By Laurén A Doamekpor PhD, MPH and Diana Zuckerman PhD

Letter to the Editor, Cancer Epidemiology, July 2014

A study published in Cancer Epidemiology in April 2014 showed that between 1990 and 2009, the disparity in breast cancer mortality between Black and White women has widened in our country’s most populated states. Like most cancer experts, the authors (Ms. Hunt and her colleagues) conclude that although there have been advances in breast cancer screening, prevention and treatment, these advances have not been equally available to Black and White patients.1

In July 2014, Dr. Laurén Doamekpor and Dr. Diana Zuckerman of the National Center for Health Research published a response in the same journal, agreeing that access is a problem but pointing out that a lack of racial and ethnic diversity in clinical trials may exacerbate this disparity.2 Scientific knowledge gained from clinical trials on prevention and treatment is important; unfortunately, patients of color continue to be underrepresented in clinical trials that are submitted to the Food and Drug Administration (FDA) when a company applies for FDA approval.3  Since relatively few people of color are included in studies submitted to the FDA, and even fewer studies analyze and report safety and efficacy separately by race or ethnicity, it is unlikely that scientists will discover treatments that are safe and effective for minority patients if those patients metabolize certain drugs differently or tend to have different types of cancer, such as triple negative breast cancer. This contributes to racial disparities when treatments are not adequately tested for  safety or efficacy in these populations.

 By: Sara Exler

 

What is blood pressure?

Blood pressure is the force of blood against the arteries as the heart beats. Two numbers measure blood pressure: systolic (the pressure when the heart beats) and diastolic (the pressure when the heart rests between beats). Blood pressure is usually written as systolic over diastolic like this: 120/80 mmHg.

High blood pressure (also called hypertension) affects approximately one in three adults in the United States, and fewer than half of all those people have their blood pressure under control. 4 High blood pressure is the most commonly diagnosed health problem in this country; it can lead to heart attack, stroke, kidney failure, and death if not detected early and appropriately treated.5

What is normal blood pressure? For adults 59 and under, it’s less than 120/80 mmHg. Between 120/80 and 139/89 mmHg is considered pre-hypertension, meaning you are on your way to developing high blood pressure. Hypertension or high blood pressure is diagnosed when a person under 59 regularly has blood pressure measurements of 140/90 mmHg and higher.6

New guidelines issued in 2013 by a panel of 17 experts (the Joint National Committee) determined that since people over 60 regularly have higher blood pressure, the diagnosis of hypertension in that age group and the recommendation of getting treatment should be reserved for blood pressure that is above 150/90 mmHg.

Normal Pre Hypertension Hypertension Hypertension for those aged 60 and above based on new JNC8 7
Under 120/80 mmHg 120/80 mmHg – 139/89 mmHg Over 140/90 mmHg Over 150/90 mmHg

What causes high blood pressure?

High blood pressure can be caused by family history, behaviors, and age. Individuals with a family history of high blood pressure and those with diabetes are more likely to have high blood pressure. Race is also a factor; African Americans are more likely than whites to have high blood pressure. Aging increases the likelihood of high blood pressure. Lack of physical activity, an unhealthy diet, being overweight, alcohol use, stress, and smoking also contribute to your chances of having high blood pressure. 8

What you should do to keep your blood pressure in the healthy range?

  • Get Enough Sleep
  • Reduce Stress
  • Eat more fruits, vegetables, and whole grains
  • Exercise
  • Drink less alcohol
  • Don’t Smoke

 You can reduce your blood pressure by changing some of your habits. Improvements include maintaining a healthy diet, quitting smoking, limiting alcohol intake, exercising regularly, and controlling levels of stress. 9 Stress reduction not only helps reduce high blood pressure, but also helps alleviate many other health issues. Even sunshine can help; one recent study found that for those with mild hypertension, 30 minutes per day in the sun reduced blood pressure a small but significant amount, and overall blood pressure rates improved in the summer compared to the winter.10

Exercise

 Regular exercise can significantly help lower your blood pressure. Different studies have shown that at least 30 minutes of moderate exercise 3 or 4 times a week can help lower blood pressure 4-9mmHg.11 Getting exercise doesn’t have to mean spending all day at the gym; parking further away and walking to the store, taking the stairs instead of the elevators, taking a brisk walk around the neighborhood, and joining local fitness groups are all ways to start increasing your activity levels. Don’t try to do too much too suddenly; work slowly and gradually increase your exercise intensity. Finding a particular type of movement you enjoy will help you better stay on track with a regular exercise routine. Always talk with your doctor before starting a new exercise routine.

Salt

 Eating less salt to reduce your high blood pressure mainly works in combination with other lifestyle changes, such as a better diet and more exercise.12 Less salt has been found especially effective in lowering high blood pressure in African Americans, people middle aged and older, and people who are overweight.13  The previously accepted recommended sodium intake was 1500mg per day (less than half a teaspoon of salt) for those with high blood pressure.14 Newer studies, however, have shown that 2300 mg is a good sodium goal. 15 Processed foods are very high in salt, making up more than 75% of our salt intake.  Most salt in your diet may be from foods you do not think of as salty, such as bread, cold cuts and processed meats, pizza, soup, cheese, pasta dishes, chicken dishes, sauces like ketchup and soy sauce, and frozen food. 16  The easiest way to reduce your salt consumption is to cook your own food from fresh ingredients, because even if you add salt it is likely to be much less than in processed foods. In fact, studies show that when people are served low sodium foods but are allowed to add salt from a salt shaker, most people only add back 20% of the sodium removed from their food.17 To lower your salt intake without sacrificing taste, consider experimenting with different spices and seasonings like garlic, lemon juice, ginger, cilantro, pepper, rosemary, or sage.

Monitoring Blood Pressure at Home

If your doctorrecommends you monitor your blood pressure at home, there are a few good tips you should follow. Buy a good quality digital home blood pressure monitor. Make sure the cuff is the right size; different size cuffs are available to buy and the wrong size cuff will give inaccurate results. Take your home blood pressure monitor to the doctor and have it checked to make sure it is accurate, and to make sure you are using it correctly. Do not take your blood pressure for 30 minutes after eating, exercising, smoking, or drinking caffeine. 18 Monitoring your blood pressure at home is a good way to track any changes made by either better habits, medicine, or both. A 2013 study found that people who monitored their blood pressure at home were significantly more likely to have their blood pressure under control after six months then those who did not. 19

 Medicines for treating high blood pressure

 In addition to changing the recommendations for the diagnosis of high blood pressures for people over 60, the Joint National Committee also simplified theirrecommendations for different types of medications. If you are diagnosed with high blood pressure that you can’t control with diet and exercise, you will likely be prescribed one or a combination of the medicines below. African Americans shouldn’t take ACE inhibitors or ARBS. 20

Blood Pressure Medications Description 21
thiazide-type diuretic Diuretics are also known as “water pills” because they help flush excess water and sodium from the body.  Hydrochlorothiazide (HCTZ) is a popular example.
calcium channel blocker (CCB) A CCB helps prevent calcium from entering the muscle cells in the arteries. This causes blood vessels to relax and blood pressure lowers.
angiotensin-converting enzymeinhibitor (ACEI) Angiotensin converting enzyme (ACE) inhibitors prevent the formation of angiotensin II, which narrows blood vessels. The blood vessels relax and it lowers blood pressure.  Lisinopril is a popular ACE inhibitor.
angiotensin receptor blocker (ARB) ARBS block blood vessels from angiotensin II. As a result, the vessels become wider and blood pressure goes down.

 Bottom Line

High blood pressure is a common problem for American adults. If you have high blood pressure (hypertension), it’s important to establish a treatment plan with your doctor that includes not only medicine, but also healthy lifestyle choices such as lowering salt intake, a healthier diet, quitting smoking, exercising, and stress control.   If those strategies don’t work, you may need medication as well, but remember that all blood pressure medications have side effects.

 

 

By Nell Greenfieldboyce, NPR

July 21, 2014

041019-N-5821P-019

No one likes it when a new drug in people’s medicine cabinets turns out to have problems — just remember the Vioxx debacle a decade ago, when the painkiller was removed from the market over concerns that it increased the risk of heart attack and stroke.

To do a better job of spotting unforeseen risks and side effects, the Food and Drug Administration is trying something new — and there’s a decent chance that it involves your medical records.

It’s called Mini-Sentinel, and it’s a $116 million government project to actively go out and look for adverse events linked to marketed drugs. This pilot program is able to mine huge databases of medical records for signs that drugs may be linked to problems.

The usual system for monitoring the safety of marketed drugs has real shortcomings. It largely relies on voluntary reports from doctors, pharmacists, and just plain folks who took a drug and got a bad outcome.

“We get about a million reports a year that way,” says Janet Woodcock, the director of the FDA’s Center for Drug Evaluation and Research. “But those are random. They are whatever people choose to send us.”

Figuring out from those reports if a drug is really to blame for the symptom isn’t easy. And if the side effect is something common, such as a rash or a stroke, there might not even be any reports because doctors might not connect a patient’s symptoms to the drug.

“We need a rapid way to find out what’s happening with drugs, especially safety of drugs, after they’re approved and on the market,” says Woodcock.

That’s why the FDA is turning to workers at the operations center of Mini-Sentinel, which is located in a brick office building near Fenway Park in Boston.

Here, people in cubicles can send out queries to 18 data partners that include health plans and insurance companies. Their health records include nearly 180 million Americans.

If you have insurance through a private health plan, the chances are “pretty good” that your data may have been used in one of these studies, says Dr. Richard Platt, the principal investigator for Mini-Sentinel and a professor at Harvard Medical School’s Department of Population Medicine.

Almost all of that data comes from billing records — not what your doctor has scribbled in your chart, but rather the codes for any diagnostic tests and procedures you undergo. (These codes are on your medical bill.) Platt says the data partnership has been carefully set up to preserve patient privacy.

His own records have probably been part of a study, says Platt, but he has no way of knowing that. “We’ve built the system so the analyst here would have no way to say, ‘Let’s see if Dr. Platt’s data is in there,’ ” he says. “The system is built so that that is an impossible question to ask.”

It’s taken five years for Platt’s team to build Mini-Sentinel from scratch and make it a routine part of FDA’s work. “We’re now doing hundreds of queries a year,” he says.

For example, they ran a search when the FDA got some troubling reports tentatively linking intestinal problems to a blood pressure drug called olmesartan.

“They had noticed that patients who had received this drug were having these complications that they thought might be related to the drug,” recalls Platt.

At first, Mini-Sentinel found no connection. Then the FDA asked for a second search, this time focusing on people who took the drug for long periods. They found a link, Platt says. As a result, the FDA added a warning to the drug’s label.

“It was possible to provide an answer that otherwise would frankly just be unavailable to them,” says Platt.

Mini-Sentinel has been an experiment to see what’s possible, but its contract ends in September and the FDA will be deciding what to do next.

Almost everyone agrees that the ability to sift through huge amounts of patient data is the way of the future — but not everyone believes that we really know how to best do that sifting yet.

“I think it’s a good and important step that the FDA is moving in this direction,” says Thomas Moore, a senior scientist at the nonprofit Institute for Safe Medication Practices. “The problem is, I think, they have underestimated how far they have to go.”

Billing data were never meant to be used this way, says Moore, who questions how well they can reveal side effects from drugs. He thinks “the biggest danger is that people will get a false reassurance about safety.”

He points to a controversy over a new blood-thinning medicine called dabigatran. Reports had come in about serious bleeding episodes, says Moore, but “the FDA published an article in a leading medical journal, basically discounting all that — saying that, using Mini-Sentinel, they had seen no unusual risk for this drug.”

The agency is now taking another, more nuanced look at dabigatran, after critics questioned how well the FDA had designed the original Mini-Sentinel study.

People at the FDA are aware that when doing this type of research with big data, the way you set up the question can affect the answer.

“Things may change depending on what claims data set you use, or how you run your definitions, how you set up your parameters and so forth,” says Woodcock. “And there’s no doubt these studies are vulnerable to all these changes.”

She says that’s why the FDA is also involved in another effort to explore all these potential effects. This effort is led by The Reagan-Udall Foundation for the FDA, a foundation set up by Congress. Right now its major project is research on how to use large databases to study the safety of medicines on the market.

“This is a new science, and much work needs to be done to develop and continue to improve the methods behind this,” says Troy McCall, who is managing the project for the Reagan-Udall Foundation.

The foundation does get some money from the FDA, but supports its research with other funds. And so far, all the money for this particular project has come from pharmaceutical companies like Merck, Pfizer, Novartis, Johnson & Johnson, GlaxoSmithKline, AstraZeneca and Eli Lilly.

McCall says he doesn’t think it’s seen as an “‘industry project,” because the work is so important. “Clearly at the end of the day this is all to benefit patients and ensure that drugs that are on the market are safe,” he says, adding that patients also benefit when safe drugs are not unnecessarily removed from the market.

Drug companies do have a big interest in what stays on or off the market. But Woodcock sees no problem with industry funding the development of the scientific methods that will then be used to help make regulatory decisions.

“It would be very difficult to develop a method that was going to favor your drug when you’re developing a general method, I think,” Woodcock says. “But what I would say is, OK, who is going to develop these methods? We need them developed.”

Officials at the Reagan-Udall Foundation say it operates transparently and has different stakeholders represented on its governing boards. But one board member who represents consumers says she finds the lack of independent funding troubling.

“I think that creates an appearance of a conflict of interest and potentially a real conflict of interest,” says Diana Zuckerman, president of the nonprofit National Center for Health Research. “If all the money is coming from the pharmaceutical companies whose livelihoods are going to be affected by what the project finds, I just think that’s an untenable situation.”

Zuckerman says she worries that industry funding might influence the methods that eventually get used for finding signals in big data that drugs are unsafe — and that could potentially limit what this new approach will reveal about the medicines Americans take every day.

Listen to the interview here!

July 17, 2014

Lawmakers appeared to respond to concerns that the House Energy & Commerce Committee is receiving a one-sided, industry-backed patient perspective as part of its 21st Century Cures Initiative by soliciting comments July 11 from other groups and calling for a balance between the push for more expedited approvals and public health safeguards.

The National Center for Health Research recently criticized the committee for primarily working with patient groups focused on expediting approvals and excluding others that seek establishment of better safety and efficacy standards (see FDA Week, July 11).

During a Friday Energy & Commerce health subcommittee hearing, Rep. Michael Burgess (R-TX) suggested interested groups contact members about the 21st Century Cures Initiative. “I know there are many people who may be watching and thinking I would like to be involved with committee staff, so that’s the way to do it,” he said.

Rep. Frank Pallone (D-NJ) also made it clear that he supports expediting patient access to innovative treatments but is also concerned about the safety and efficacy of medical products. Lawmakers should be cognizant of the risks that come with speed, he said.

Further, in his testimony Cystic Fibrosis Foundation President and CEO Robert Beall pressed for a balance between efforts to advance the development of innovative treatments and those to ensure patient safeguards.

“As policy options are considered, it is important to find a balance between accelerating drug discovery and innovation, while also ensuring that patients have safe, effective treatments,” he said. “There must be clear communication about the benefits of current therapies so that patients can make informed decisions…as new therapies are developed.”

Rep. Jan Schakowsky also echoed women’s health advocates’ concerns about the possibility that the smaller clinical trials being pushed as part of expedited approval could work against efforts to collect more clinical trial data for women and demographic subgroups. NCHR has predicted that as clinical trial populations grow smaller they will also become less diverse.

Leonard Lichtenfeld, deputy chief medical officer of the American Cancer Society, agreed that there was a risk of excluding important patient populations but advised against using that possibility to argue against the expedition of innovative product approvals. There is the other side of the coin, he said, noting that some treatments can elicit a response from the vast majority of patients.

“When you suddenly see moments like that no one would want to hold back,” he said. “Develop the data, yes, but don’t hold back the opportunity.”

Still, the National Women’s Health Network said it remains concerned about what it sees as the committee’s focus on expediting product approvals as the best way to speed patient access to innovative treatments. “The idea that you can get treatments more quickly to patients by only accelerating the FDA process is ridiculous,” said Kate Ryan, NWHN’s senior program coordinator.

She said she would like to see the committee put more emphasis on the need to invest in diversifying scientific research to provide patients with more options. But she noted the group has talked with the office Rep. Diana DeGette (D-CO) — DeGette leads the committee’s innovation initiative alongside Chair Fred Upton (R-MI) — and feels there are lawmakers that share those priorities and concerns about women’s health.

 

By Diana Zuckerman, Ph.D.

My friend Barry just got out of the hospital, four months after having an almost deadly reaction to a prescription medication. Talking to him about his experience has made me realize how overwhelming it is for most people to figure out what medical treatments are best for them, which doctors to trust, and how to recover from a serious illness.

Thousands of people contact our organization with medical questions, and most of them–no matter how smart they are–contact us because they don’t know how to get the medical information they need to stay well or to recover from illness. Going online can be very helpful, very confusing, or even dangerous.

Here are some simple tips to help:

Don’t wait.

My friend Barry needs an outstanding physician to help him recover from a rare, life-threatening disease that was impossible to prevent. But had Barry sought the best medical attention immediately after experiencing serious symptoms rather than toughing it out at home, he probably could have avoided the hell he’s been through. He asks me to tell you not to wait the way he did.

Be thoughtful about treatment decisions.

Be cautious about taking treatment advice from friends or relatives who’ve had your illness, or know someone who’s had it. There’s no guarantee that they got the best treatment. And medical advice can change weekly; they may have had a different form of the illness than you do; or they may have other health problems that influenced their treatment options but wouldn’t be relevant to you.

Avoid the Food and Drug Administration’s (FDA’s) “breakthrough drugs.” This designation sounds as if the drug is the best new treatment, but that’s not what it means. Weirdly, FDA’s designation refers to new drugs that seem potentially promising but are not yet proven to be safe or effective. A more accurate term would be “new but unproven” drugs. It often takes 10 years or more to know exactly what the risks and benefits of a new drug are. “Breakthrough drugs” are also usually extremely expensive, despite the weak evidence. Who would want a new, unproven drug if proven alternative treatments are available?

If you start having symptoms within days or weeks of taking a new medication, find out if they might be related to your medication. Go online and search the name of the medication and the word “risks” or the term “side effects.” Find the website for the company that makes the drug or go to drugs.com. Although the company will praise its medication, by law it must list all the possible risks or side effects found in research studies. That’s why a company’s site (or drugs.com, which lists the same risks and side effects) is more informative than many other websites.

Research doctors and medical facilities.

I live in Washington, DC, but suddenly, the local hospital has become a Johns Hopkins facility (even though the real Hopkins is in Baltimore), and there is a Cleveland Clinic medical center nearby (but Cleveland is more than 300 miles away). Mayo Clinics are all over the place too. Don’t be fooled by those famous brand names. The actual Johns Hopkins, Cleveland Clinic, and Mayo Clinic attract some of the best doctors for many reasons. But your local medical center usually won’t have the same ability to attract the best, even when it’s bought an affiliation with a famous name.

If you need surgery, find a board-certified surgeon who does many of those same surgeries every year. Practice really does make perfect, relatively speaking. Of course, also look to see where and when they were trained, and look online for additional information about a surgeon. If you have a choice, choose someone old enough to be experienced but still young enough to be at the top of his or her game.

Don’t choose a doctor who doesn’t ask you lots of questions about your health history and symptoms or doesn’t listen to what you say. On the other hand, don’t expect a doctor to always agree with you. For example, too many doctors prescribe antibiotics for colds or the flu just because patients demand them. But antibiotics don’t work for colds or flus, and they can have side effects that are harmful. You want a doctor who knows more than you do but listens carefully. And if the doctor tells you that you don’t need medication, take that as a good sign.

Stick to reputable websites.

When looking for medical information online, I recommend going to the main website of the National Institutes of Health at nih.gov, click “health information” and then write the name of the disease you want to learn about in the search box. For cancer information, we recommend cancer.gov. The “.gov” tells you that the website information is based on research.

Avoid the websites that are selling something–whether it’s a hospital, a drug company, or nonprofit disease support organization (because some are so much better than others, and many depend on pharmaceutical companies for their funding). This can be tricky though, because many sites are selling something, though it isn’t always obvious.

Here are a few pointers when it comes to medical websites:

  • Avoid websites with “sponsored links,” which is just another term for ads.
  • Avoid websites with “popular responses,” such as Yahoo Answers. Medical advice from nonprofessionals can be helpful or harmful. The bottom line is there are better sources of information than advice from strangers who may or may not know what they are talking about.
  • The first websites to come up in Google, Yahoo, or other search engines are probably not the best. Most include sponsored links at the top and the side–and those are paid ads. Even if they’re not sponsored links, top websites on these search engines often pay for placement.
  • Wikipedia is a mixed blessing because anyone can edit it–a Nobel laureate or the preteen next door. Some medical information on Wikipedia is great; some is awful. Don’t play Russian roulette with your health.
  • Often, websites that seem to be created by patients or caring doctors aren’t. Some advertise products that are unsafe or ineffective, and others will delete comments of patients expressing concerns about medical products. The physicians featured on the site often pay for the privilege–it’s a form of advertising, and I’m willing to bet that the best physicians in the country are not the ones paying to be included.

If you have any questions about any of this or for free information with no ads, feel free to contact us at info@center4research.org

Testimony of Dr. Anna E. Mazzucco

Obstetrics and Gynecology Devices Panel of the Medical Devices Advisory Committee

 

July 11, 2014

Thank you for the opportunity to speak today on behalf of the Cancer Prevention and Treatment Fund of the National Center for Health Research. My name is Dr. Anna Mazzucco, and after completing my Ph.D. in Cell and Developmental Biology from Harvard Medical School I conducted research at the National Cancer Institute. I speak from those perspectives today.

Our nonprofit organization conducts research, scrutinizes data in the research literature, and then explains the evidence of risks and benefits to patients and providers.  Our president is on the Board of Directors of the Alliance for a Stronger FDA, which is a nonprofit dedicated to increasing the resources that the FDA needs to do its job.  Our organization does not accept funding from medical device companies, and therefore I have no conflicts of interest.

About 600,000 hysterectomies are performed annually in the United States, according to the Centers for Disease Control and Prevention, and approximately 65,000 myomectomies.  Of the hystectomies alone, the FDA estimates that 50,000 to 150,000 use power morcellation.  The FDA also estimates that 1 in 350 women undergoing hysterectomy or myomectomy for the treatment of fibroids has unsuspected uterine sarcoma which could be spread and worsened if a power morcellator is used.  Based on these numbers, as many as 400 women could have undiagnosed malignancy spread each year from hystectomy alone  – when you add myomectomies, it is probably much higher.  We agree with the FDA that there is currently no reliable method to distinguish between uterine fibroids and sarcoma before surgery.  Training physicians is, unfortunately, not the answer.

The estimate of one in 350 women undergoing surgery having an unsuspected uterine cancer is based on recent studies and is much higher than the 1-in-10,000 chance of undiagnosed cancer typically quoted to patients.  The FDA has also estimated that undiagnosed cancer will be spread or worsened by morcellation in 25-65% of cases.  The estimated 5-year survival is 60% for patients with stage I disease, compared with 22% for those with stage III and 15% for those with stage IV.

Minimally invasive surgery can offer many advantages to patients, but as you have heard at this meeting, the mortality benefits of such procedures are unclear. In contrast, it is absolutely certain that malignancy spread by morcellation can be life-threatening.  In light of these findings, we agree with the FDA’s statement in their safety communication that power morcellators should no longer be used during removal of uterine fibroids. 

The question is whether this warning from the FDA is enough to save lives?  Or, is this new evidence sufficient to alter the classification and labeling of these devices?

Power morcellators were originally approved as class II moderate -risk devices under the 510(k) process, which does not require clinical trials prior to allowing the device on the market.  It also does not require inspections to make sure the device is made and working as designed — such inspections are required for all prescription drugs.  Since morecellators were not studied in clinical trials, the risk of undiagnosed sarcoma spreading was not detected prior to clearance through the 510(K) pathway.  As a result, patients were irreparably harmed.  We’ve heard some of those tragic stories at this meeting.

Class III devices are defined as those which pose a significant risk of illness or injury, and require clinical testing for safety and efficacy.  Clearly, power morcellators meet this definition and should be classified as Class III devices.  Therefore, they should undergo clinical studies before any more patients are harmed.  Non-clinical performance testing studies are simply not sufficient to address these safety concerns.   If an adequate number of patients had been studied in clinical trials, we would have known years ago that morcellators could cause a fatal spread of uterine cancer.

I think everyone on this panel agrees that more research is needed. Clinical studies must evaluate risk mitigation strategies, such as use of a companion containment bag. However, as the FDA briefing material cites, there are adverse events associated with current specimen bags.  For this reason, bags need to be specifically designed for use with power morcellators specifically for uterine fibroids.  Surgical techniques must also be refined for use with these bags.

Clinical trials are also needed to improve the accuracy of patients’ diagnostic outcomes when morcellators are used.

After these studies are completed, the FDA should consider whether and under what circumstances power morcellators can be used for uterine fibroids.   On the basis of research, black box warnings must inform physicians and patients of the risk of spreading malignancy and the required risk mitigation strategies.  This label should also include the warning that these devices should never be used in patients with suspected malignancy.  Training and certification should also be required before physicians can use power morcellators to ensure that these risk mitigation techniques have been mastered.

As the FDA has stated in its summary for today’s meeting, the current voluntary reporting system for medical devices is underused, and thus underreports adverse events for all medical devices, including power morcellators. Unfortunately, surgeons and other medical personnel are not reporting these incidents to the FDA, and their reporting is voluntary, so they don’t have to.  On the other hand, the hospitals where these incidents occur are required to report them to the device companies, and the device companies are required to report them to the FDA.  For some reason, those reports were not being made either.  As a result, many more patients died before the risks of morcellation became known — primarily as the result of a physician whose life was put at risk when a morcellator used for a uterine fibroid resulted in Stage 4 uterine cancer.

We agree with the American Congress of Obstetricians and Gynecologists that a patient registry should be created to follow patients whose fibroids were previously removed with the assistance of power morcellation to more scientifically monitor their health outcomes. But, that is not enough.  And the current FDA warnings are not enough.

We need higher standards to ensure that morcellation devices are safe and effective, and that require clinical trials with sufficient numbers of patients to determine the risks of rare but fatal outcomes.

July 8, 2014

Good morning. My name is Dr. Laurén Doamekpor, and I am a senior fellow speaking on behalf of the National Center for Health Research. Thank you for the opportunity to speak today at this important meeting.

An important part of our work as a non-profit think tank is to assess scientific and medical data and provide objective health information to patients, providers and policy makers. Over the years, we see the same recurring problem. Racial and ethnic minorities are rarely included in sufficient numbers in clinical trials used as the basis of FDA approval of drugs and medical devices.  And yet, these are the medical products that all patients — including millions of people of color — rely on.

As you know, scientific knowledge gained from clinical trials on prevention and treatment is crucial. However, most of the focus has been on diversity in clinical trials at NIH.  While that is extremely important, it is the FDA that is making the decisions about what medical products can be sold in the US, and what the indications are for those products.  When a medical product is studied on a small number of patients of color, and those patients are too few to analyze separately in subgroup analyses, the result is that drugs and devices are put on the market as FDA approved for all patients, but they may not be equally effective for men or women of color.

This lack of diversity contributes to disparities in health or mortality because treatments have not been tested to be safe or effective in populations of color. For example, some diabetes medications might be more effective for African Americans than others, but for the most part that information is not available because the FDA has not required that the drugs be studied on adequate samples of African American patients before they are approved.

Our Center participates in numerous FDA Advisory Committee meetings to share our concerns on this topic. Two weeks ago, for example, I spoke at an FDA advisory committee meeting for a weight-loss device. Even though four out of five Black women in the U.S. are overweight or obese and Hispanic women are more likely to be overweight compared to White women as well, the clinical trial was 93% White. Only 11 patients were Black and only 2 were Hispanic. Although the Advisory Committee voiced their concerns, they voted for approval of this device anyway.  This shows 2 problems: 1) the FDA is not requiring diversity in clinical trials and 2) when companies fail to include sufficient numbers of minority groups to conduct subgroup analyses, the FDA is not providing instructions to their Advisory Committees that such diversity is essential for FDA approval and for public health.

Unfortunately, patients of color have historically been disproportionately underrepresented in studies submitted to the FDA and this under-representation continues to this day. The FDA’s own “907 Report,” published last year, highlights that racial and ethnic minorities are included in too small numbers in clinical trials submitted to the FDA.

As we consider methods to reduce health disparities in diseases that are particularly harmful to communities of color, a first step should be for the FDA to require diversity in clinical trials submitted to them by companies seeking FDA approval.

As part of the conversation today and tomorrow, we hope that you will discuss ways to strengthen partnerships with the FDA’s Office of Minority Health and FDA’s Center for Drugs (CDER) and Center for Medical Devices (CDRH) to work towards ensuring adequate racial and ethnic minority representation in clinical trials as well as approval decisions based on subgroup analysis and clearly delineated in FDA approved labels.

Jon Kamp, Wall Street Journal, appeared on Fox News

July 8, 2014

A U.S. Food and Drug Administration database that catalogs medical-device problems holds thousands of entries on a tool called a laparoscopic power morcellator, used since the 1990s.

But before December, none of those reports linked the tool to a potentially deadly risk: worsening a rare and often-hidden uterine cancer. Instead, older entries documented malfunctions and occasional injuries to patients.

Medical studies and literature had highlighted the risk since the tool made its debut. But the FDA didn’t begin to closely examine the danger until late last year, when a Boston doctor took her case public in The Wall Street Journal.

As the FDA prepares to hold hearings this week in Maryland on the future of the device, critics say the power morcellator case demonstrates weaknesses in the surveillance network for medical tools after they hit the market. An agency panel will hear testimony Thursday and Friday.

Morcellators use tubular blades to slice up common and generally benign uterine growths called fibroids so they can be removed through small incisions in minimally invasive procedures. In April, the FDA discouraged these procedures while estimating one in 350 patients could have a concealed cancer that the devices may spread.

Through medical records and interviews with doctors, the Journal has identified at least two dozen cases before December where morcellators likely worsened cancer. Medical journal articles and researchers have also identified numerous cases since the devices were introduced.

“Why is it that even when we know numerous women have already had this happen to them, it’s not in the database?” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit organization that advocates for product safety.

The FDA approves nearly all devices, including morcellators, through a process that typically allows products deemed substantially similar to something already marketed to be sold without clinical-trial data. The FDA has said it is unlikely a more intense review would have highlighted the morcellator’s cancer risk sooner, but this approval process in general requires close post-approval monitoring, experts said.

The FDA in recent years developed a blueprint for significant surveillance improvements. But for now, the agency’s principal tool remains a system that gathers reports on problems in a huge database called Maude, which can be mined to find or analyze these problems, said Rita Redberg, a cardiologist at the University of California San Francisco Medical Center who is critical of FDA surveillance efforts.

The agency requires hospitals and device makers to chronicle cases linking devices to deaths and serious injuries. Doctors don’t have to report issues, although they are encouraged to and can even do so by smartphone.

The FDA said it long knew morcellators could spread uterine cancer, but the fresh attention prompted new analysis that found a higher-than-realized cancer risk. The agency said it didn’t know why it didn’t receive reports of morcellator-worsened cancer before December but noted doctors would have to connect advanced cancer cases to an earlier procedure.

Magdy Milad, chief of gynecology and gynecologic surgery at Northwestern Memorial Hospital in Chicago, agreed. He wrote a recent paper citing underreporting of complications linked to morcellators but said it is a particularly daunting problem to catch.

There were times connections were made but not reported. Boston’s Brigham and Women’s Hospital confirmed two instances where morcellation worsened cancer, including the case that sparked public debate last year. While the hospital regularly files device reports, risk managers there didn’t think this was a reportable issue, a spokeswoman said.

“The devastating outcome was the result of the undetected cancer, not device malfunction or failure,” she said.

Regulatory experts said the hospital should have reported the cases. But they also said lax enforcement gives hospitals and doctors little incentive to call out mishaps.

The FDA said it found that encouraging more and better reports from hospitals and doctors, rather than levying sanctions, is the best use of limited resources. The agency said it focuses more on enforcement with medical-device companies.

The lack of earlier morcellator alarms worries Jim Leary, a Greece, N.Y., police officer whose wife, Barbara, died in September. She was diagnosed with uterine cancer in early 2009 after a doctor at Rochester General Hospital used a morcellator to remove what looked like a benign fibroid, according to the patient’s records.

A month after she died at age 52, the same doctor at the same hospital performed a similar procedure on another woman who is now battling advanced cancer, according to the patient’s records. The hospital and doctor have declined to discuss these cases.

More than 20 reports linking morcellation and cancer have filtered into Maude since December. Some came from patients, others from doctors, including the husband of the Boston patient who raised alarms, and several came from Johnson & Johnson, the largest manufacturer of the device. They were triggered by lawsuits and news stories, a J&J spokesman said.

J&J halted global morcellator sales after the FDA’s April advisory, though it has defended the devices’ safety.

The FDA’s planned surveillance improvements include rolling out device codes over the next several years that could link to patient records. Other steps include planned improvements to the adverse-event reporting system and analysis tools.

But these reforms are coming slowly, said Michael Carome, director of the health research group at consumer advocate Public Citizen.

“The FDA has known for decades that their post-market surveillance methods for devices are inadequate,” he said. “They have been very sluggish.”

As new drugs and medical devices make their way through the FDA’s approval process at an accelerated rate, research on their safety and efficacy is not always as rigorous as it should be, experts cautioned at a AAAS event.

Read the article: http://www.aaas.org/news/faster-approval-new-medical-products-heightens-uncertainty-over-risks

Coalition Comments of Members of the Patient, Consumer, and Public Health Coalition on the “Proposed Risk-Based Regulatory Framework and Strategy for Health Information Technology Report”

July 7, 2014

Division of Dockets Management (HFA-305)Food and Drug Administration

5630 Fishers Lane, Rm. 1061

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Comments of Members of the Patient, Consumer, and Public Health Coalition  “Proposed Risk-Based Regulatory Framework and Strategy for Health Information Technology Report” Docket No. FDA-2014-N-0339

As members of the Patient, Consumer and Public Health Coalition, we appreciate the opportunity to comment on the Proposed Risk-Based Regulatory Framework and Strategy for Health Information Technology Report.

The Food Drug and Administration Safety and Innovation Act (FDASIA) required the Food and Drug Administration (FDA) to work in consultation with Office of the National Coordinator for Health Information Technology (ONC) and the Federal Communications Commission (FCC) to prepare a report on a framework for health information technology (IT), including mobile medical applications. The report reviews strategies to promote innovation, protect patient safety and avoid regulatory duplication.1

We are unable to support the proposed framework because we are very concerned that it will undermine the FDA’s mission to protect the public health. The framework “primarily relies” on the ONC and private sector capabilities to coordinate activities with FDA and FCC, which leaves the FDA in a secondary role. The FDA has overseen medical devices, including medical device software, for nearly four decades. The Agency has been regulating software on mobile platforms for more than a decade and has cleared approximately 100 mobile medical apps such as ECG machines and smartphone based ultrasounds.

We strongly disagree with the report’s recommendation that “no new or additional areas of FDA oversight are needed.”1 Since many health IT products—especially mobile medical applications–are in their infancy, we do not know how much FDA oversight these products will require. To broadly state that no new FDA oversight is needed is premature and dangerous.

The report focuses on the potential benefits of health IT and downplays the risks. For example, in the Introduction section, there is a long paragraph about the “tremendous benefits” of health IT followed by a single sentence that “it can pose risks to patients.” In fact, poorly regulated health IT devices can seriously harm huge numbers of patients, and can be fatal.

Below are our comments on specific sections of the report:

Administrative

The report states that administrative health IT functions (such as billing and claims processing) pose little risk to patient safety. We agree with this assessment and are fine with ONC oversight of these products, as long as they are not defined as medical devices.

Health Management

We strongly disagree with FDA’s proposal to selectively enforce medical devices that fall into the health management category. FDA stated, “If a product with health management health IT functionality meets the statutory definition of a medical device, FDA does not intend to focus its oversight on it.”

This will lead to more inconsistencies in the clearance or approval of medical devices—something that the medical device industry has complained about for years. We also do not agree with the report’s conclusion that the safety risks for health management functions “are generally low compared to the potential benefits” because there is no scientific evidence to support that statement. We do not have a reliable measure of safety risks. The report also notes that most health management health IT “products, services, or systems are not devices…and are not required to register and list with the FDA.” The report then provides a short list of health management medical devices currently regulated by FDA. We would like to see the FDA provide a list of all health management devices to the public.

Medical Devices

We agree with the FDA that it should focus on the functionality of medical products such as mobile application devices rather than its platform, in deciding whether or not it is a device. We agree with Center for Devices and Radiological Health Director Jeff Shuren who cited an EKG device as an example. He said that “Traditionally, it’s [an EKG device] a box and it comes on a table. Today, there’s a software program we’ve cleared and it will transform your smartphone into an EKG machine. It’s used for the same purpose, and we treat it the same.”2

Promote the Use of Quality Management Principles.

This needs to be better defined. The report states that “Quality management principles help to identify, prevent, track, and monitor safety hazards and to reduce risks.” But it does not state how that should be done, except to note that health IT developers “must have flexibility.” Flexibility can lead to subjective and inconsistent standards. The statement: “The Agencies [FDA, ONC, and FCC] view this strategy, rather than a formal regulatory approach, as the appropriate method for advancing health IT quality framework,” is not backed up by evidence comparing quality management principles to regulations, especially in regards to safety and effectiveness.

Identify, Develop, and Adopt Standards and Best Practices

This section is silent about enforcement. Since the standards are “not binding,” what happens if the standards are not met?

Leverage Conformity Assessment Tools

The conformity assessment tools are voluntary. If the product fails testing or certification or other standards, will it still be allowed on the market? We strongly oppose the “development [of] post-implementation tests [that] could help users monitor whether their systems meet certain safety benchmarks.” Safety benchmarks should be met before products are allowed on the market. We strongly disagree with the report’s recommendation that voluntary conformity assessment tools should be implemented by the private sector. This would create conflicts-of-interest because the companies doing the assessments would want to please their clients (the ones whose products they are certifying) and this would lead to less rigorous reviews.

Create an Environment of Learning and Continual Improvement

This section would create a voluntary system to identify adverse events and near misses and analyze events and identify patterns. This proposal is directly contradicted by a 2012 IOM report noted that there is “Persistent underreporting of patient safety events and near misses, even when there are well-established programs in place encouraging health professional to report.”3 We are concerned that this voluntary system will understate the problem, as is the case for FDA’s voluntary adverse reaction reporting system.

Safety

The ONC has also suggests creating a public/ private Health IT Safety Center. Public-private partnerships are often primarily funded by industry, and frequently represent industry’s views more than consumers’ views. The report also states that IT stakeholders want a “reporting environment that is non-punitive, arguing that disincentives to transparent reporting include fear of liability, [and] punitive action…” That may be true for industry stakeholders but not for consumer stakeholders or patients who have been injured or killed by faulty health IT products. The report states that “the ultimate goal” of the public-private partnership is to create a “health IT learning system that avoids regulatory duplication.” That is an inappropriate goal for a “Safety” center. The report noted that ONC released a report in July 2013 with short-term strategies to improve health IT safety that included increasing the quantity and quality of data by using the FDA’s Manufacturer and User Facility Device Experience (MAUDE) database and using the Agency for Healthcare Research and Quality (AHRQ) to analyze patient safety information from private companies. This is inadequate because MAUDE is known to greatly underreport adverse events. If private companies are not required to report all adverse events, they would most likely under-report as well.

Risks

The report found several challenges to a successful health IT system including “poor human-computer interactions can contribute to serious injury and Death;” and “Significant knowledge gaps exists in our understanding of the benefits and risks to patients associated with different health IT functionalities.” The report also notes evidence that health IT can “result in adverse consequences, such as medication dosing errors or delays in diagnosis and treatment.” These are serious risks, and that is why these products should be regulated by the FDA as medical devices that require evidence of safety and effectiveness, not a public-private partnership whose interests may not be aligned with public health.

Conclusions

Health care providers and patients rely on the FDA to establish that a device is reasonably safe and effective. If FDA does not carefully scrutinize health IT products to evaluate evidence that those that are devices have benefits that outweigh the risks, patients may be seriously harmed. Even if the health IT product is not harmful, if it is ineffective, then patients could be harmed by inaccurate results that are either anxiety-producing or erroneously reassuring. These outcomes could result either in unnecessary testing or serious illness or death.

For the above reasons, we believe that the proposed health IT regulatory framework will undermine the FDA’s primary mission, which the FDA Commissioner has emphasized is to protect the public health. Health IT products must be shown to be safe and effective and the results they claim must be shown to be accurate and supported by sound scientific evidence. The FDA is the agency best suited to make those determinations, and will need resources to do so. All health IT products that are medical devices (including health management devices) should be regulated by the FDA, not overseen by ONC or FCC. American Medical Women’s Association Annie Appleseed Project National Center for Health Research Center for Science and Democracy at the Union of Concerned WoodyMatters The Patient, Consumer, and Public Health Coalition can be reached through Paul Brown at pb@center4research.org or (202)223-4000.

1 Food and Drug Administration (2014). FDASIA Health IT Report; Proposed Strategy and Recommendations for a Risk-Based Framework. http://www.fda.gov/downloads/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDRH/CDRHReports/UCM391521.pdf

2 Ravindranath, M (April 13, 2014). FDA report hints at relaxed approach to IT rule-making. The Washington Post. http://www.washingtonpost.com/business/on-it/fda-report-hints-at-relaxed-approach-to-it-rule-making/2014/04/11/8fe88938-bf30-11e3-bcec-b71ee10e9bc3_story.html

3 Institute of Medicine (2012). Health IT and Patient Safety: Building Safer Systems for Better Care