Healthy Living & Prevention Medical Care For Adults Child & Teen Health Violence & Risky Behavior Work & Family Resources

Tracy Rupp, PharmD, MPH, RD and Mingxin Chen, MHS

January 2017

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.1  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.2

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).3

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.4 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.5 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.6 The Institute of Medicine recommends the following daily amount of vitamin D for average healthy adults:7

  • 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.8

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

January 17, 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.

Sincerely,

350.org
350PDX
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
Battlement Concerned Citizens
BECAUSE
Bold Alliance
Breast Cancer Action
California Kids IAQ
Cancer Prevention and Treatment Fund
Center for Biological Diversity
Center for Environmental Health
Center for Food Safety
Center for International Environmental Law
Central Valley Air Quality Coalition (CVAQ)
Chesapeake Climate Action Network
Chesapeake Physicians for Social Responsibility
Citizens Action Coalition of IN
Clean and Healthy New York
Clean Water Action
Clean Wisconsin
Climate Action Alliance of the Valley
Climate Law & Policy Project
ClimateTruth.org Action
Coalition For A Safe Environment
Coalition for Clean Air
Community Dreams
Concerned Residents of Portland, NY + People Like Us (CropPlus)
Conservation Colorado
Conservation Voters New Mexico & CVNM Education Fund
Defenders of Wildlife
Downwinders at Risk
Earth Action, Inc.
Earthjustice
Earthworks
Eco-Justice Collaborative
Ecology Center
Ecology Center (Michigan)
Elders Climate Action
Emerald Cities Collaborative
EMERGE
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 Working Group
Farmworker Justice
French Broad Riverkeeper
Friends of the Earth
Gasp
Grand Canyon Trust
Green America
GreenLatinos
Heal the Bay
Hispanic federation
Institute for Agriculture and Trade Policy
Interfaith Power & Light
Iowa Citizens for Community Improvement
Iowa Interfaith Power & Light
Labadie Environmental Organization (LEO)
Latino Decisions
League of Conservation Voters
Let’s Talk Climate
Louisiana Bucket Brigade
Maine Audubon
Maine Wilderness Guides Organization
Mass Audubon
Missouri Coalition for the Environment
Moms Clean Air Force
Montana Conservation Voters
Montana Environmental Information Center
National Audubon Society
National Parks Conservation Association
Natural Resources Defense Council
Nevada Conservation League
New Virginia Majority
NextGen Climate
North Carolina League of Conservation Voters
Northern Plains Resource Council
Ocean Futures Society
Oceana
Ohio Environmental Council
Ohio Valley Environmental Coalition
Oil Change International
Oregon Aviation Watch
Oregon Environmental Council
Partnership for Policy Integrity
PennEnvironment
PennFuture
People’s Action
Pesticide Action Network
Physicians for Social Responsibility
Potomac Riverkeeper Network
Prevent Harm
Public Citizen
Renew Missouri
RESTORE: The North Woods
Rootskeeper.org
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
Texas League of Conservation Voters
The Alaska Center
The Climate Reality Project
The Climate Trust
The Environmental Justice Center at Chestnut Hill United Church
The Greenlining Institute
The Trustees
Union of Concerned Scientists
Virginia Organizing
Voices for Progress
WaterWatch of Oregon
Western Colorado Congress
Wholly H2O
Wilmington Improvement Network
Wiscon League of Conservation Voters
Wisconsin Environment
Young Fresnans for the Environment

https://www.nrdc.org/resources/letter-opposing-nomination-scott-pruitt-epa-administrator-173-organizations

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 9.

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 10. 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 (http://bit.ly/2fsbEnw). 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 (http://bit.ly/2fsbEnw).

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.

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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.


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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.

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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.11 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.

Summary

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).12

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.13 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 (http://www.facebook.com/#!/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.14 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: dz@center4research.org

 

To see the original letter, click here.

The excellent article by O’Connell et al.15 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,16 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.17 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?18 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.19 Several hypotheses have been suggested for implant-related ALCL, implicating an immunological mechanism likely attributable to tissue response to silicone.20,21 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.2,22 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.

Sarah Romano

Updated 2016

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 14.23 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)24

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.25 The second concussion will probably come with symptoms that last longer than the first.26 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.” 27

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.28

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.29

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.30 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. 31 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.32

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. 33

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).34

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.35

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.36

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, NJ.com

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.” 37 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 38. Studies also indicate these excessive consecutive work hours are harmful to patients, with a “greater than 2-fold increase in preventable adverse events.” 39 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.” 40

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 41.

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.” 42 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.

Sincerely,
Diana Zuckerman, PhD
President
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.43

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

Safety
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.44,45

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.

Conclusions
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.

cost-by-plan

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 https://localhelp.healthcare.gov/#intro to find local assistance.

 

Here are some useful links:

healthcare.gov

https://www.socialinterest.org/

 

[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]
and
“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.46  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.47

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.

Exemptions
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.”48 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.

Conclusions
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 (https://www.anthem.com/medicalpolicies/policies/mp_pw_c192386.htm).

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.

Off-Label

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.

Nonconformities

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.

Conclusions

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 ClinicalTrials.gov. 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 ClinicalTrials.gov 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 ClinicalTrials.gov. 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, clinicaltrials.gov 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).49 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.50  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.51 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.

Conclusion

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

A recent study of 173 women with cancer of the immune system caused by breast implants [1] 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.

Results

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: http://www.ncbi.nlm.nih.gov/pubmed/25490535

To read about another study on ALCL, click here.

To read more about what you need to know about ALCL, click here.

[1] Brody GSDeapen DTaylor CRPinter-Brown LHouse-Lightner SRAndersen JSCarlson GLechner MGEpstein 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?

Reference

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.

HERPES

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.52 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.53

Male anatomy

Female Anatomy

CHLAMYDIA

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. 54

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.55

GONORRHEA

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). [Diseases and Conditions: Gonorrhea (2014 January02). Retrieved June 13, 2016 from, http://www.mayoclinic.org/diseases-conditions/gonorrhea/basics/definition/CON-20020917?p=1]

Antibiotics can be taken to treat the infection, but will not reverse any damage that the bacteria caused before the treatment.56

SYPHILIS

Syphilis is not as common as the other STDs above. It is caused by bacteria that infect the lips, mouth, genital area, or anus.57

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.[Syphilis: Symptoms (2014 January 02). Retrieved June 13, 2016, from http://www.mayoclinic.org/diseases-conditions/syphilis/basics/symptoms/con-20021862] This can lead to neurosyphilis, which can result in impaired balance, chronic pain, lack of muscle coordination, and nausea.58

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 (HBV)

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. 59  

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.60 If you are diagnosed with chronic hepatitis B, you should see a doctor to develop a treatment plan. 

DIAGNOSES

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.

PREVENTION

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.61 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.62 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.  

This article was updated in 2016. All articles on our website have been approved by Dr. Diana Zuckerman and other senior staff.

 

 

 

Laura Gottschalk, PhD, National Center for Health Research

2016

 

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. http://bit.ly/2btczBT

[…]

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.

Sincerely,

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.

 

Conclusions

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]. https://www.federalregister.gov/articles/2016/07/19/2016-16916/prescription-drug-user-fee-act-public-meeting-request-for-comments

[2] Food and Drug Administration (December 7, 2015). What We Do. http://www.fda.gov/AboutFDA/WhatWeDo/

[3] Light DW, (July 17, 2013). Risky Drugs: Why The FDA Cannot Be Trusted, Harvard University, Edmond J. Safra Center for Ethics  http://ethics.harvard.edu/blog/risky-drugs-why-fda-cannot-be-trusted

[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. https://www.washingtonpost.com/business/economy/high-prices-make-once-neglected-orphan-drugs-a-booming-business/2016/08/04/539d0968-1e10-11e6-9c81-4be1c14fb8c8_story.html?_hsenc=p2ANqtz- _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. http://archinte.jamanetwork.com/article.aspx?articleid=2467779

 

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 center4research.org/child-teen-health/early-childhood-development/young-children-and-screen-time-television-dvds-computer

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

Stephanie Fox-Rawlings, PhD, National Center for Health Research

Polychlorinated bisphenyls (PCBs) were banned in the US in the late-1970s, but they still pose a danger to our health. They were widely used in electrical equipment (such as fluorescent light fixtures) and building materials (such as caulk and paint). Today they are found everywhere on Earth and in all people.

PCBs are endocrine disruptors, which means that they can mimic, block or change the effect of the body’s hormones.63 64 Exposure has been linked to increased risk for many different health problems, including cancer, diabetes, and increased risk of infection. Exposure early in life can cause children to have difficulties with learning and memory. In addition, PCBs can increase blood pressure, triglyceride levels and cholesterol levels, which can increase the risk for heart attack, diabetes, and stroke.

Once PCBs are in our air, water, soil, homes, communities, or bodies, most do not break down easily. This means PCB levels are still a problem for all of us decades after they have been banned and will continue to be a problem for decades in the future. It also means that they build up in people’s bodies over the years.

The good news is that PCB levels in the environment and in humans are much lower now than they were before they were banned.65 66

So are PCBs still a health risk? Sadly, yes. Recent studies have shown that current levels of exposure still harm children’s health, including brain development.

For example, higher levels of prenatal exposure to PCBs increased the risk of language delay in 3-year-olds67 and resulted in less ability to focus on a task in 11-year-olds.68 Prenatal exposure also affects immune system development: babies exposed to higher levels while in the womb tend to have more infections as infants and toddlers.69

Ongoing exposure also affects children’s development. A study of preschoolers found that higher levels of PCBs in house dust were linked to more anxiety/depression, aggression, and poorer motor skills.70

A study of 3-year-olds found that children exposed to higher levels of PCBs had poorer hearing ability.71 And a study of 7-9-year-olds found that children with higher levels of some PCBs tended to have higher blood pressure and triglyceride levels.72

People are primarily exposed to PCBs through food and air. For many people, the largest exposure comes from food, particularly meat, dairy and fish.73  PCBs build up in the fat of animals, so foods with more fat can contain more PCBs. However, for people who spend a lot of time in older buildings that were built with PCB materials, the air in these buildings is a major source of continued exposure.11 74 An estimated 13,000 to 26,000 schools in the US still contain PCB-contaminated building materials.75 In addition to schools, many other older buildings also have contaminated construction materials.

Fluorescent light fixtures, caulk, adhesives, sealants, floor finish, and paint were often produced with high concentrations of PCBs.1 PCBs from these materials contaminate surrounding brick, concrete, carpet, dust, and soil. These indirectly contaminated materials can also have high levels of PCBs. In addition, PCBs move from these products into the air.

The main source of air contamination in many buildings are the caulk used to seal windows and doors.76 This is because it was often made with very high concentrations of PCBs (up to several thousand parts per million).14 77 The levels of PCBs in the air of these buildings can be 10-to-hundreds of times greater than outdoor levels.11 13 78

Although it is illegal to purposely make PCBs, some are produced in the process of making pigments.79 80 81  Therefore, PCBs are found in some paints, inks, clothing, cosmetics, plastics, and food packaging. They may also be created when making silicone and vinyl chloride (primarily used to make PVC).82 These contribute to the buildup of PCBs in the environment and our bodies.83

There are ways to reduce exposure.

  • Schools and other buildings can hire experts to remove or seal PCB-contaminated84
  • Choose low fat dairy products and trim the fat from meat, because PCBs accumulate in fat.85
  • If you fish or hunt, watch local advisories to know if there are concerns about PCBs in fish or animals in that location.
  • Be careful around old electrical equipment if appears to have been leaking, because the coolant or lubricant could have high levels of PCBs. Wear gloves when handling the equipment because PCBs can be absorbed by skin. Don’t let children play near this equipment because the PCBs contaminate the surrounding soil and other materials. If you have contaminated materials, dispose them safely (see https://www.epa.gov/pcbs/managing-remediation-waste-polychlorinated-biphenyls-pcbs-cleanups).

This article was updated in 2016. All articles on our website have been approved by Dr. Diana Zuckerman and other senior staff. 

Diana Zuckerman, Chicago Tribune

July 21, 2016

This article was published in the Chicago Tribune, Baltimore Sun, Orlando Sentinel, Sacramento Bee, and syndicated in many other newspapers across the country.

Imagine that you or someone you love has a potentially fatal disease with no proven treatment, but there is a new experimental treatment available.

Would you rather be given that treatment for free by a top physician who carefully monitors your treatment as part of a clinical trial to study whether it works, or, would you rather pay more than $100,000 a year for the same experimental drug and hope your doctor gives you the right dose?

For more than a year, a bill misnamed the 21st Century Cures Act has been a major focus of Congress, with grand promises that it would save the lives of desperate patients, including those with rare diseases.

Despite unusual bipartisan support from Republicans and Democrats, the bill has not become law. Here’s why you should be relieved, rather than disappointed.

The 21st Century Cures Act, which some experts refer to as the 19th Century Fraud Act, is the worst threat I’ve seen to the health of adults and children, whether rich or poor.

Simply put, this proposed legislation is based on hype and hope, not reality.

It has been marketed like a Super Bowl halftime ad campaign featuring families with dying children praising members of Congress for giving them hope. Supporters have included scientists, university officials and investors who are likely to benefit financially, should the legislation become law.

Desperate patients also support it, not realizing it would pave the way for unsafe and ineffective medical treatments. Worse yet, those patients would need to pay hundreds of thousands of dollars a year while being unwitting guinea pigs in experiments that are falsely hyped as “promising treatments.”

There are thousands of patients who urgently need new medical treatments. Many feel like they have nothing to lose, and assume that a promising new treatment is their best hope. But new isn’t always better which is why too many promising new drugs are subsequently found to be a harmful waste of money.

If a patient has only one year to live and the new drug or device kills them even sooner, or causes a stroke or unrelenting nausea or horrible pain that won’t ever stop, it becomes obvious that they had something to lose after all.

That’s why these medical products were still being studied because decades of disasters such as Vioxx, metal hip replacements, DES, and thalidomide have taught us that there is a right way to test medical products. And that testing them the right way and waiting for the results to be conclusive will save more lives than taking short cuts.

One of the most dangerous aspects of the 21st Century Cures Act is that it urges the Food and Drug Administration to settle for preliminary study results based on “surrogate endpoints” which are little more than well-educated guesses that a treatment works, rather than scientific evidence of real benefits such as living longer or having fewer complications. The FDA already relies on those surrogate endpoints for new medical products more often than they should.

For example, a study published in one of America’s most prestigious medical journals found that half of new cancer drugs are approved based on such short cuts, but many of those promising results were later reversed when more conclusive studies show that the drugs do not help patients live longer.

The National Center for Health Research has found that many of those ineffective often toxic drugs cost more than $100,000 per year a cost that can bankrupt individual patients and also bankrupt Medicare.

You’d think Congress would want to stop this travesty, but campaign contributions and the power of well-connected lobbyists have convinced Congress that this bill is a good idea.

If you or a loved one has a deadly disease, whether it is cancer or a rare disease, you deserve treatments that work. If experimental drugs are your only hope, they should be free as part of a study that you can choose to participate in.

Changing the rules so that patients have to buy experimental treatments that could harm them is a dream come true for snake oil salesmen. We all deserve better.

To see original article, click here.

 

James Clark, Healio

July 21, 2016

A panel of the Medicare Evidence Development and Coverage Advisory Committee of the CMS voted that it has moderate confidence that there is sufficient evidence for intervention that improves health outcomes for certain adults with chronic venous disease.

The moderate-confidence vote applied to patients who have varicose veins and/or other indicators of chronic venous insufficiency and who present with symptoms.

However, the panel voted that it has low confidence of adequate evidence for an intervention for members of that cohort presenting without symptoms but with signs.

The panel also voted that it has low confidence of sufficient evidence for intervention that improves both immediate/near-term and long-term health outcomes for adults with chronic venous thrombosis — including individuals with post-thrombotic syndrome — who present with signs or symptoms. […]

Other panel members agreed that reliable data was lacking.

“Just about everything has an evidence gap for people over the age of 65,” Diana Zuckerman, PhD, president of the National Center for Health Research Cancer Prevention and Treatment Fund, said. “What we need are some good studies that are specific. That way, we can pursue effective, affordable outcomes.” […]

The purpose of the meeting was to examine the scientific evidence underpinning the benefit and risk of existing lower extremity chronic venous disease interventions that aim to improve health outcomes in the Medicare population, according to the CMS.

It also sought to identify evidence gaps that exist related to lower extremity chronic venous disease, including heterogeneous disorders that encompasses a variety of primary etiologies.

But, according to the CMS, common to most of these venous conditions is a permanent alteration in venous structure and function, which leads to altered venous hemodynamics.

To see original article, click here

Patient Advocates Wanted!

Researchers in government and at universities are listening to patients’ perspectives on the risks and benefits of treatments. They need to hear from patients like you! That’s why we are holding several free workshops for individuals who want to be advocates for patients. The Workshops will train patients and family members to understand research that evaluates the safety and effectiveness of treatments and prevention strategies, and teach them about opportunities to share their own views and perspectives in ways that will most benefit patients.

Most patient advocates help a small number of individual patients. Our goal is different. Our workshops and training are designed to help patient advocates get the information, skills, ad confidence they would need to help patients on a national level by influencing the development and evaluation of treatments used by patients across the country.​

You might be surprised to learn that many studies of new treatments measure changes that do not necessarily improve patients’ health or quality of life. For example, it is faster and less expensive to study whether a cancer drug shrinks tumors than to study whether that same drug helps a patient live longer or have a better quality of life. Some patients might be fine with taking a chance on a drug that is not proven to help them live longer, because they have run out of options. Others only want to try a new drug if it is likely to help them live longer or have fewer side effects. Those kinds of patient perspectives are very important to researchers and to health professionals, and we will help patients have the expertise they need to share their views with researchers and opinion leaders.

Our next workshop will be held on Friday October 14 and Saturday October 15, 2016. The 2-day workshop will be free, and meals will also be included. Hotel rooms and travel scholarships to Washington, DC will be available for those who live at least 40 miles away.

Anyone can apply, but we are especially interested in adults who have previously been involved in patient advocacy, either as an individual or as a member, volunteer, or staff for a nonprofit organization or government agency.

Please complete our application at http://goo.gl/forms/CxFrg61Qk15pxvyw1. Preference will be given to applications received by August 15, 2016. And feel free to share with other patients who are well enough to advocate for themselves and others.

Make your voice heard! 

Find out more about this year’s Patient Advocacy Workshop HERE.

Patient advocate Diana Levine discusses her experience with a serious adverse reaction at the June 12 Workshop

Patient advocate Diana Levine discusses her experiences, at the June 12, 2014 Workshop

By Erin Raftery, InsideHealthPolicy.com

July 18, 2016

In contrast to most other physician societies, the National Physicians Alliance opposes exempting journal articles and other so-called continuing medical education from Sunshine Act reporting because the group’s members say drug and medical device makers should not pay for educating doctors.

The Sunshine law requires drug and medical device companies to disclose payments and other items of value that they make to physicians. The law includes 12 exempted areas, including for educational materials that directly benefit patients or are intended for patient use, and CMS officials say medical textbooks and journal reprints are intended for doctors, not patients.

The American Medical Association and a large number of physician groups support a bill by Sen. John Barrasso (R-WY) that would exempt certain continuing medical education from Sunshine Act reports. The groups say S. 2978, the Protect Continuing Physician Education and Patient Care Act, “would protect the dissemination of peer and independent third-party reviewed services and products that improve patient care.” The legislation is important, the groups say, because it would make it easier to obtain independent peer-reviewed journals, medical textbooks and “continuing medical education.”

However, W. Bill Jordan, immediate past president of the National Physicians Alliance, said the medical journal publishers and companies that run continuing medical education events are doing well financially and don’t need to be exempted from reporting. He said such offers are gifts and doctors don’t need to rely on drug and device makers for them. Jordan also said journal reprints on the latest research about medication often is a backdoor way to market off-label uses to doctors. Jordan said article dissemination should be discouraged and viewed the same as off-label advertising.

Diana Zuckerman, president of the National Center for Health Research, said pharmaceutical and device companies sponsor most continuing medical education programs because they’ve got something to sell. Zuckerman said she doesn’t think the proposal will gain traction, in large part because Sen. Chuck Grassley (R-IA) opposes it. Zuckerman has previously said she hopes the Senate will fix the House bill’s language that exempts peer-reviewed journals, journal reprints, journal supplements and medical textbooks given to doctors at continuing medical education events as well as transfers of value to physicians for certain aspects of independent CME sessions from reporting under the Sunshine Act. She doubts the Senate includes the measure in its version of the 21st Century Cures bill.

By Joyce Frieden, MEDPAGE

July 5, 2016

The Affordable Care Act (ACA), high prescription drug costs, and reproductive health all figure prominently in the Democratic Party’s new draft platform.

The draft platform, released Friday, touched several other items of interest to health analysts, including community health centers, gun violence, and mental health.

The health insurance plank in the draft platform hewed to presumed Democratic presidential nominee Hillary Clinton’s goal of improving upon the ACA, rather than moving toward the goal of her challenger, Sen. Bernie Sanders (I-Vt.), who wants to replace the current system with a single-payer system. “Thanks to the hard work of President Obama and Democrats in Congress we took a critically important step towards the goal of universal health care by passing the ACA, which has offered coverage to 20 million more Americans and ensured millions more will never be denied coverage on account of a pre-existing condition,” the document read.

However, the draft platform did offer a slight concession to advocates for single-payer, along with a knock at Clinton’s presumed Republican opponent: “Democrats will never falter in our generations-long fight to guarantee health coverage as a fundamental right for every American. As part of that guarantee, Americans should be able to access public coverage through Medicare or a public option. By contrast, Donald Trump wants to repeal the ACA, leaving tens of millions of Americans without coverage.”

[…]

In an email, Diana Zuckerman, PhD, president of the National Center for Health Research in Washington, called the draft platform “very solid.” “It is important that the platform emphasizes the need for NIH research as the strategy needed to develop new cures and treatments.”

She praised the draft platform’s rejection of efforts by Congress and the Obama administration to lower approval standards at the FDA, “even if it does so by not mentioning it rather than directly opposing it. Recent national polls show that Americans are more concerned about the safety and effectiveness of [currently available] medical products than they are about having ‘new’ ones.”

The draft platform’s support for lowering drug prices by importing drugs from licensed pharmacies in Canada “is an extremely important step because it would have an immediate, measurable impact,” said Zuckerman. “Several other platform strategies for lowering drug prices would also be very effective in the short-term and long-term.”

Zuckerman liked the document’s inclusion of sexual assault as a public health issue, but was concerned that sexual assault in the military wasn’t specifically included.

The Democrats will be holding a last set of platform hearings this Friday and Saturday in Orlando and then finalizing the platform for passage at the upcoming Democratic National Convention in Philadelphia later this month.

To see original article, click here.

By Michelle Llamas, Drugwatch

2016

Convincing people they are sick and need a drug is a multi-billion dollar industry. In 2015, Big Pharma dropped a record-breaking $5.4 billion on direct-to-consumer (DTC) ads, according to Kantar Media. And it paid off for Big Pharma. The same year, Americans spent a record $457 billion on prescription drugs. The U.S. and New Zealand are the only countries where DTC is legal. Americans also pay more for drugs and devices than any other country.

The bulk of these ads appear on TV at a rate of 80 ads per hour of programming, according to Nielsen. Behind the drug and device ads saturating TV, radio and digital media are hidden costs and devastating side effects that companies don’t advertise, and critics say the ads drive up drug prices and erode the patient-doctor relationship.

With the price of drugs skyrocketing, politicians and health-care providers question Pharma’s DTC spending, which exceeds money spent on research and development. Even presidential hopeful Hillary Clinton called for an end to tax breaks for drug ads and for tougher regulations.

But the money spent on DTC is just one small cog in Big Pharma’s well-oiled marketing machine. Companies spend billions more on getting doctors to write prescriptions for their expensive brand-name drugs or devices for uses not approved by the Food and Drug Administration — a controversial practice called off-label marketing. […]

“Americans tend to think newer is better. If it costs more, therefore it’s better. If it’s new and it costs more, then certainly it is better,” Diana Zuckerman, president of the National Center for Health Research, told Drugwatch. “They sometimes think that the FDA only approves new drugs if they are better. There is no requirement in the law, nor does the FDA require that a new product be better. I’ve heard FDA officials say, ‘We’ve approved this drug. It doesn’t mean we recommend it.” […]

“Obviously, the companies are paying a lot of money for these ads for a reason. They know it affects how many people take these drugs and how many prescriptions are written. And to pretend that they don’t know what the impact is seems disingenuous at best.” […]

“If the real goal is to educate, they wouldn’t look like this. The goal is to persuade. These ads educate people as much as the ads for the GAP or ads for makeup educate. It is educating you to tell you that this product exists and that it’s great.” […]

“The FDA has written policies that sound good, but the reality is very different from the written policies.” […]

To see original article, click here

By Claire ZillmanFortune

July 12, 2016

Ikea expanded the recall of its problematic Malm dressers to China on Tuesday as the country’s General Administration of Quality Supervision, Inspection, and Quarantine announced that the Swedish retailer had submitted a recall plan that applies to about 1.7 million products in mainland China.

In late June, Ikea announced the recall of 8 million Malm dressers and 21 million other chests in the U.S. and 6.6 million units in Canada. That notice followed the deaths of three children who were crushed by the dressers over a two-year span. Ikea had introduced a repair program in July 2015 that urged customers to attach Malm units to the wall, but it reexamined its response to the tip-over risk after the third child’s death in February 2016.

The June recall’s limited geographical scope irked regulators in China. The nation’s official Xinhau news agency published an editorial on Sunday that said Ikea’s decision against a more comprehensive recall illustrated the furniture maker’s “arrogance” and irresponsibility. Ikea told Fortune earlier this year that it sold 65 million Malm units worldwide over 13 years. Ikea said last week that it would offer Chinese customers free anchoring kits, but it would not recall the Malm products in China since the units met local standards. Ikea’s recall also didn’t apply to the EU, the company’s biggest market.[…]

Ikea’s approach to its troublesome Malm series has prompted criticism in other arenas too. In April, U.S. safety officials blasted the retailer for its attempt to fix the tip-over problem by telling customers to attach the item to a wall. The “repair” campaign didn’t mention the word “recall,” and representatives from groups like the Consumer Federation of America and the National Center for Health Research said that language choice “reduce[d] the impact” of their efforts to “educate and encourage consumers to recognize the need for action when they hear about a recall.” Safety officials also said the Malm didn’t meet the U.S.’s voluntary industry standards that require each drawer of a dresser to withstand 50 pounds of weight, and that they should stop being sold.

In announcing the recall in North America in June, Ikea said that “taking further action was the right thing to do.”

To see original article, click here

Paul Brown, Tracy Rupp, and Steven Findlay

Health Affairs

July 11, 2016

The full Senate may in the next few days consider companion legislation to the 21st Century Cures Act that passed the House last year. The legislation—currently 19 separate bills—makes substantial changes to the way the Food and Drug Administration (FDA) approves drugs and devices. Set to adjourn for an extended election-year summer recess on July 15, the clock is ticking. The congressional calendar in the fall is full and the Senate may simply not have the time to take up the complex legislation, and reconcile it with the House version, before the November elections.

We believe that’s a good thing. The legislation, while containing some useful measures, is flawed. Instead of hastily agreeing to it, Congress should postpone consideration until 2017 and attach the best of the 19 bills (see below) to must-pass legislation on FDA funding through industry user fees.

At that time, we believe the Senate should reject those bills that lower standards for drug and device approvals and safety, and risk adding to the rising cost of prescription drugs.

This is a story two years in the making. Proponents of the proposed legislation—drug and device companies, and members of both parties in the House and Senate—argue that the FDA stifles innovation and advances in treatment by approving drugs and devices too slowly compared to other countries.

That premise is faulty. Nearly two-thirds of the novel drugs approved in 2015, for example—29 of 45, 64 percent — were approved in the United States before being approved in any other country. The proportion was even higher in 2012 and 2013. The majority of these drugs (60 percent) took advantage of existing FDA expedited review programs—fast track, breakthrough, priority review, and accelerated approval—and nearly half (47 percent) were approved to treat rare or orphan diseases.

As for devices, research shows that “it takes the same amount of time or less for patients to gain access to innovative, high-risk medical devices” in the U.S. as compared to Germany, France, Italy, and Britain.

The House and Senate bills ignore the above facts. They essentially seek to speed-up the approval process by relaxing FDA’s safety and effectiveness standards. And to make that more palatable, sponsors have attached the changes to increases in funding for the National Institutes of Health and the FDA.

But while the public supports increases in biomedical research funding, it is deeply skeptical about lowering the standards for drug and device approval. In the most recent poll on this issue, just under 60 percent of Americans opposed changing federal regulations to speed the development and approval of drugs; 38 percent favored speedier FDA action, according to a STAT-Harvard poll released in May. Respondents’ main concern: faster approval would allow products on the market that don’t work or are unsafe.

The survey echoed a previous poll by Consumers Union that found 82 percent of Americans believe that preventing safety problems is more important than limiting safety testing to speed the clearance or approval of devices or promote innovation.

The 21st Century Cures Act received broad bipartisan support in the House in part because it put significant amount of money for research on the table. The House bill pledges a $9 billion increase in mandatory funding for the National Institutes of Health over five years, gaining the support of universities and medical schools. It also promises $550 million to the FDA (which, according to the CBO’s analysis, would not fully pay for the additional workload the Act assigns the FDA).

The drug and device industries intensely lobbied House members to pass the legislation. The Pharmaceutical Research and Manufacturing Association (PhRMA) increased its lobbying from $4 million to $5.4 million in the quarter before the 21st Century Cures Act passed. The Advanced Medical Technology Association upped its spending from $550,000 to $740,000 in the same quarter. The Senate’s lobbying database listed more than 1,100 lobbyists working on the legislation. Many patient groups have also voiced their support for the legislation, based on their belief that the bill would result in more treatment options. An analysis of public comments for the House bill completed by Avalere noted that 43 percent were from patient advocacy groups.

The Senate bills are an improvement over the House’s 21st Century Cures Act. But, on balance, they are still weighted heavily in favor of speeding medical products to market by weakening FDA approval standards.

New drugs and devices can, of course, be an improvement over existing products. And when they clearly are, FDA has established pathways to get them to market and patients as fast as possible. The agency, for example, grants more than one-third of requests from industry for “breakthrough” designation for new drugs. But the history of medicine is replete with examples of drugs and devices that caused more harm than good, some of which were approved too hastily — such as Avastin for breast cancer, Vioxx for arthritis, metal-on-metal hip implants, and power morcellators. Innovative drugs and devices simply must be required to actually work and not harm patients. Methods of testing drugs and devices in patients are improving all the time, with new tools that help detect products that warrant further testing or could be dangerous. The default mode of this technical process must always be “safety first.”

Below are our concerns with some of the Senate bills followed by a list of bills we believe would improve public health.

Senate bills that would, in our opinion, increase risks to patients:

  • The MEDTECH Act would prevent the FDA from collecting adverse events due to flawed electronic medical records, and from recalling certain types of defective medical software. Some of this software has had life-threatening flaws in the past, such asoncology electronic medical record systems that calculated and recorded incorrect drug dosages for highly toxic chemotherapy drugs.
  • The PATH Act would allow antibiotics to be approved with minimal evidence of safety and effectiveness through a “limited population” approval pathway. But, antibiotics approved in this way are promoted by companies so that they are more widely prescribed in order to increase sales. As they are, we won’t have information about whether they’re actually safe or effective for those groups of patients. For example, fluoroquinolones are today widely overprescribed for urinary tract infections despite guidelines recommending other antibiotics and data linking their overuse to the development of resistance.
  • The Advancing Breakthrough Devices for Patients Act would encourage shorter and smaller clinical trials for medical devices. Abbreviated clinical trials will make it difficult, if not impossible, to include sufficient participation from subpopulations such as women, seniors, and racial and ethnic minorities in the analysis of the trials. For example, a recent study of high-risk medical devices found that the median number of participants was only 65 patients. With so few patients, it’s difficult to draw reliable conclusions about safety and effectiveness, especially for subgroups of patients. In an increasingly diverse America, this is unacceptable. The bill also pushes FDA to rely on post-market studies for devices rather than ensuring safety effectiveness before hospitals and patients pay for the products.
  • The Advancing Hope Act would continue the existing pediatric priority review voucher program through 2022. The program is currently set to expire at the end of September. The program’s ability to stimulate innovation is questionable: a recent GAO review of the program concluded that the six drugs for which vouchers have been awarded so far were in development before the program existed. By allowing drug makers to buy a priority review, the bill removes FDA’s ability to set its work priorities and resource allocations based on public health needs. In a time of threats such as the Zika virus, our government agencies must be able to prioritize public health, and not be bound by vouchers that were sold to the highest corporate bidder.

Senate bills likely to promote innovation and protect public health:

  • The Preventing Superbugs and Protecting Patients Act will help prevent drug-resistant infections from contaminated duodenoscopes and other reusable medical devices that have caused harm and deaths. If enacted, this bill will require certain reusable medical devices to have validated cleaning, disinfection, and sterilization procedures prior to marketing. This will reduce the likelihood that these devices will transmit dangerous infections from patient to patient.
  • The Advancing Research for Neurological Diseases Act will help people suffering from neurological diseases by providing critical information to researchers as they work on new treatments and cures. The bill requires the Department of Health and Human Services to collect neurological disease surveillance data, such as demographic information, risk factors, outcomes, and treatments. Providing such information is likely to enhance understanding of these diseases and the development of new treatments.
  • The Next Generation Researchers Act recognizes the importance of investing in the brightest young researchers to ensure that the United States remains at the forefront of biomedical research.
  • The FDA and NIH Workforce Authorities Modernization Act will make it easier for FDA to recruit and retain top scientific and technical experts by making salaries more competitive with those offered by industry. It will also lead to the development of standards for regenerative medicine — such as replacing, engineering, or regenerating human cells, tissues, or organs to restore or establish normal function.

Lawmakers are also considering adding the REGROW Act to the Senate’s package of bills. The bill would allow complex regenerative medicine therapies to be conditionally approved based on preliminary evidence. Since at least half of all drugs fail in the last stage of testing, many patients could end up receiving therapies that are later found to be unsafe or ineffective.

Looking Ahead

On Saturday June 25, six former FDA commissioners from Democratic and Republican administrations suggested at the Aspen Ideas Festival that Congress make the agency independent of the Department of Health and Human Services — similar to the Securities Exchange Commission, for example. With regulatory purview over products that represent a quarter of the U.S. economy, the group said the FDA is harmed by an unstable federal budget process and persistent political meddling. The group said they would issue a white paper on their proposal for the next administration. That’s another reason why Congress should postpone consideration of these bills until 2017.

Read the full article here.

By Celia WexlerWho.What.Why.

July 11, 2016

EU Trade Commissioner Cecilia Malmström listed several priorities for the European Union when it resumes trade negotiations with the United States on July 11. They include: […]

• Greater regulatory cooperation for prescription drugs and medical devices. […]

Prescription drugs and medical devices

Malmström insists that the EU and the US “have the best regulators in the world,” who should be figuring out ways to collaborate more intensively. If regulators can “work together to share knowledge, data and expertise,” that can create “very high global standards.”

An EU factsheet calls for regulators on both sides of the Atlantic to meet together, and work out common standards and share data. The EU also proposes that companies should have to undergo only one clinical trial, rather than separate trials in the US and the EU.

But Diana Zuckerman sees dangers in the EU’s push for more regulatory harmonization for drugs and medical devices. Zuckerman, president of the National Center for Health Research, says that there are significant differences in the way the US and the EU regulate.

Zuckerman has led efforts to strengthen US Food and Drug Administration (FDA) oversight of drugs and devices, which she has criticized for not being strong enough. Even so, Zuckerman believes that adopting an EU approval process would mean even lower standards for the US.

The EU does not regulate either pharmaceutical drugs or medical devices as rigorously as the FDA, she says. Medical devices, in particular, can be sold throughout the EU if a third-party company reviews the device and finds it safe.

The device maker pays the third-party company, so the pressure is on the reviewing company to approve the device. “No third-party company can stay in business unless they approve,” she says.

The EU’s lax oversight led to tens of thousands of European women receiving industrial-grade silicone in their breast implants, rather than sterile medical-grade silicone. As a result, the implants were prone to leakage, exposing women to an untested chemical whose damage to human health continues to be assessed. The FDA did not permit the product to be sold in the US.

But the situation is more complicated than that.

In some respects, companies often find it harder to sell their products in the EU, she says. That’s because drug and device companies must scale another hurdle. Since most European countries pay for their citizens’ health care, governments determine which devices and drugs they will purchase.

And in many cases, their standards are very high. Many government payers, Zuckerman says, not only insist that a drug or device be safe and effective, but that it is better than medical products that are older and well established, and usually less costly.

There is another public health and safety protection that the US lacks, she adds. In the EU, companies are not permitted to advertise their products. So physicians aren’t being pressured by patients who want what they see being hyped on television.

She has no problem with one set of rules for drugs and devices, she says, but those rules should benefit patients, not companies. Those rules should include the FDA’s more stringent approval process and the EU’s restrictions on advertising, and government reimbursement for medical products.

She worries that drug and device companies will successfully lobby for the opposite: the EU’s relaxed approval process, along with the freedom to sell and market their products with no restrictions in the US. That could mean that the US has to accept lightly regulated medical products, with Medicaid and Medicare having to foot the bill for them.

To see original article, click here

Natalie Rosseau

2016

Did you know that more than 230 million passengers are scheduled to fly on U.S. airlines this summer, setting a record high for summer air travel? If you or your loved ones are among them, here are some tips for safe and healthy flights this summer (and some are good for other long-distance travelers too):

  • Protect yourself from Deep Vein Thrombosis (DVT)! Sitting without moving around puts airline passengers at an increased risk of blood clots. These clots become life-threatening if a piece of the clot travels and blocks a main artery of the lungs (pulmonary embolism). Anyone could develop a DVT, but it is more common among older travelers, passengers on long flights, pregnant women, people who are overweight, those who have had surgery recently, and women taking oral contraceptives or hormone replacement therapy. To reduce your risk of blood clots, avoid crossing your legs, wear loose clothing, walk around and stretch your feet and ankles throughout the flight, wear compression stockings, stay hydrated (more water, less alcohol & sugary beverages), and stretch out your legs (remove items under the seat in front of you).
  • Carry any medications that you are taking and your medical insurance card in your carry-on luggage. This is important in case your checked luggage is lost or delayed.
  • If you are traveling internationally, research health-related conditions in the country that you are visiting. Be sure that your immunizations are current.
  • Delay your travels if you recently had a concussion, significant sinus congestion, serious respiratory illness, a colonoscopy, abdominal, cardiac, orthopedic, or neurosurgery, surgery on a lower extremity, or extensive dental work. For these conditions, in-flight pressure changes can lead to significant discomfort and/or serious medical complications. For medical reasons, experts recommend that you cancel your trip if you are more than 36 weeks pregnant, or plan to travel with a baby who is less than 2 days old. If you are planning to travel with a cast, the injured area may swell or hurt during the flight; ask your physician for a splint or soft cast, and be sure to keep the limb elevated while on the plane. If you are considering canceling or postponing your flight due to illness, talk to an airline representative. Some companies will waive change/cancellation fees under certain emergency situations if you send them a doctor’s note. Purchasing travel insurance while booking your trip often covers the cost of changes due to sickness.
  • Those with common contagious illnesses who are hesitant to cancel their flight should take precautions to prevent spreading their sickness to other passengers. Make sure to thoroughly wash your hands, drink water to remain hydrated, and cover your mouth and nose while coughing or sneezing. Consult a physician if you have any questions.

Airplane Travel Safety Tips (2)

Simon Essig Aberg

2016

Adderall picApproximately 2.5 million Americans are prescribed prescription stimulants such as Adderall or Ritalin to treat Attention Deficit Hyperactivity Disorder (ADHD).86 ADHD is a brain disorder that makes it difficult to concentrate and increases impulsive behavior.87 Prescription stimulants help to reduce these symptoms. However, many people use these drugs for non-medical purposes and without a prescription, especially college students who buy them from a friend with a prescription.1

The use of drugs like Adderall and other so-called “study drugs” has skyrocketed over the past two decades. One reason is that the Food and Drug Administration (FDA) approved a version of the drug, Adderall XR, which lasts much longer than other prescription stimulants. Another reason is that more young Americans are diagnosed with ADHD and this results in more prescriptions. In fact, over the past decade, the manufacturing of prescription stimulants has increased by a whopping 9 million percent!88

How many of these pills are used to treat ADHD? A study of more than 10,000 college students from across the country found that more than half of students with an Adderall or other ADHD drug prescription were asked to sell the medication to peers and friends.89 Almost every student who abuses these drugs in college gets the drug from a friend or classmate with ADHD.1 One study of students at Bates College, a small Maine liberal arts college, found that one in every three students had abused Adderall at some point.90 In contrast, a national study of 10,000 students found that approximately 7% had abused “study drugs,” although the percentage varied at different colleges.4 Whether the typical abuse rate is 7% or 33%, or something in between, the extent of the problem is alarming.

People who abuse Adderall and similar drugs tend to have several characteristics in common. For example, a disproportionate number are white, in college, and belong to a fraternity or sorority. Because these drugs are commonly used to help students focus on reading or studying, abusers tend to have grade point averages of a B or lower.4

Causes and Common Justifications

Not surprisingly, around three quarters of prescription stimulant-abusers use the drug for academic purposes—to help them stay awake, focus, and study before a big exam.1 However, ADHD medications do not make students ‘smarter.’ It may help them focus and stay awake, but abusers should not expect the drugs to help with more complex learning such as writing better papers or studying for college-level exams.91

Prescription stimulant abuse may also occur for social reasons because students want to stay up later than they would otherwise. Some students claim that taking Adderall makes them more talkative and better company. Additionally, about a third of students have taken to get high or experiment. Most of those that take the drug for this reason are men. These experimenters usually only use the drug to get high a few times, whereas regular users tend to use stimulants for academic purposes. Approximately one in five women who take the drug do so to lose weight.92 Because Adderall and other stimulants reduce appetite, some abusers see it as a means to simultaneously study and stay in shape. However, taking these drugs to reduce appetite is unhealthy for a number of reasons, especially because the body does not get the nutrients that it needs.

Many students with an Adderall or other ADHD prescription believe that distributing the drug is legal and a good way to make money. However, these drugs are Schedule II drugs, meaning the Drug Enforcement Agency has labelled it highly addictive. The minimum sentence for distributing a Schedule II drug like Adderall is five years in prison.4 Very few students are aware of this and it is not commonly enforced.

Why “Study Drug” Abuse is a Problem

Students often do not feel guilty after abusing Adderall and similar drugs because they believe they are self-medicating for an attention deficit disorder or do not consider the drugs to be as dangerous or illegal as other Schedule II drugs, such as cocaine or opioids. Educators, however, may need to decide if the abuse of “study drugs” is comparable to an athlete using steroids, and if more attention should be paid to the prevention and punishment of illegal prescription stimulant use. Even though these drugs do not make students smarter, does using them the night before the test to stay up cramming give a student an unfair advantage the next day? Even if the advantage is not greater than caffeine, should it be treated differently if the drug was illegally obtained?

Every drug comes with risks, and Adderall and other ADHD drugs have more risks than many other commonly used medications. When physicians prescribe a patient that has ADHD a drug like Adderall, they should carefully weigh the risks against the benefits it provides. Students who abuse these stimulants often do not consider the risks of the drug either because they are not aware of them or they assume that, as an FDA-approved drug, there are no risks. The most common side effect of Adderall and other stimulants is insomnia, which appeals to students who use it stay awake. In short-term studies of Adderall’s safety, conducted by the manufacturer, they found that the drug caused gastrointestinal problems, blurred vision, increased body temperature, increased blood pressure, increased heart rate, reduced circulation, and irritability. While these effects are minor for most young people, if a student has a heart condition, Adderall could be deadly. In rare cases, Adderall can cause hallucinations, cardiac arrests, and even death for people with a heart condition. The likelihood of these risks occurring increases if Adderall is used without a prescription and at higher than recommended dosages, as often is the case among abusers.93 Similarly, a study of Ritalin found that the drug significantly increases risk for heart arrhythmia, and, for users with pre-existing heart conditions, may lead to serious conditions like heart failure.94

Bottom Line

The attitudes of parents, students, college administrators, and law enforcement towards Adderall and Ritalin abuse have been ambivalent; since they are used for studying, they are often assumed to be just a little different than caffeine. However, if students have serious attention issues, they should see a doctor for a medical diagnosis instead of asking a friend to illegally use Adderall or other prescription stimulants. At the very least, students need to be aware of the serious risks that can occur; although the most serious are rare, that doesn’t matter if it happens to you or someone you care about. Parents, health care professionals, counselors, and college administrators should be sure to include Adderall and other “study drugs” in any conversation about the dangers of substance abuse.

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

Related Articles:
ADHD in children: treatment options
ADHD treatment: medications and alternatives
Are Bisphenol A (BPA) plastic products safe?

By John Fauber and Kristina Fiore, Milwaukee Journal Sentinel

July 5, 2016

Drug companies have made billions of dollars treating erectile dysfunction in men.

Seeing the potential for billions more, they have turned to a condition some doctors don’t see as a medical disorder at all: lack of interest in sex.

A diminishing interest in sex naturally occurs as people age. It is not life-threatening. But the treatments created to date carry substantial physical risk with minimal benefit. And for years regulators have allowed them on the market.

Consider the new drug Addyi and the condition known as female sexual interest/arousal disorder.

Low sexual desire has dubious origins as a medical condition. Some 43% of women were said to suffer from some form of sexual dysfunction, including many who simply lacked interest in sex, according to a 1999 paper. But that study was conducted by researchers with drug-company ties and did not measure what percent were distressed by it.

And Addyi, termed the “female Viagra,” can cause a dramatic and dangerous drop in blood pressure if alcohol is used. The U.S. Food and Drug Administration — which had twice rejected the drug — ordered Sprout Pharmaceuticals to do tests on this point.

Sprout conducted a test of only 25 people.

Of those, 23 were men.

Several passed out or had dangerous blood pressure drops of 22 to 48 points.

The FDA staff member reviewing the drug noted that female nondrinkers may have an even more severe reaction to the drug and said it shouldn’t be approved until a more thorough test was done involving women — “the only gender for whom this drug is indicated.”

The FDA approved Addyi anyway.

“I disagree with that on so many different levels,” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit think tank that analyzes health research.

“You call something a medical condition; you find a solution for it; the solution actually doesn’t work and it has substantial risk,” she said. “The scientists say don’t approve it and the FDA approves it.”

Less than 48 hours after Sprout won approval for Addyi, its owners sold the small company and the rights to the drug to Valeant for $1 billion.

And Addyi, the first drug approved to treat lack of interest in sex among women, soon was being prescribed at $800 per month — a little pink pill for women billed as a counterpoint to the little blue Viagra pill for men. […]

A Journal Sentinel/MedPage Today investigation into how lack of interest in sex among men and women entered mainstream medicine found drug-company influence at every step in the process.

For instance, in 2013 when the FDA rejected Addyi for the second time, it noted the drug’s benefits were modest at best, providing a slight increase in sexually satisfying events (less than one per month) compared with a placebo. Those benefits, regulators determined, did not outweigh the potential risks.

What followed was an extensive political campaign that charged the FDA with sexism for not approving a sexual medication for women despite having approved Viagra, Cialis and a host of testosterone products for men.

Much of the pressure came from the advocacy group “Even the Score,” which — according to an editorial in the April edition of JAMA Internal Medicine — was partially funded by Sprout Pharmaceuticals.

The group persuaded several members of Congress to sign a letter to then-FDA Commissioner Margaret Hamburg that called on her to approve the drug. Even the Score also submitted a petition with tens of thousands of signatures.

Ultimately, Addyi was cleared for use.

As for the small study involving mostly men, a review of FDA documents found the agency accepted Sprout’s contention that it could not find moderate female drinkers to enroll.

In an email response to questions, FDA spokeswoman Sarah Peddicord said the agency recognized the drug’s serious side effects and told the company that markets Addyi to conduct three studies in women to better understand the risk of sudden blood-pressure drops that can occur.

The agency also has required doctors who prescribe Addyi and pharmacies that dispense it to be certified in a program designed to reduce the risks of the drug — though the program amounts to a PowerPoint produced by the drug company and completed on its website.

The drug carries the FDA’s most stringent warning. Women who use it are advised not to drink.

“As with all medications, some patients will obtain more benefit than others,” Peddicord said. “Because (the condition) is symptomatic, patients can gauge whether they have meaningful benefit from Addyi.”

A paper published in April in JAMA Internal Medicine found that Addyi produced just 0.5 additional satisfying sexual events per month for women on the drug compared with a placebo.[…]

Valeant, the company that markets Addyi, said that while women may have a drop in blood pressure or pass out if they use alcohol, the incidence of that is low — though the company’s statement did not cite any numbers. It said the ongoing studies will evaluate the “real world” effects of alcohol in women who use the drug.

“Our goal is to make Addyi affordable and accessible to the millions of women (with the condition),” the statement said. “We believe this product has strong potential and are committed to realizing that potential.”

Valeant has been embroiled in a price-gouging scandal since 2015, for vastly inflating the price of two heart drugs it acquired, and for its connections to Philidor, a mail-order pharmacy that allegedly steered customers to expensive Valeant drugs.

The company has been under investigation by two U.S. attorneys and by the U.S. Securities and Exchange Commission.

Unlike omnipresent ads for erectile dysfunction drugs, Addyi will not be peddled in commercials — at least for the next year or so. In an agreement with the FDA, the company has said it will not embark on TV or radio advertising for 18 months, a period that started with the drug’s approval in August 2015. […]

To see original article, click here

Diana Zuckerman, Ph.D., Health Progress

July/August 2016

Bullying used to be considered an unfortunate, inevitable rite of childhood, but researchers now tell us that bullying often occurs in conjunction with more serious aggressive and antisocial behavior. They conclude that bullying, therefore, should not be considered a normal and accepted part of growing up.

Usually defined as the use of physical or emotional power to control or harm others, bullying can include making threats, spreading rumors, physical or verbal attacks and name-calling or intentionally excluding someone from a group. To be called bullying, the behavior must have the potential to happen more than once. […]

Why is it so difficult to prevent bullying? Decades of psychology research shows that people who witness others in trouble are more likely to help if they are the only ones available to do so. When bullying takes place in front of an audience, it is more difficult to stop.

In general, people are more heroic when somebody is completely dependent on them. Whether the problem behavior is bullying, a physical attack, sexual harassment or racial slurs, each “bystander” tends to look to see how others respond before saying or doing anything. As the bystanders see each other hesitating, subconsciously trying to figure out the “appropriate” way to respond, the minutes pass, they see each other doing nothing, inaction becomes the norm and the chances increase that nobody will help.

The bottom line is that while bullying is acknowledged to be a serious and widespread problem in the U.S., the only bullying prevention programs that have been evaluated are programs for children and teens, and those evaluations often lack rigor. Little is known about which programs work best in which types of schools or for which types of students.19

We have come a long way in recognizing that bullying needs to be taken seriously — but we haven’t made much progress in actually reducing it. To do so requires all of us to tackle a larger problem in our society: people who flaunt the power they have at the expense of others, and the hesitation of those watching to muster the courage to stop it.

To see more click here.

Testimony of Dr. Laura Gottschalk
FDA Meeting of the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee

 

June 29, 2016

Thank you for the opportunity to speak today. My name is Dr. Laura Gottschalk. I received my Ph.D. from Johns Hopkins School of Medicine and previously worked as a cancer researcher. I am speaking today on behalf of the National Center for Health Research. Our research center scrutinizes scientific and medical data and provides objective health information to patients, providers, and policy makers. We do not accept funding from pharmaceutical companies, and therefore I have no conflicts of interest.

The passage of the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act has resulted in labeling changes for hundreds of drugs so that they may be used in pediatric populations. However, despite this success and advances in both basic science and clinical trials in pediatrics, off-label drug use in children and adolescents remains a problem. Moreover, off-label use of drugs presents an even larger and more complex issue for children with chronic and/or rare diseases like the cancers discussed here.

That’s why we strongly support FDA advisory committee meetings, such as this one, to garner input from experts on how best to conduct clinical trials in pediatric patients.  The panel has done a great job in asking specific questions of the drug sponsors on their trial design while also offering helpful suggestions and input when needed. However, despite the extraordinarily rare populations of patients to test these drugs, the scientific integrity of these trials needs to be kept in mind while moving forward. When possible, randomized or well-matched control group/comparison samples for new drugs should be used, because it is the ethical and scientifically valid design for proving whether a product is safe and effective. When a proper control group is not available, a robust single agent response rate should be observed in support of drug efficacy.

During the analysis of these proposed clinical trials, also keep in mind the possible pitfalls associated with using biomarkers and surrogate endpoints in lieu of overall survival. A study published last year looked at cancer drugs approved over 5 years using surrogate endpoints. In post-market studies, only 14% of these approved cancer drugs were found to improve patients’ survival.  And yet, our Center found that all the unproven cancer drugs were still on the market, many costing more than $100,000 per year.  These results show that surrogate endpoints such as objective response rate too often provide false hope while costing patients more they can afford.

Additionally, as discussed with several of the drugs, there are clearly subpopulations of patients who respond better to treatment than others. We encourage the sponsors to further characterize the positive responders in hopes of targeting the population of patients who would benefit the most from their treatment.

In conclusion, we realize that all five of the drugs discussed at this meeting are for treating very rare pediatric cancers that desperately need new treatments. For that very reason, if approved, these drugs may be tempting to use off-label in pediatric patients. Therefore, we commend the FDA and the panel for providing an open discussion on the best way in which to test these five new drugs in pediatric populations. This is a step in the right direction to help ensure that drugs are safe and effective for everyone for whom they are prescribed.

Thank you for your time.

June 28, 2016

On June 28, 2016 the Consumer Product Safety Commission (CPSC) and IKEA announced the recall of 29 million IKEA Malm dressers responsible for the deaths of three toddlers in tip-over accidents. IKEA has agreed to immediately stop selling the dressers and is offering consumers either full or partial refunds or a free wall-anchoring kit. IKEA has sold 65 million of these dressers worldwide over the past thirteen years.

The National Center for Health Research, together with the American Academy of Pediatrics, Consumers Union, Consumer Federation of America, Kids in Danger, and the Shane’s Foundation, issued the following joint statement:

“This recall is critically important to protect children and prevent future injuries and deaths caused by these dangerous IKEA dressers. While we wish that this safety hazard had been addressed sooner to protect young lives, we’re pleased that this recall will stop the sale of these dressers and give consumers options for a refund or the assistance to make these products safer by anchoring them to the wall. This recall is significant—involving 29 million products and exceptional remedies available to consumers. We commend staff at the CPSC for their hard work to secure a recall that also commits IKEA to designing and selling furniture that meets safety standards going forward. IKEA must now pull out all the stops and do everything it can to ensure consumers know about the recall, are aware of their options, and can easily take action to make their homes safer. We urge consumers to take the steps to remove or repair these dressers, and minimize all furniture and appliance tip-over hazards in their homes.”

In April 2016, the National Center for Health Research joined other safety groups in a letter to CPSC Chairman Elliot Kaye calling on the safety agency to take strong, immediate action to better protect children from the tip-over hazard of certain IKEA Malm dressers. The letter followed a Philadelphia Inquirer report of the February death of a 22-month-old boy in Apple Valley, Minnesota, who died when the Malm dresser in his room tipped over on him. His death was the third confirmed tip-over death from IKEA Malm dressers.

If you own an IKEA Malm dresser, click here for more information to learn about how you can contact IKEA for a wall-anchoring kit or full or partial refund. The recall applies to the 3-drawer, 4-drawer, 5-drawer, and 6-drawer models that were manufactured and sold through June 2016.

By Megan Cole and Claire Karlsson, National Center for Health Research

Are processed meats more dangerous than other red meats? Yes!

You have probably heard it many times already: don’t eat too much red meat or processed foods. But research shows processed red meats, like bacon, hot dogs, and salami are the biggest problem. Here’s why.

Red meats vs. Processed red meats

In October 2015, the International Agency for Research on Cancer of the World Health Organization concluded that processed meats cause cancer. Based upon a review of over 800 studies, 22 scientists from ten countries determined that processed meats can cause colorectal cancer and probably stomach cancer.95 Although people who eat more red meat are more likely to develop pancreatic and prostate cancer, nobody knows whether people who eat more red meat tend to have other poor health habits that are the real causes of these cancers, rather than the red meat itself.

Bacon, hot dogs, bologna, and other processed meats are now blamed for causing cancer, and they also increase your chances of developing heart disease and diabetes. A 2010 study led by Dr. Renata Micha from the Harvard School of Public Health analyzed 20 previous studies and concluded that while eating more red meat didn’t predict whether a person developed heart disease or diabetes, eating processed meat did. 96  A person who ate one portion (about one hot dog or two slices of deli meat) of processed meat every day was 42% more likely to develop coronary heart disease and 19% more likely to develop diabetes than if that person did not eat processed meat every day.  That risk was the same if the person ate 2 portions of processed meat every day instead of one, and doubled if the person ate 2 portions a day instead of none.  In other words, even if you like the taste or convenience of processed meat, eating less processed meat is always better for your health than eating more.

Why the difference?

When comparing red meats with processed meats, there are some key nutritional differences. While levels of saturated fats and cholesterol are usually similar in processed and unprocessed meats, processed meats generally have four times the amount of sodium and 50% more preservatives than red meats.97 Researchers suggest that these increased levels of sodium and preservatives may explain the increase in health risk. To determine if that is true, further research is needed. What is known, however, is that sodium increases blood pressure and preservatives have been shown to reduce tolerance to sugars. High blood pressure contributes to heart disease and reduced tolerance to sugars increases the risk of diabetes. Other studies have found that processed meats that have been cured, smoked and barbequed at high temperatures are more likely to cause colon cancer than other red meats.98 Cured meats like salami may pose particular risks for cancers because the nitrate and nitrite salts used in the curing process can promote cancer cell growth. Yet much more research is needed to clarify how processed meats can lead to cancer.

…but don’t pick up that steak so fast.

Does this mean that you are now free to eat all the red meat you want as long as it isn’t processed? Well, no.  Studies have shown that red meat raises the level of “bad cholesterol,” because it is high in saturated fat. Chicken and fish are much lower in saturated fat.  And there is some evidence that eating other red meat increases the risk of developing various cancers. Plus, eating less red meat may help reduce climate change, because cows emitsharmful greenhouse gases.99 Additionally, a study of 150,000 women, published in a major medical journal in 2016, found that eating red meat for protein instead of eating plants increases the chances of developing heart disease and dying at a younger age.100

What meats should I eat and what meats should I avoid?

As outlined by the US Department of Agriculture (USDA), consider the following when selecting meats for you or your family:
• Choose lean or low-fat meat and poultry. Avoid ground beef that is less than 80% lean (the leaner, the better), and choose skinless chicken.
• If you do buy processed meats, be sure to read the ingredients and Nutrition Facts label to avoid foods high in salt. Look for products labeled “low sodium,” “reduced sodium,” or “no salt added.” To be considered “healthy,” products must not have more than 600 mg of sodium per serving.
• Consider eating fish rich in omega-3 fatty acids, such as salmon, trout, and herring, or getting protein from other non-meat sources, such as beans, legumes, almonds, sunflower seeds, and egg whites.101

Is ALL processed meat worse than red meat?

All processed meats are not necessarily worse than all other red meats, as the “healthiness” of a meat depends upon the number of calories per serving as well as its sodium and fat content. For instance, lean deli meat may be healthier than a fatty unprocessed hamburger or steak. However, in general, bacon, sausage, hot dogs, pastrami, and many other processed meats are fattier, saltier, higher in calories, and contain more additives than unprocessed red meats such as beef, pork, and lamb. Lean and low-sodium varieties of processed meat are less unhealthy, but still not as healthy as most non-processed meats.

The bottom line

Foods that are higher in calories, saturated fat, and sodium tend to increase weight, fat, and blood pressure, which in turn, may lead to the development of heart disease and/or diabetes. So? Eat a balanced diet with plenty of whole grains, fruits, vegetables, low-fat dairy, and lean proteins. Enjoy red meat in moderation and remember: if you have to choose between a hot dog or a hamburger, the unprocessed meat of the hamburger is the safer bet when it comes to avoiding cancer, coronary heart disease and diabetes. (But also remember that the salt, ketchup, and pickles you add to that burger can make it even saltier than a hot dog).

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

This article was updated in 2016.

Margaret Dayhoff-Brannigan, PhD

Updated 2016

Lyme disease, deer tick

Lyme disease is a bacterial infection caused by ticks, primarily from an infected deer tick. Lyme disease infects approximately 300,000 people each year according to the Centers for Disease Control and Prevention (CDC). However, some experts believe that the number is much higher since fewer than half will develop the bull’s-eye rash that is the well-known early sign of the disease. Lyme disease is commonly misdiagnosed when there is no bull’s eye rash. When these patients go to their doctor for other symptoms, it can take months or even years for them to be diagnosed with Lyme disease.102

Part of the problem is the lack of an accurate test.  The most common test for Lyme disease has at least a 35% chance of missing the disease. Delaying the treatment for Lyme disease can make it much more difficult to treat. When Lyme is not treated quickly and effectively, patients can have long-term serious health problems that experts refer to as either post-Lyme syndrome, chronic Lyme disease, or multiple chronic infection disease syndrome (MCIDS).

COMMON SIGNS AND SYMPTOMS

  1. Bulls-eye rash (fewer than 50% of  infected people will get the rash)
  2. History of tick bite (not all patients recall a bite or think it likely they were exposed to a tick)
  3. Flu-like symptoms
  4. Fever
  5. Headache
  6. Extreme fatigue
  7. Pain in the neck, back, joint, bone or jaw.
  8. Chest pain
  9. Heart attack like symptoms
  10. Eye floaters
  11. Tingling or numbness in hands and/or feet
  12. Inability to fall asleep or stay asleep
  13. Night and/or day sweats

DIAGNOSIS AND TREATMENTS

The laboratory tests for Lyme disease have high levels of false negatives (which means that the test fails to recognize the disease) so often doctors will diagnose Lyme disease based on clinical symptoms, particularly if you have a bulls-eye rash or recent tick exposure. Early treatment increases your chances of a full recovery from Lyme disease, but the diagnostic tests are less accurate in early stages of the disease. Unfortunately, many insurance policies won’t pay for treatment without a test indicating the disease. The treatment for Lyme disease caught early is a several week course of antibiotics. However, since the test is often inaccurate in the early stages, many patients are not diagnosed early enough to benefit from standard antibiotic treatment.

Antibiotics are the first line of treatment, but do not always cure a Lyme disease infection or prevent later health problems. Symptoms such as fatigue or joint pain may return months after the initial infection has been treated. It is not clear whether these symptoms are caused by damage to your joints and immune system during the original infection or if it is due to ongoing infection. A long-term course of antibiotics might be suggested to treat the illness, however the effectiveness is controversial so insurance companies may refuse to pay for this treatment. It is unknown how many patients with long-term health problems due to Lyme disease with improve with time.

RELATED INFECTIONS

There are other diseases carried by ticks that can also infect humans, such as Babesia or Bartonella. Lyme disease patients often are infected with more than one of these infections at the same time. If you suffer from Lyme disease on a long-term basis, it is important to talk to your doctor about the possibility that you also have one or more of these other infections.

CHRONIC LYME DISEASE

When Lyme disease goes undiagnosed, it can be much more difficult to treat effectively.  If the disease is not treated immediately and effectively, that can lead to a chronic infection, characterized by headaches, debilitating joint pain, and severe fatigue.  However, the diagnosis of chronic Lyme disease is controversial because it can be very difficult to prove Lyme disease caused the symptoms.103

PREVENTION

There are many steps you can take to reduce your risk for Lyme disease. Lyme disease has been diagnosed for individuals who spent time outdoors anywhere in the U.S., although the risks may still be higher in the Northeast from Virginia to Maine, or the West coast. To reduce your chances of getting Lyme disease when you spend time in or near wooded areas, make sure you wear light colored clothing that covers your skin and use insect repellent. Be sure to check yourself and any loved ones for ticks as soon as possible after you get inside. The chances of developing Lyme are highest if the tick is attached for at least 24-48 hours in order to spread the disease, so if you see them early try to remove them before they can infect you. The best way to avoid Lyme disease is prevention.

Research published in 2014 confirms that individuals who were never exposed to a tick can develop Lyme disease that is sexually transmitted.104 So if you or a sexual partner has Lyme disease, be sure to use protection until the infection has been cured.

More information about signs and symptoms can be found here.

May 27, 2016

Open Letter to Dr. Margaret Chan, Director-General, WHO (Copied to the International Olympic Committee):

We are writing to express our concern about the upcoming Olympic and Paralympic Games in Rio de Janeiro. WHO’s declaration of Zika as a “Public Health Emergency of International Concern,” coupled with new scientific findings that underscore the seriousness of that problem, call for the Rio 2016 Games to be postponed and/or moved to another location—but not cancelled—in the name of public health.

We make this call despite the widespread fatalism that the Rio 2016 Games are inevitable or “too big to fail”. History teaches this is wrong: the 1916, 1940, and 1944 Olympic Games were not just postponed, but cancelled, and other sporting events were moved because of disease, as Major League Baseball did for Zika, and the Africa Cup of Nations did for Ebola.

Currently, many athletes, delegations, and journalists are struggling with the decision of whether to participate in the Rio 2016 Games. We agree with the U.S. Centers for Disease Control recommendation that workers should “Consider delaying travel to areas with active Zika virus transmission”.1 If that advice were followed uniformly, no athlete would have to choose between risking disease and participating in a competition that many have trained for their whole lives.

But our greater concern is for global health. The Brazilian strain of Zika virus harms health in ways that science has not observed before. An unnecessary risk is posed when 500,000 foreign tourists from all countries attend the Games, potentially acquire that strain, and return home to places where it can become endemic. Should that happen to poor, as-yet unaffected places (e.g., most of South Asia and Africa) the suffering can be great. It is unethical to run the risk, just for Games that could proceed anyway, if postponed and/or moved.

In our view, several new scientific findings require WHO to reconsider its advice on the 2016 Olympic and Paralympic Games. For example:

1. That the Brazilian viral strain causes microcephaly2 and probably Guillain-Barré syndrome3. Further, because human4, animal5 and in vitro studies6 demonstrate that the virus is neurotrophic and causes cell death, it is biologically plausible that there are other as yet undiscovered neurological injuries, as exist for similar viruses (e.g. dengue).7

2. That while Zika’s risk to any single individual is low, the risk to a population is undeniably high. Currently, Brazil’s government reports 120,000 probable Zika cases,8 and 1,300 confirmed cases of microcephaly (with another 3,300 under investigation)9, which is above the historical level of microcephaly.10

3. That Rio de Janeiro is highly affected by Zika. Brazil’s government reports Rio de Janeiro state has the second-highest number of probable Zika cases in the country (32,000) and the fourth-highest incidence rate (195 per 100,000), demonstrating active transmission.8

4. That despite Rio’s new mosquito-killing program, the transmission of mosquito-borne disease has gone up rather than down. While Zika is a new epidemic and lacks historical data, using dengue fever as a proxy, cases in Rio from January thru April 2016 are up 320% and 1150% over the same periods in 2015 and 2014, respectively.11 In the specific neighborhood of the Olympic Park (Barra da Tijuca) there have been more dengue cases in just the first quarter of 2016 than in all of 2015.11

5. That Rio’s health system is so severely weakened as to make a last-minute push against Zika impossible. Recently Rio’s state government declared a health sector emergency,12 and Rio’s city government cut funding against mosquito-borne disease by 20%.13 While the virus is the infectious agent of Zika, its real cause is Rio’s poor social conditions and sanitation—factors that lack a quick fix, and that are not helped when shrinking health resources are diverted to the Games.

6. That it is possible to eradicate the Aedes aegypti mosquito, which transmits Zika, from Rio. Actually that mosquito was totally eradicated from Brazil in the 1950s, but came back after control efforts lapsed.14 Thus holding the Games, in the presence of Zika-carrying mosquitoes, is a choice and not necessary.

7. That one cannot count on nature for defence. While lower mosquito activity during Rio’s winter months reduces the individual risk to travelers of infection, that is partly offset when travelers who became infected return home during the northern hemisphere’s summer months and peak mosquito activity, which increases the public health risk that local mosquitos acquire and spread the virus—meaning that both seasons are relevant to the epidemic’s course.15 Also, infection can spread through blood donations and transfusions, particularly in poor countries that lack screening for Zika.16

In sum, the evidence shows: (i) that Brazil’s Zika virus strain has more serious medical consequences than previously known, (ii) that Rio de Janeiro is one of the most affected parts of Brazil, and (iii) that Rio’s mosquito-killing efforts are not meeting expectations, but rather mosquito-borne disease is up this year. It is therefore imperative that WHO conduct a fresh, evidence-based assessment of Zika and the Games, and its recommendations for travelers.

Because Zika is a new emergency, its many uncertainties—of travel flows during the Games, of epidemiology, and of entomology—currently make it impossible for mathematical models to predict the epidemic’s course accurately. Therefore, for now, any decision about Zika and the Games has to be more qualitative than quantitative. If one considers the following options:

(a) Holding the Games in Rio in 2016 as scheduled;
(b) Holding the Games in Rio at a later date after Zika is controlled, and; (c) Holding the Games at Zika-free sites having Olympic-standard facilities.

It is indisputable that option (a) of holding the Games as scheduled has a greater risk of accelerating the spread of the Brazilian viral strain than the alternatives. Postponing and/or moving the Games also mitigates other risks brought on by historic turbulence in Brazil’s economy, governance, and society at large—which are not isolated problems, but context that makes the Zika problem all but impossible to solve with the Games fast approaching.

We are concerned that WHO is rejecting these alternatives because of a conflict of interest. Specifically, WHO entered into an official partnership with the International Olympic Committee, in a Memorandum of Understanding that remains secret.17 There is no good reason for WHO not to disclose this Memorandum of Understanding, as is standard practice for conflicts of interest. Not doing so casts doubt on WHO’s neutrality, for reasons described further in the Appendix.

WHO must revisit the question of Zika and postponing and/or moving the Games. We recommend that WHO convene an independent group to advise it and the IOC in a transparent, evidence-based process in which science, public health, and the spirit of sport come first. Given the public health and ethical consequences, not doing so is irresponsible.

Signatories (including 225 others): Dr. Diana Zuckerman, President, National Center for Health Research, Washington DC, USA

Statement of Dr. Stephanie Fox-Rawlings

Consumer Product Safety Commission

June 15, 2016

The National Center for Health Research is a nonprofit research center staffed by scientists, medical professionals, and health experts who analyze and review research on a range of health issues. Thank you for the opportunity to share our views concerning the Consumer Product Safety Commission’s (CPSC) priorities for fiscal year 2017 and 2018. We respect the essential role of the CPSC, as well as the challenges you face in selecting the most important priorities

Phthalates and flame retardants need to be among your top priorities because they are in all our homes and they migrate from products into the our daily environment. Multiple phthalate metabolites and flame retardants are detectable in nearly all people in the U.S.105 and scientists agree that their impact on health can be dangerous and long-lasting.

Additional bans on phthalates in children’s toys and care products

We applaud the current permanent and temporary bans on six phthalates in children’s toys and child care articles.106 However, these bans need to be expanded. The rule “Prohibition of Children’s Toys and Child Care Articles Containing Specified Phthalates” proposed in spring 2015 following the Chronic Hazard Advisory Panel (CHAP) would provide essential additional protections for children.107 108

We support the permanent bans on four additional phthalates (DIBP, DPENP, DHEXP, and DCHP) and making permanent the interim ban on DINP.3 However, the CHAP report also recommended an intern ban on DIOP, which should also be included in the rule. We strongly disagree with the proposal to lift the interim bans on DNOP and DIDP. While they may not be associated with antiandrogenicity, they are associated with organ toxicity and altered development.

The CHAP report also recommended additional studies on three other phthalates (DMP, DPHP, and DEP) and six phthalate alternatives.4 The final rule should include a timeline for the completion of these studies.

In summary, we strongly urge the CPSC to finalize the proposed rule on phthalates in children’s toys and child care articles, including consideration of our safety concerns.

It is also important for CPSC to expand its work on phthalates to include safeguards for older children. There is increasing evidence of the impact of these chemicals on early puberty,which itself is associated with drug abuse, sexual exploitation, and suicide.109

Bans on flame retardants

The CPSC has the responsibility and ability to protect consumers from toxic flame retardants under the Federal Hazardous Substances Act.

We agree with others groups commenting today that the CPSC should propose and finalize regulations that would ban additive, non-polymeric organohalogen flame retardants in four categories of household products as proposed in Petition No. HP 15-1.110 Like phthalates, these chemicals move from products to our daily environment and from there into consumers’ bodies where they can cause irreparable harm. All of the organohalogen flame retardants studied have been associated with chronic health effects.

The most well-studied organohalogen flame retardants are the polybrominated diphenyl ethers (PBDEs), which have been phased out in part due to their effects on human health.111 The alternatives in the same class are proving to have similar problems. These alternatives found in a large percentage of people tested in various communities.112 They have been linked to cancer, reproductive problems, neurotoxicity, developmental toxicity, endocrine disruption, and behavioral changes in models and/or humans.6

We strongly urge the CPSC to develop and finalize a ban on these chemicals in the proposed residential products to protect consumers from their toxic effects.

In conclusion, we urge the CPSC to prioritize the research and rulemaking to limit exposure of consumers, and especially children, from the phthalates and flame retardants that have been found to have negatively impact health and development.

Thank you for your time and consideration of our views.