangerous therapies are allowed to be sold. And there are many stories of the dangers. Here’s one. ICYMI, Brett’s story on REGROW. Note: Kirk policy hand Andrew Vogt said in an email that Kirk’s office would not be attending the FDA meeting. […]

… At a preliminary meeting on the science of stem cell treatments last week at FDA, Stanford’s Irving Weissman, a prominent stem cell researcher, said that the best way forward for the field would be “the triumvirate” of strong FDA requirements to prove the efficacy of treatments, revoking the medical licenses of providers who participate in unregulated, unproven trials and strong penalties against false advertising.

… Diana Zuckerman, president of the National Center for Health Research told Prescription PULSE that “it’s only the cowboys who want to do their own thing without anyone looking over their shoulder.”


To see the original article, click here

Michael D. Williamson, Bloomberg BNA

September 8, 2016

High-risk medical devices are sometimes approved using low-quality clinical data and this increases Medicare costs, a member of a Medicare congressional advisory panel said Sept. 8.

The Medicare Payment Advisory Commission’s meeting session reflected its first major examination of the medical device industry. The session may portend a deeper examination of the device industry’s practices, which could cause Congress and/or the Department of Health and Human Services to change payment rates for devices and related services, as the commission’s recommendations are fairly influential among policy makers.

Commissioner Rita Redberg, a cardiologist at the University of California San Francisco Medical Center, called for further MedPAC examination of how to push the Centers for Medicare & Medicaid Services to more broadly consider quality over an entire episode of care when deciding whether to cover a device.

Commissioners didn’t vote on recommendations. However, they debated how MedPAC staff should further research device company practices and their role in driving Medicare costs. Results of that research could prompt the commission to draft recommendations in the future.

In a separate session, commissioners discussed MedPAC staff progress on developing a uniform set of quality measures that could be used under a unified Medicare payment system for all post-acute care providers.

Tie to Rising Medicare Costs

Devices with a high risk to patients are sometimes approved by the Food and Drug Administration using poor quality data, Redberg said. This can result in low-quality devices being implanted into patients. These low-quality devices then need to be deactivated or removed, which drives up Medicare costs. […]

Redberg’s stance received praise from the leader of a patient safety group. “I completely agree with Dr. Redberg,” Diana Zuckerman, president of National Center for Health Research, told Bloomberg BNA Sept. 8. The National Center for Health Research is a nonprofit that encourages new and more effective programs and medical treatments.

In addition, the CMS should advise the FDA about the lack of data and safety and efficacy analysis specifically on patients over age 65, Zuckerman said. The lack of data “is a major problem for drugs and devices,” she told Bloomberg BNA. […]

To see the original article, click here

Damian Garde, STAT

September 19, 2016

The experimental drug that federal regulators approved Monday will only be used by a few thousand patients.

But the approval may have set a precedent that could rocket through the health care system, opening the door for drug makers to get more medicines to market — even with scant evidence that they work.

The Food and Drug Administration’s decision elated families struggling with Duchenne muscular dystrophy, a rare and deadly disease. It sent the stock of the drug maker, Sarepta Therapeutics, soaring. It also touched off a barbed debate between those who applauded the move as giving hope to desperate patients — and those who warned it would backfire since there is no clear evidence the drug works.

The approval sets “a dangerous precedent,” said Diana Zuckerman, president of the National Center for Health Research. “A decade from now, will we look back at this approval as a turning point when the FDA ceased to function as a public health agency?”

The approval was based on a trial of the drug in just 12 patients. It was shown to help some patients make dystrophin, a key protein that DMD patients are lacking. The idea is that boosting dystrophin will reverse or at least delay the disease’s muscle-wasting effects. But the trial didn’t find evidence that the drug improved patients’ ability to walk. (The trial did not include a placebo arm, so Sarepta compared results of patients on the experimental drug with historical data from other DMD patients.)

Sarepta Therapeutics ignored the FDA’s requests for a larger clinical trial and the FDA’s own staff decided last spring that there wasn’t enough evidence to approve the drug. But patients and politicians mounted an extraordinary campaign to pressure the agency. Boys with DMD and their parents crowded a public hearing in April, begging the FDA to give the drug a chance.[…]

In the decision on Monday, the FDA ordered Sarepta to conduct more research over the next two years. But it allowed the company to begin selling the drug almost immediately.

“I have never seen such an extraordinarily low level of evidence,” said Dr. Michael Carome, director of health research for the watchdog group Public Citizen. “The FDA decision is very disappointing and reflects that the leadership of the FDA caved in to political pressure.”

The furor over Sarepta’s drug, called eteplirsen, left the FDA with “a series of bad options,” said Rachel Sachs, an associate professor of law at Washington University in St. Louis. Rejecting it would have incensed the public, Sachs said.

But approving a drug that doesn’t work, Sachs said, would imperil the FDA’s most valuable asset: its reputation.

“If this changes their reputation, that could be critical,” Sachs said. “What we need is public trust in the drugs that are sold in this country, and if people can’t be sure that drugs they’re purchasing are safe and effective, that’s something we should be concerned about.”

Supporters of the FDA, however, say it’s simply following the law.

A 2012 law, known as the Food and Drug Administration Safety and Innovation Act, allowed the agency to approve new medicines for grave diseases based on surrogate evidence of efficacy — such as proof that they affect some biological processes in the body believed to be associated with the illness. The agency does not need to see proof that the drugs actually improve the patients’ ability to function or extend their lives before granting approval.

The goal is to speed approval of drugs that would otherwise take many years to rigorously test because the patient population is so small.

Sarepta’s treatment falls into that category, said Erika Lietzan, a University of Missouri law professor who specializes in drug regulation.

“I would push back on the notion that exercising flexibility in the interest of improving patient health is an overreach,” Lietzan said. “It strikes me that that is central to the agency’s mission at the end of the day.” […]

And the agency did attach some strings to its approval. Sarepta must run a larger study to test its drugs, with the results expected in 2021. If the results are negative, the agency can revoke eteplirsen’s clearance.

But some observers, including some top FDA officials, say it’s inappropriate to put the drug on the market before those results are in.

Dr. Ellis Unger, acting director of the FDA’s Office of Drug Evaluation, wrote over the summer that Sarepta’s drug is “essentially a scientifically elegant placebo.” Approving it, he said, would “send the signal that political pressure and even intimidation — not science — guides FDA decisions.”

His appeal went all the way up to FDA Commissioner Dr. Robert Califf, who acknowledged “major flaws” in Sarepta’s supporting evidence.

But last week, Califf deferred to Dr. Janet Woodcock, who is head of the FDA’s drug review division and has championed eteplirsen’s approval.

Woodcock’s decision will have “profound” ramifications, Unger wrote, “strongly encouraging public activism and intimidation as a substitute for data, which is one of the [worst] possible consequences for communities with rare diseases.”

To see the original article, click here

Rhodi Lee, Tech Times

September 20, 2016

The U.S. Food and Drug Administration (FDA) has approved the first drug for treating patients with Duchenne muscular dystrophy. Patients who have this condition are typically boys who lose their ability to walk by 12 years old and are likely to suffer from heart or respiratory failure by the time they reach their 20s.

The approval went against the recommendation of experts who did not find enough evidence that the drug was effective. The agency’s decision, however, showed the growing power of patients and advocates over evaluation of drugs. […]

The newly approved drug eteplirsen can be used by only about 13 percent of the patients. Other drugs are currently being developed for patients who have different mutations.

The drug is supposed to work by partly correcting genetic defect so the muscle cells can produce a somewhat functional form of dystrophin.

Eteplirsen maker Sarepta said that the cost of the drug would be about $300,000 per patient per year.

The decision on the drug was delayed for months and the approval was so controversial it prompted a dispute among FDA employees. While the decision delighted the advocates of the drug who actively lobbied for its approval for years, drug policy experts are worried about FDA’s decision.

Earlier this year, an FDA panel of experts said that Sarepta was not able to show significant evidence that its drug can boost the production of dystrophin to a level that can be clinically beneficial to patients. The committee also did not find substantial evidence that the drug is an effective treatment for people who have the degenerative disease.

“The agency has set a dangerous precedent,” said National Center for Health Research president Diana Zuckerman. “To prove something works, you have to compare it to something else — a placebo or a treatment. They didn’t do that.”

To see the original article, click here

Liz Szabo, Kaiser Health News

September 19, 2016

The Food and Drug Administration Monday approved the first drug to treat Duchenne muscular dystrophy, a rare and lethal muscle weakening disorder that affects about 15,000 Americans.

The FDA’s approval of the drug, Exondys 51, also known generically as eteplirsen, came over the objections of its own advisory committee, which voted not to approve the medication earlier this year. Patients and their families had lobbied hard for the drug, made by Sarepta Therapeutics of Cambridge, Massachusetts, noting that people with the disease have few treatment options. […]

The FDA’s decision speeds up the approval process for Exondys 51, allowing it onto the market based on preliminary data that suggests the drug will strengthen children’s muscles, even though the company has not yet produced clear proof that the medication will delay paralysis or improve symptoms.

In clinical trials, some patients treated with Exondys 51 had more dystrophin in their skeletal muscles, which people use to move their arms and legs. The FDA will require Sarepta to launch another clinical trial to show whether it actually improves patients’ symptoms. If the drug doesn’t help, the FDA could withdraw approval. […]

Some health advocates criticized the FDA’s decision.

Overruling the advisory committee shows “a disturbing disregard for the agency’s  legal standards for approving new drugs,” said Michael Carome, director of Public Citizen’s Health Research Group, a nonprofit that studies drug safety. “In particular, such action eviscerates the agency’s long-standing requirement that there be substantial evidence of effectiveness for new drugs — even drugs for serious rare diseases — before they are marketed.”

Diana Zuckerman, president of the National Center for Health Research, a nonprofit research group, noted that the Exondys 51 clinical trial was poorly done. Doctors leading the trial didn’t compare patients who received the drug with a “control group” of untreated patients.

“It sets a dangerous precedent if the FDA is going to start approving drugs that aren’t compared to anything,” Zuckerman said. “Why would a company choose to do a careful, well-designed study that might show that its product isn’t particularly safe or effective if it can get away with doing a tiny, poorly designed study with ambiguous results?” […]


To see the original article, click here

Jennifer Block & Liz Canner, New York Magazine’s The Cut

September 8, 2016


The “It” drug was Addyi, Sprout Pharmaceutical’s answer to low libido in women, approved by the FDA last August and now in the news for its low sales and for turning out to be a bit of a dud, effectiveness-wise. A meta-analysis in April’s JAMA Internal Medicine looked at previously unpublished clinical trials, along with those reviewed by the FDA, and found that Addyi works more like a roofie than a love potion, more than earning its black-box warning for causing dizziness, drowsiness, nausea, and, when mixed with alcohol, unconsciousness. All this for just half an extra “sexually satisfying event” per month (an industry term that includes everything from actual sex to a randy thought).

None of these questions about Addyi’s effectiveness were hidden during the FDA approval process — it had already been rejected twice. Nonetheless, bringing this drug to market seemed so important a year ago that several well-respected health and women’s groups rallied around it as part of the Even the Score campaign, a supposedly grassroots effort to expose gender bias at the FDA as the root of the disparity in treatment options — men have Viagra and several other sex boosters to choose from, they argued, while women have none (there actually are FDA-approved treatments for vaginal pain and sexual-arousal problems). When Addyi was approved, organizers called it a win for sexual parity, but some suspected they had been manipulated to score one for Big Pharma, and that troubling changes at the FDA had played a role. […]

Last August, the FDA delivered its approval, overturning its own internal reviewers’ recommendations. In an editorial accompanying the recent JAMA review, Steven Woloshin and Lisa Schwartz, physician-researchers at Dartmouth Medical School and the founders of pharmaceutical education company Informulary, wrote that FDA administrators “overrode” scientists’ evaluations that the drug didn’t warrant approval. “When we looked at the new data for Addyi, it was hard to understand why the drug was approved the third time around because there was a new alarming drug interaction study — so alarming that it was stopped early because so many people had low blood pressure or fainted,” Schwartz said.[…]

Even the Score’s leaders said the group “evolved from discussion among the women’s groups,” as the New York Times reported. But several organizations we called said the idea had been introduced to them by two close friends: Susan Scanlan, “a DC institution when it comes to women’s groups,” as one activist put it, and Audrey Sheppard, the former head of the Office of Women’s Health at the FDA. Sheppard especially had feminist-health street cred. But in this case she was working for Sprout.

After her tenure at the FDA, Sheppard began working closely with pharmaceutical companies, and by 2008 had become a full-time consultant, organizing show-and-tells for device and drug manufacturers to introduce them to the women’s health Establishment in D.C. — products like the female condom, a breast-tissue expander for reconstructive surgery, and Plan B. Cindy Pearson, who heads the National Women’s Health Network, told us that “Sheppard often encouraged her clients to make individual contributions to us or our event.”

In 2013, Sheppard was organizing luncheons, conference calls, and private meetings with feminist and medical leaders to introduce them to Sprout’s libido drug, then known as flibanserin. At first, those meetings were about the drug’s ostensibly promising new data. “Audrey’s job was to get people like me interested in a product and to support it. With flibanserin, it wasn’t just her job. I believe she thought this drug deserved to be approved,” said Diana Zuckerman, director of the National Center for Health Research. “But as a scientist, I wasn’t impressed with the data.” Other feminist health movement leaders echoed the sentiment.

By early 2014, Zuckerman and others say Sheppard was back knocking on doors with Sprout CEO Cindy Whitehead, this time with a different sales pitch. Now there was talk of the need to fight gender bias at the FDA. […]

The Even the Score campaign included well-placed parodies of Viagra ads, a website, a petition drive, and a joint letter from 11 members of Congress to the FDA urging the agency to approve the drug, with few mentions that the effort was funded by Sprout. But anybody who scrolled down the About Us page of its web site, past the logos of the nonprofit members, would have seen Sprout Pharmaceuticals and Trimel Pharmaceuticals (which also has a female-libido drug in the pipeline) listed as sponsors.

In June, a paper by a bioethicist and a social psychologist in the Journal of Medical Ethics(part of the British Medical Journal) argued that Even the Score was “cleverly disguised as a campaign to empower women” based on “deceitful and inaccurate information” that employed an “unethical use of moral arguments.”

Watchdog groups like Wood’s and Zuckerman’s, along with the New View Campaign, the National Women’s Health Network, Our Bodies Ourselves, and Pharmed Out wrote letters to the FDA and op-eds arguing that drug approvals should be based on science, not politics. Still, the coordinated effort to warn mainstream women’s organizations about Addyi’s safety risks and lack of efficacy was slow to get off the ground. One feminist health advocate recalled a meeting at a coffee shop one early morning the summer after Even the Score had been launched, at which several women’s health leaders and Sheppard agreed their disagreement would be civil.

“A lot of us who know and respect Audrey through the years, even though we disagreed with her on flibanserin, we were reluctant to actively work against her,” Zuckerman said. “By the time the campaign was created, we were too late,” Wood added. “The groups had already signed on.” […]

These days, the FDA seems to be approving almost everything. From January to August 2015 (when Addyi was approved), the agency cleared 96 percent of new drugs, and boasted about how many and how efficient the process has become. In 2010, when Addyi first went before the FDA (as flibanserin) that rate was 57 percent. According to a study by Harvard Medical School, since pharmaceutical companies started funding the FDA’s Center for Drug Evaluation and Research, drugs have a one in three chance of being slapped with a black-box warning or taken off the market for safety reasons. Sidney Wolfe, of government watchdog Public Citizen, advocates for a “seven-year rule” for drugs — consumers should wait that long before trusting an approved drug is safe, he says.

With only 4,000 prescriptions written since February, a monthly cost of $800, and many insurers refusing coverage, Addyi’s lasting legacy isn’t likely to be that of a game-changer for women. On the other hand, it may well be a test case for sped-up drug approval processes. In April, Zuckerman attended a hearing for Sarepta, a drug to treat a rare form of muscular dystrophy in children. It was tested in only 12 subjects, with no control group, and scientific reviewers gave a “scathing” review. (As part of the bargain for funding the FDA, certain drugs can now come up for approval without placebo-controlled clinical trials.) A thousand distraught parents attended the meeting and pushed for approval. “It’s quite clear to me that the company learned from the Even the Score campaign how to manipulate the FDA through patient advocacy,” says Zuckerman. “The companies have learned that even if they have lousy data, or lousy product, the way to get it approved is to get patients to advocate for it. And the patients don’t need to be particularly persuasive from a scientific point of view.” […]

To see the original article, click here

Diana Zuckerman, PhD,

National Center for Health Research

In 2015, plastic surgeons who have been well known for defending the safety of breast implants published a study of 173 women with cancer of the immune system caused by breast implants. [1]  The study was paid for by a plastic surgery medical association and written by plastic surgeons who have defended the safety of breast implants for decades.

ALCL (Anaplastic Large Cell Lymphoma) develops near a breast implant but is not breast cancer – it is a cancer of the immune system.  The authors of this study point out that the first silicone breast implant was implanted in 1962 and the first publicly reported case of ALCL in a woman with silicone breast implants was in 1997. The authors reviewed 37 medical articles reporting on 79 patients and collected information about an additional 94 women with ALCL caused by breast implants.


Physicians first identified these 173 women with ALCL based on either seromas (a collection of fluid under the skin), a mass attached to the scar capsule surrounding the implant, a tumor that eroded through the skin, in a lymph node near the breast, or discovered during surgery to replace a breast implant. Whether the women had silicone gel or saline breast implants didn’t seem to make a difference, but many of the women had at least one textured breast implant.  Cosmetic augmentation patients and women who had breast implants to reconstruct their breasts after undergoing a mastectomy were both at risk of developing ALCL because of their implants.  Of the women whose ALCL spread outside of their scar capsule surrounding the implant, about half died from ALCL.

The authors pointed out that ALCL can be difficult to diagnose.  Although the fluid and scar capsule usually appear abnormal, they sometimes look normal. The authors recommend “that all fluid and capsule tissue from patients with seromas” should be tested for ALCL.  They point out that if the tumor is inside the capsule, removing both implants and the capsules may be the only treatment necessary.  However, if the tumor has developed just outside the capsule, chemotherapy with or without radiation is needed and usually effective.  Unfortunately, aggressive ALCL that has spread beyond the scar capsule area is usually fatal, regardless of treatment.

To read the official summary of this article, click here:

To read about another study on ALCL, click here.

To read more about what you need to know about ALCL, click here.

[1] Brody GS, Deapen D, Taylor CR, Pinter-Brown L, House-Lightner SR, Andersen JS, Carlson G, Lechner MG, Epstein AL. Anaplastic Large Cell Lymphoma Occurring in Women with Breast Implants: Analysis of 173 Cases. Plastic and Reconstructive Surgery. Vol 135: 695, 2015.

Diana Zuckerman, PhD

Cancer Prevention and Treatment Fund

A growing body of evidence suggests that using talc in the genital area can increase a woman’s chances of developing ovarian cancer.  And the more years she uses talc, the more likely she is to develop ovarian cancer.

A study published in 2016 suggests that the body develops inflammation as a result of the talcum powder, and this is what can result in cancer years later. The authors of the study are from the prestigious Brigham and Women’s Hospital in Boston, and their study was supported by a grant from the National Institutes of Health.

The study compared approximately 2,041 women living in Massachusetts and New Hampshire who had been diagnosed with ovarian cancer, and compared them to 1,578 women of the same age and geographic location.

The study found that the women who used talc in the genital area, whether or not they used it elsewhere in their body, were significantly more likely to have been diagnosed with epithelial ovarian cancer.  Most reported using Johnson & Johnson baby powder or Shower to Shower powder.  Many body powders are now made with cornstarch instead of talc; women who used those powders but did not use talc were not considered talc users.

Overall, the women using talc were about 33% more likely to develop ovarian cancer.  However, among women who were sterilized prior to menopause (underwent a tubal ligation or hysterectomy) or who took hormone therapy for menopausal symptoms, using talc was even more likely to predict developing ovarian cancer.

How big a risk is talc for developing ovarian cancer?  Perhaps a better question is: why take the risk?


Cramer, DW, Vitonis, AF, Terry, KL, Welch, WR,Titus, LJ. “The Association Between Talc Use and Ovarian Cancer: A Retrospective Case–Control Study in Two US States.” Epidemiology May 2016. 27(3): 334-346.

Kristine Chin

National Center for Health Research

Sexually transmitted diseases (STDs) are infections transmitted from one person to another through sexual activity. Sexually transmitted infections (STIs) are different from STDs in that a person with an STI can have the infection, but will not show symptoms. In other words, the infection has not developed into a disease.

STDs can cause a lot of symptoms from rashes and genital warts to infertility and liver failure. Here is a list of the most common STDs, their symptoms and treatments, and how to prevent getting the infection in the first place.


Genital herpes is an STD caused by two viruses, called Type 1 and Type 2.  Both kinds of herpes can infect the genital area and the mouth. However, infection of the mouth is usually caused by Type 1 and infection of the genitals are usually caused by Type 2 The herpes viruses can be inactive for weeks or months or even years, but can keep coming back as an active infection throughout life. You can be infected by both types of herpes at the same time.

Some infected people don’t get any symptoms from the virus at all, but can still infect others.89 When symptoms develop, they can be painful blisters or ulcers at the site of infection (mouth or genitals or anus). Herpes is transmitted through direct contact, such as kissing, sex, and contact with the genital area or fluids of someone who is infected.

There is currently no cure for herpes. However, antiviral drugs can help to shorten outbreaks, decrease symptoms, and make the virus less contagious to others.90

Male anatomy

Female Anatomy


Chlamydia is a common bacterial STD that causes the inflammation of the urethra, rectum, and anus in men and women and also inflammation of the cervix in women (Figure 2). It typically does not cause symptoms, but infected persons can experience a burning feeling when they urinate or have an abnormal discharge from the vagina or penis. The infection can cause chronic pelvic pain.  It can also cause pelvic inflammatory disease (PID) or block the fallopian tubes, either of which can cause infertility.  It can also increase the chances of an ectopic pregnancy, which always results in the death of the fetus and can also kill the mother. 91

Chlamydia is contagious during oral, vaginal, or anal sex and can be passed from the mother to baby during childbirth. Antibiotics can cure chlamydia but will not reverse damage that the bacteria already caused to your body, such as scarring.92


Gonorrhea, aka “The Clap,” is caused by bacteria that infects mucous membranes, affecting the urethra, rectum or throat in both males and females. It can also affect the uterus, fallopian tubes, and cervix in females (Figure 2).

The infection is spread during oral, vaginal, or anal sex and from mother to baby during childbirth. Gonorrhea doesn’t usually cause symptoms, but some patients experience a painful, burning sensation during urination and/or have an unusual discharge. It can also cause vaginal bleeding between periods, abdominal pain, and pelvic pain in females. Gonorrhea, like chlamydia, can cause pelvic inflammatory disease (PID). 93

Antibiotics can be taken to treat the infection, but will not reverse any damage that the bacteria caused before the treatment.94


Syphilis is not as common as the other STDs above. It is caused by bacteria that infect the lips, mouth, genital area, or anus.95

There are four stages of syphilis but the infection does not have to happen in the same order.

Primary syphilis causes a small sore (a chancre) in the spot that was infected. The sores will appear 3 weeks after infection and will heal by themselves about 6 weeks after that. Secondary syphilis usually begins several weeks after the sore heals. During this stage, a full body rash will start on the trunk of the body and will spread. The rash could either disappear completely or come and go for as long as a year. After those obvious symptoms, most people will have been diagnosed and treated for syphilis. However, if an infected person hasn’t gotten treatment, the infection can progress into latent or tertiary syphilis.

Latent syphilis is when there are no longer symptoms. Either the disease will never come back or it will move to tertiary stage. Tertiary syphilis is when the infection comes back and can result in damage to the brain, nerves, eyes, heart, liver, and joints.96 This can lead to neurosyphilis, which can result in impaired balance, chronic pain, lack of muscle coordination, and nausea.97

If treated at its early stages, syphilis can easily be cured by an injection of penicillin. Latent syphilis can possibly be treated with multiple injections of penicillin every week for an undetermined duration. The treatment time is different for everyone because the bacteria can become dormant at different times for different people. People who have been cured of syphilis are still capable of getting a new syphilis infection if they are exposed.


Hepatitis B is a virus that infects the liver. The virus is transmitted through body fluids such as blood and semen. The infection is typically spread through sex, sharing needles, or from the mother to child during childbirth.  It is considered chronic if it lasts more than 6 months.

Symptoms vary depending on your age.  About 30-50% of people older than the age of 5 have symptoms that include fever, fatigue, vomiting, dark urine, and jaundice. Hepatitis B does not always go from acute to chronic. However, in 90% of infants, 25-50% of children age 1-5, and 5% of adults, the acute infection does become chronic infection. This happens if the infection is not treated or if the treatment is not effective and the virus remains in the body for longer than 6 months. 98  

Children are typically vaccinated for hepatitis B at age 11-15. If you have not been vaccinated and are aware that you have come into contact with the virus, you see a doctor as soon as possible. You need a shot within 12 hours of infection to avoid developing the disease. There is no treatment for acute or chronic hepatitis B. It is recommended that a person with an acute infection gets adequate fluids and nutrition to make sure the body has resources to kill the virus. If acute infection does turn into chronic infection, there are treatments to reduce the amount of virus in the blood and the risk of developing liver disease. Such treatments include antiviral medications, interferon alfa-2b injections, and liver transplant in serious cases.99 If you are diagnosed with chronic hepatitis B, you should see a doctor to develop a treatment plan. 


Most of these STDs can be diagnosed by a doctor by a urine test or a swab test of the affected areas. In some cases of Hepatitis B, doctors may want a liver sample to determine how bad the liver damage is.


More than half of the population will get an STD or STI within their lifetime, so it is very easy to catch one from sexual activity.  The most effective way to avoid getting STDs or STIs is abstinence.100 The vaccines for hepatitis B and HPV are not as effective as abstinence but offer very good protection (although it is not known how long the HPV vaccine is effective). When used correctly and consistently, male and female condoms and dental dams provide physical barriers that can protect you from getting an STD or STI. However, some STDs, such as herpes, can spread if blisters are not covered by the condom.

Having only one sex partner or a small number of sex partners also will decrease your chances of getting STDs.101 Even so, you should get tested regularly. If you are diagnosed with an STD or STI, you should tell your partner or partners to prevent spreading the disease to others.  

All articles on our website have been approved by Dr. Diana Zuckerman and other senior staff.




Laura Gottschalk, PhD, National Center for Health Research



Soapy handMany people choose soaps labeled “antibacterial” because they think these products prevent more infections than just plain soap. However, the Food and Drug Administration (FDA) announced on September 2, 2016 that there is no evidence that is true.  And, these bug-killing substances can be harmful.

The FDA has banned soaps that contain 19 of the most common ingredients used in these products. The ban was announced after the FDA gave the companies 3 years to prove that these ingredients worked any better than soap and water in preventing the spread of infection, but they were not able to do so. In addition, there aren’t any studies showing that these ingredients are safe for long-term use. Since some of the ingredients affect hormones, they could be harmful.

So far, this rule only applies to soaps and body washes that you rinse off with water. The FDA is still collecting information to figure out how safe and effective hand sanitizers are at reducing bacteria on the skin.

Despite this new ban on antibacterial soaps, companies have a full year to start following it. That means that you could still buy these ineffective—and possibly dangerous—products through August 2017.

So how do you avoid these newly banned soaps?  The good news is that some companies had already switched their soap formulas because they knew the ban was coming.  As a good rule of thumb, skip any soaps or body washes that are labeled as “antibacterial” since they probably contain one of the banned ingredients. You can also check the label to see what the active ingredients are. Two of the most common to watch out for are triclosan and triclocarban.  But remember, even if a soap ad or packaging says “triclosan-free” or “tribocarban-free,” it might still have one of the other 17 banned ingredients.  The full list of all 19 are found at the bottom of this article.

Despite the fact that some of the most popular soaps in the U.S. are now banned, it is still true that washing your hands with plain soap and water is still one of the most effective ways to prevent the spread of disease and infection. So make sure to keep washing those hands, but just skip the antibacterials while you do it!


Full list of the 19 banned chemicals:

  • Cloflucarban
  • Fluorosalan
  • Hexachlorophene
  • Hexylresorcinol
  • Iodophors (Iodine-containing ingredients)
    • Iodine complex (ammonium ether sulfate and polyoxyethylene sorbitan monolaurate)
    • Iodine complex (phosphate ester of alkylaryloxy polyethylene glycol)
    • Nonylphenoxypoly (ethyleneoxy) ethanoliodine
    • Poloxamer–iodine complex
    • Povidone-iodine 5 to 10 percent
    • Undecoylium chloride iodine complex
  • Methylbenzethonium chloride
  • Phenol (greater than 1.5 percent)
  • Phenol (less than 1.5 percent) 16
  • Secondary amyltricresols
  • Sodium oxychlorosene
  • Tribromsalan
  • Triclocarban
  • Triclosan
  • Triple dye


Chris Glorioso and Evan Stulberger, NBC4 New York

August 31, 2016

The CEO of Mylan Pharmaceuticals announced the launch of its own cheaper version of the EpiPen this week on the heels of a price hike uproar. But for years, Mylan and its business partners have fought fiercely behind the scenes to block a cheaper generic from hitting the market, the I-Team has learned.

Documents reviewed by the I-Team show the Pennsylvania-based drug firm paid for opposition research on a generic EpiPen proposed by Teva, an Israeli pharmaceutical company.

The study concluded the Teva product would have a 93 percent failure rate.

But experts on research methodology say the study has significant flaws, including the lack of any control group, the lack of any statistical significance, and the fact that study participants were instructed “not to actually manipulate the device or perform the injection.”

“The study was just flawed from start to finish,” said Diana Zuckerman, who heads the National Center for Health Research, a nonprofit that monitors Food and Drug Administration regulatory policy. […]

That study, part of a submission to the FDA called a Citizen Petition, was just one flank in a strategy aimed at blocking the cheaper Teva generic. […]

Citizen Petition, including opposition research, routinely delay FDA decision-making because the agency is required to review the material, no matter how scientifically sound. In some cases, health regulators take weeks, months or even years to respond to Citizen Petitions.

Michael Carrier, a Rutgers law professor who reviewed 15 years’ worth of Citizen Petitions, found only a small minority of the filings actually come from individual citizens. Instead, he found more than two thirds come from brand name companies and most of those petitions seek to block cheaper generics from hitting the market.

“It’s really about the brand company delaying the generic from entering the market,” Carrier said. “Because every day that they can delay generic entry could be millions of dollars lining its pockets.” […]

Meanwhile, Mylan has announced its own generic EpiPen will be out in a few weeks and priced around $300. But the company will continue to enjoy a monopoly, also selling the brand-name version for about $600.

To see the original article and broadcast, click here

Shannon Firth, MedPage Today

August 20, 2016


PDUFA Again: Still Trying to Get It Right
Industry and patient representatives embraced the sixth version of the Prescription Drug User Fee Act, aka PDUFA, while consumer groups balked at the unabashed enthusiasm shown by their counterparts at a public meeting held by the FDA on Monday.
As Marc Boutin, JD, CEO of the National Health Council, a patient advocacy group, expressed gratitude for the inclusion of the patient voice in the FDA’s commitment letter, so that “not a single company is going to develop a product without engaging the end user (the patient) throughout that development,” Paul Brown, government relations manager for the National Center for Health Research, leveled a long-standing objection: “User fees should focus more on the safety and efficacy of drugs, not just the speed of approval,” he said.

He also cautioned the agency against treating industry as “a customer it needs to please.” Brown and Boutin spoke at a public meeting convened to solicit feedback from stakeholders regarding the payments made by drug and device manufacturers for human drug reviews. The latest reauthorization, if it passes, will determine the agency’s user fee policies from 2018 to 2022.

Brown described the tenor of the meeting as “almost like puppy dogs and rainbows.”


To see the original article, click here

Sarah Karlin-Smith and Brett Norman, Politico Prescription Pulse

With help from Mary Lee

August 22, 2016


Quick Hits

Whistleblower lawsuit — Medical device giant Medtronic is facing allegations that it promoted a device for uses not approved by FDA, leading to patient injuries and false claims submitted to government payers, STAT reports. The whistleblower suit alleges the company sought approval for the device under false pretenses. Patient advocates say the case exposes the shortfalls of medical device regulations. If the device had been tested in a clinical trial it would have been clear that it was designed to fit a part of the spine the company wasn’t seeking approval for, Diana Zuckerman of the National Center for Health Research explained to STAT. Medtronic has not yet responded to the suit.


To see the original article, click here


August 18, 2016


Robert M. Califf, M.D., Commissioner

Food and Drug Administration

10903 New Hampshire Ave

Silver Spring, MD 20993-0002


Dear Commissioner Califf:

We are writing to express our deep concern regarding the lack of public health and consumer perspectives at the August 15, 2016 Public Meeting on Prescription Drug User Fee Act (PDUFA) Reauthorization and the lack of those perspectives in the commitment letter itself.

There were three stakeholder panels of invited speakers: to discuss post-market safety, regulatory decision tools, and administrative enhancements. Not one stakeholder on any of the panels was a consumer or public health advocate. Instead, the panels included only stakeholders from industry-affiliated patient groups as well as BIO and PhRMA representatives. Four of the five panel members on the first and third panels were on both panels. None of the panel members pointed out that the commitment letter, which is 46 pages long, included only 2 pages that mention patient safety. Not one of the panel members pointed out that the commitment letter, which is 46 pages long, included only 2 pages that mention patient safety.

We understand that the FDA is working hard to include more patient perspectives, but the vast majority of taxpayers who depend on the FDA for their health and who support the FDA with their tax dollars consider themselves consumers rather than patients, and are not affiliated with patient advocacy organizations that are primarily funded by pharmaceutical companies. Their voices are not being adequately heard or respected at the FDA. There are numerous public health advocates and researchers as well as consumer advocacy organizations that could represent the views of consumers at FDA meetings, but were not invited to do so. In addition, there are nonprofit patient organizations that are independent of pharmaceutical companies, many of which focus on safety and are members of the Patient, Consumer, and Public Health Coalition, and they were not included either.

At the August 15, 2016 Public Meeting on Prescription Drug User Fee Act (PDUFA) Reauthorization, our organization, the National Center for Health Research, commented on the commitment letter during the public comment portion of the meeting (see attached copy of our comments). Previously, we also attended all the PDUFA VI stakeholder meetings, and we participated in PDUFA IV and PDUFA V reauthorization cycles as well. If FDA was unable to find consumer or public health stakeholders to participate on the panels, the agency could have contacted our Center and we would have gladly produced a list of at least a dozen well-qualified potential participants.

FDA asked public comment speakers to state whether or not they had any financial conflicts of interest, but did not ask panel members to disclose any conflicts of interest, and none of them volunteered that information. That makes no sense. We strongly support disclosing conflicts of interest at FDA meetings for all participants who speak. When an organization advocates for faster FDA approval of medical products and less conclusive evidence of safety and efficacy by relying on surrogate endpoints and shorter and smaller clinical trials, the public has a right to know if that organization is tied financially to the prescription drug industry.

The August 15 PDUFA meeting is just the latest example of how public health researchers and experts, independent nonprofit patient organizations, and national consumer organizations representing millions of Americans are being excluded from FDA public meetings and FDA decision-making while patients with ties to organizations funded by industry are being used to support industry perspectives. FDA is repeatedly ignoring the views of academic researchers, public health advocates, and consumer groups. The unmistakable message is that the FDA does not value our views.


Diana Zuckerman, PhD, President


cc: Dr. Janet Woodcock, Senator Lamar Alexander, Senator Patty Murray, Congressman Fred Upton, Congressman Frank Pallone


Division of Dockets Management (HFA-305)

Food and Drug Administration

5630 Fishers Lane, Rm. 1061

Rockville, MD 20852


Comments of the National Center for Health Research on

PDUFA VI Commitment Letter

[Docket No. FDA-2016-N-1895]



The National Center for Health Research respects the Food and Drug Administration (FDA) and we’re committed to ensuring that it has the resources it needs to keep our medical products safe.  Given the inadequate appropriations provided to the FDA, we strongly support increasing user fees to improve FDA’s resources in order to enable the agency to fulfill its public health mission.


We are disappointed that the focus of the FDA’s commitment letter is on speed.  The commitment letter states that “PDUFA’s intent is to provide additional revenues” so the FDA can make medicines available to patients “sooner without compromising review quality or FDA’s high standards for safety, efficacy, and quality.”[1] We believe that user fees should be used to improve the safety and efficacy of approved drugs, not just the speed of approval. PDUFA focuses too much on speeding new drugs to the market and not enough on safety issues. In the 46-page PDUFA VI commitment letter, only two pages are devoted to the FDA drug safety system.


In PDUFA V, we supported earlier and increased meetings between FDA and drug companies during the drug approval process.  PDUFA VI calls for even more meetings. The result is that the percentage of drugs that the FDA is approving is higher than ever.  But that isn’t necessarily a good result, because numerous recent studies have shown that too many of these drugs are not effective.  It seems likely that these meetings result in pressure on FDA staff to approve products based on inadequate scientific evidence, just because the agency has invested so much staff time in the review process.


FDA’s primary mission is “protecting the public health by assuring the safety, efficacy and security” of medical products.[2]  User fees should fund an independent review of how the program has affected overall public health.  Have user fees changed FDA’s priorities?  Is FDA now treating industry as a customer that it needs to please, instead of acting as a regulator to ensure the public health?   Independent researchers suggest that user fees are harming, not helping, the FDA’s public health mission.[3], [4]  That must change.


Regarding the Sentinel program, we agree it deserves additional funding.  However, we urge FDA to make Sentinel databases available to independent researchers so other stakeholders can perform their own assessments of drug safety. One option FDA should consider is to ensure that the Reagan-Udall Foundation’s Innovation in Medical Evidence Development and Surveillance (IMEDS) program has the resources to provide access to these databases to independent researchers at low or no cost. This is essential because FDA is approving more and more drugs through expedited approval pathways, based on surrogate endpoints and other preliminary evidence.  In addition, the smaller, shorter-term studies typical of expedited pathways provide completely inadequate evidence of safety, since uncommon and long-term risks are unlikely to be evident.


Although the FDA does not directly influence the price of drugs, they are indirectly contributing to skyrocketing costs of drugs by approving products that have little if any benefit.  For example, a recent analysis indicated that the average yearly cost per patient for the 100 top selling orphan drugs was nearly $112,000 compared to $23,000 for non-orphan drugs.[5]  One chilling example is when Keveyis was classified as orphan drugs and the price went from $50 a bottle to $13,650 a bottle.5


Another example is Cabometyx, which the FDA approved for thyroid cancer based on preliminary data.  Subsequent research indicates it is no better than placebo for thyroid cancer.  And yet our Center has learned that it costs $169,000 per year per patient and FDA has not rescinded approval for that indication.


PDUFA VI emphasizes a flexible approach to approving orphan drugs, based on biomarkers and other research designs and endpoints that may show promising results that are ultimately found to be nothing but false hope.  These faster, less thorough reviews will cost patients and taxpayers billions of dollars but many will later be found to offer risks that far outweigh the benefits.  The FDA needs to find a way to prevent that from happening and when it does happen, FDA needs to have access to user fee funds to rescind approval very quickly.


PDUFA VI should provide funding to monitor off-label uses of drugs.  Although physicians may use their own judgment to prescribe drugs off-label, drugs used in this manner have less information about the benefits and harms for the condition for which they are prescribed. A  JAMA Internal Medicine article stated that, researchers found a 54% increase in adverse events when drugs were prescribed for off-label uses. The risk was highest for patients taking more than one drug, which includes most Medicare patients.[6] With PDUFA funding, FDA could identify the top drugs prescribed off-label and target them for increased postmarket surveillance to better protect the public health. User fees should also be used to fund the development and implementation of informed consent procedures for drugs used off-label. Doctors should be required to tell patients and their families about contraindications and other warnings as part of the informed consent process when drugs are used off-label.


Given growing evidence that off label uses have many risks, PDUFA funds should be used to help monitor direct-to-consumer ads. In 2015, PhRMA spent $5.4 billion on direct-to-consumer (DTC) ads, and in that same year Americans spent a record $457 billion on prescription drugs.  There is a need to carefully review all ads to reduce the misuse of prescription drugs. In the past, drug makers have advertised atypical antipsychotic drugs as if they were antidepressant drugs.  Despite misleading ads that confuse patients, some of which are still on TV today, the FDA did nothing about it. PDUFA funds should be used to enhance FDA’s Division of Drug Marketing, Advertising, and Communications (DDMAC).


Regarding the August 15, 2016 Public Meeting on Prescription Drug User Fee Act (PDUFA) Reauthorization, we are deeply concerned about the lack of public health and consumer perspectives at the meeting and  the lack of those perspectives in the commitment letter itself. Three panels included invited speakers to discuss post-market safety, regulatory decision tools, and administrative enhancements. Not one stakeholder on the panel was a consumer or public health advocate. Instead, the panels included only stakeholders from industry-affiliated patient groups as well as BIO and PhRMA representatives.  Perhaps that is why not one of them pointed out that the commitment letter overwhelmingly focuses on speed of drug approval and hardly mentions patient safety.



The FDA has been under-funded for years and, in today’s budgetary climate, user fees are necessary.  FDA is struggling to manage expanded demand with inadequate appropriations. While we are happy with the increased resources for Sentinel, and for hiring and retaining employees, we are not convinced that the increased PDUFA fees will be adequate to cover the increased workload, such as resource intensive-performance goals focused on speed, not safety or efficacy. The proposed performance goals call for numerous time-consuming meetings between FDA and industry, several new draft guidance documents, and frequent public workshops or public meetings.


Patients and consumers and the groups that represent them have a right to influence how user fees are spent.  We do not pay the fees, but we do pay for the medications.  We pay directly when we use our own money to buy the products, pay for insurance and federal health programs, or pay for the health problems that may result when the medicines don’t work well.  And, as taxpayers we pay for the appropriations that are still supporting a major proportion of FDA resources. It is inappropriate that patients and consumers who are independent of industry are not included in the PDUFA decision-making process.  As a result, our priorities and needs are not adequately met in the PDUFA VI commitment letter.


[1] Federal Register (July 19, 2016).  Food and Drug Administration. Prescription Drug User Fee Act; Public Meeting: Request for Comments. [Docket No. FDA-2016-N-1895].

[2] Food and Drug Administration (December 7, 2015). What We Do.

[3] Light DW, (July 17, 2013). Risky Drugs: Why The FDA Cannot Be Trusted, Harvard University, Edmond J. Safra Center for Ethics

[4] Light DW, Lexchin J, Darrow JJ (June 1, 2013). Institutional Corruption of Pharmaceuticals and the Myth of Safe and Effective Drugs, Journal of Law, Medicine and Ethics, 2013, Vol. 14, No. 3: 590-610

[5] Johnson CY (August 4, 2016). High prices make once-neglected ‘orphan’ drugs a booming business. The Washington Post. _xsGN3x_dcjctNPxU71jgeOMWP5IBStU9q_NCcddIDRTgCtcTzar0xei8ReXtRlBu0kNDgFlI5BRsf51BB_8uZ2iMUgg&_hsmi=32543650&utm_campaign=KHN%3A%20First%20Edition&utm_content=32543650&utm_medium=email&utm_source=hs_email

[6] Good CB, Gellad, WF (January 2016). Off-label Drug Use and Adverse Drug Events (Invited Commentary), JAMA Internal Medicine.


David Coleman, Irish Independent

August 17, 2016

Parenting expert and clinical psychologist David Coleman on why a toddler should not use a tablet […].

Even if you limit your daughter’s tablet use to watching and listening to nursery rhymes, it is still bad for her. You need to get rid of the tablet and any other screens while she is around.

As far back as 1999, the American Academy of Paediatrics recommended that parents don’t let their children have any screen time, under age two, and that after this age, screen time should be limited.

That was 17 years ago. You may argue that technology and the potential benefits of technology have moved on considerably since then. But, the most recent article I came across, from 2015, from the National Center for Health Research, again highlighted all the dangers of screens for small children. The article can be found at

I do urge you to read it. It is a concise gathering of recent literature on the area of young children and screen time. I’ll give you some headline stats from it.

For example, the more TV a child under three watches, the more likely he is to have trouble with reading and paying attention later on. The more television a baby eight to 16 months old watches, the fewer words she knows.

Even having screens on in the background makes a difference. Children play less intently and for shorter periods of time if a TV is on in the room with them. Parents are distracted and less attuned to their children’s needs, affecting the quality of their interactions when there is a TV on in the room.

Research shows that the more television infants and toddlers are exposed to, the more likely they are to be inactive and obese, have difficulty sleeping, and show aggression. […]

To see the original article, click here.


Sheila Kaplan, STAT

August 15, 2016

Jerome Lew is a Hollywood screenwriter, and what happened to him could have come straight out of a horror film.

In 2009, Lew went to UCLA Medical Center for surgery to relieve numbness and pain in his hands. The operation appeared to be a success. But he later began having trouble speaking. His left eye drooped. He developed severe nerve pain and weakness in his neck, arms, and hands.

Lew concluded that the problem had been caused by an implant fused into his neck. It had never been approved to replace a bone in the neck.

“Jeremy’s injuries from this surgery have been devastating,” said his attorney, Robert Vaage. “His life was ripped away by one surgery and the devices that were used.”

In July, Lew, 52, settled with the University of California for $4.2 million; the manufacturer of the device, Medtronic, also settled for a confidential amount. Both parties denied any wrongdoing.

Yet the legal challenge is not over for Medtronic. The company, one of the world’s largest medical device manufacturers, now faces a whistleblower lawsuit that claims it sought Food and Drug Administration approval for its devices under false pretenses — and that the devices have been regularly used for a purpose that was never intended by regulators. […]

The FDA declined to say whether it believes Medtronic has violated regulations guiding the off-label use of medical devices or other regulations. But a spokeswoman said the device was “cleared explicitly for use in the thoracic and lumbar spine.”

Patient advocates say the case highlights the shortfalls of the regulatory system. Medical devices, unlike prescription drugs, are not subject to clinical trials, as is the case with prescription drugs.

Diana Zuckerman, president of the National Center for Health Research, said more scrutiny in a trial would have made it clear that the Medtronic device was sized to fit in the neck.

“Had it been tested in a clinical trial, the orthopedic surgeon would have said, ‘What the hell, this doesn’t fit in the lumbar spine,’” said Zuckerman.

The FDA “didn’t have any data about safety or effectiveness or even whether it would fit where it was supposed to,” she said. “The truth is, FDA is doing such an inadequate job of reviewing devices that if it weren’t for the lawsuits, even more patients would be harmed.” […]

To see original article, click here