By Ed Silverman, STAT

May 17, 2016

In a quest to bring new medical products to Americans, Congress is considering a grand bargain.

Legislation passed last year by the House would provide billions more dollars for medical research and encourage faster approval of prescription drugs and devices. The Senate is currently working on a set of companion bills in hopes of crafting a compromise measure.

“If we succeed, this will be the most important bill signed into law this year,” Senate health committee chairman Lamar Alexander, a Tennessee Republican, said at a committee meeting last month.

This sounds promising. After all, adding $9 billion to the National Institutes of Health’s budget over the next five years to underwrite new cures is a good idea. And in an era when desperately ill patients are clamoring for new medicines, giving the Food and Drug Administration extra tools also makes sense.

But there’s a catch. By linking the extra funds to speedier approvals, Congress appears ready to undermine regulatory standards. This is a misguided notion that, unfortunately, is more likely to help companies than patients. […]

“This is a harsh way of putting it, but this is why I call it the 19th Century Fraud Act,” said Harvard University political scientist Daniel Carpenter, who studies the FDA. “This is a part of the bill that threatens to take us back more than a century.”

The final details of the Senate bill remain uncertain, but some proposals are also prompting objections. As an example, one suggestion for speeding approvals of medical devices is to label certain products as “breakthroughs,” a designation that would offer an expedited review pathway. To qualify as a breakthrough, though, a company need only argue its product is either a significant advance over existing devices or is in the best interest of patients.

The use of the word “or” is problematic, because it creates wiggle room for unproven claims. “It’s very vague and only encourages companies to seek breakthrough status,” said Diana Zuckerman, who heads the National Center for Health Research, a nonprofit think tank. […]

There is no question that more research funding is necessary and that finding legitimate ways to get medicines to patients faster is crucial. But Congress ought to separate the debate over research funding from the rest of the legislation. Loosening regulatory standards would only create problems for which real cures will be needed.

Read the full article here.

By Thomas M Burton, Wall Street Journal

WASHINGTON—Moves in Congress to link billions of dollars in new medical research funding to revised standards for drug and medical-device approvals are troubling some public-health experts, who say the combination makes it too easy for lawmakers to support lower patient-safety standards.

These safety advocates say legislation to beef up research funding for the National Institutes of Health should be separated from product-approval changes at the Food and Drug Administration.

“This is the first time this has been done this way, and it’s a deal with the devil,” said Dr. David A. Kessler, onetime FDA commissioner during the 1990s under presidents of both parties. “It’s time to uncouple the promise of research funding from the requirement that FDA standards be lowered.”

The linkage was first made last July when the House passed a bill to ease FDA approvals of drugs and devices in ways broadly endorsed by the drug and device industry. That legislation carried a big infusion of cash for medical research—$8.75 billion for NIH and $550 million extra for the FDA, both over five years. The measure, called the 21st Century Cures Act, passed 344-77 with broad bipartisan support.

In the Senate, the leader of a parallel effort said he hopes to bring a package of 19 FDA and NIH bills to the floor soon—along with a separate measure on NIH funds. Sen. Lamar Alexander (R., Tenn.) chairman of the committee that approved the package, said finding the source of the funding is “the last remaining important issue” on the legislation, which he considers “the most important bill Congress considers this year.” […]

The new FDA commissioner, Dr. Robert M. Califf, said in a recent talk that “this legislation, if not carefully crafted, could pose significant risks for FDA and American patients…Innovative therapies are not helpful to patients if they don’t work, or worse, cause harm,” he said.

Patient-advocacy groups like the National Center for Health Research and Public Citizen have said several bills now in Congress could jeopardize safety. […]

To read the full article, click here.

Diana Zuckerman, PhD, Guest Editorial for the American Journal of Public Health. May 2016

In 1916, Margaret Sanger opened the first birth control clinic in the United States. She was arrested for this shocking act, but her work changed women’s lives. On the presidential campaign trail a century later, our political leaders are still debating women’s reproductive health and rights. Unfortunately for the approximately 120 million adult women in America, women’s reproductive organs continue to be the only women’s health issue that is getting much attention in presidential politics.

While access to health care in terms of saving, killing, or replacing the Affordable Care Act is a popular topic on the campaign trail, candidates’ medical focus is still on our reproductive organs. Even when politicians talk about cancer, the focus is usually breast cancer. To paraphrase Ronald Reagan in one of his better movie roles, what about the rest of me?

THE POST–FAMILY PLANNING GENERATIONS

While reproductive health is a very important issue for many women, there are 63 million women in the United States for whom family planning is a distant memory—for themselves and often for their daughters. For those women, Medicare and affordable insurance are very important, but so is an array of other health issues that have been too nuanced or complicated to make it into the presidential candidates’ talking points. Even the candidates that know we have the most expensive health care system in the world but rate 33rd in quality of care tend to focus on high prices and lack of access to care, ignoring the equally essential policy issues of lack of evidence-based prevention and treatment strategies and comparative effectiveness data. While those latter phrases do not make great sound bites, they have one issue in common that candidates know that women care about: the skyrocketing costs of prescription drugs. The assumption by the candidates has been that although the prices are too high, all screening and medications are essential and patients deserve to have them. However, the candidates never question the risks compared with the benefits.

AFFORDABILITY AND EFFICACY

Women want to know, “Can I afford medical care and will it help me live longer—and better?” Outrageous medical costs have become an angry topic for all Americans, not just low-income ones. It is a particularly important issue for women, who live six years longer than men on average and annually spend 26% more for health care per person. In addition to their own health care costs, women are usually the caregivers for family members with medical problems,thereby bearing even more of the burden of unaffordable medical treatments. Pharmaceutical spokespeople claim that regardless of the costs, screening tests and medications save money by reducing the need for hospitalization and other expensive care. However, research indicates that is often not true. In fact, some types of screening do more harm than good, and many highly priced drugs are not safer or more effective compared with other, less expensive treatments.3 Even those that have modest benefits may not be worth risking serious side effects or sending one’s family into debt.

For example, scientists from the National Cancer Institute and the Oregon Health and Sciences University recently coauthored an article about the newest cancer drugs.These researchers studied all the cancer drugs that were approved by the US Food and Drug Administration (FDA) from 2008 through 2012, choosing those years so that they could review the research used as the basis of FDA approval and all required studies that were completed after approval. Of the 54 cancer drugs approved, 36 were approved based on a type of fast-track review system that enabled companies to get approval for their drug based on preliminary data, using surrogate endpoints such as tumor shrinkage rather than clinically meaningful outcomes such as survival or quality of life. Although the short-term results were considered promising, post market studies were required as a condition of approval, to make sure these drugs were truly effective. When they examined the research literature for the required post market studies, the authors found that only five (14%) of the 36 drugs had been found to improve overall survival (compared with placebo or an older drug), whereas published studies for half of the 36 drugs (all of which are prescribed to women) found no evidence of such benefit. There were no post market studies of survival published for the remaining 13 drugs. The published studies also failed to show any other benefits for most of the other 18 drugs. But despite published evidence of the lack of benefit for these drugs, they are still on the market. And, our research center found that many cost more than $100 000 per year.

Cancer is the leading cause of death for women between the ages of 35 to 84 years. Surely, paying more than one’s annual salary for cancer drugs that are not better, and sometimes worse, than either placebo or older, less expensive treatments is an important women’s health issue. If our politicians promised that comparative effectiveness research would be required for all new drugs and the results would be easily available to all physicians and patients, that would be a great benefit to all patients, and especially women.

PRECISION MEDICINE

One example of current policy efforts that could improve women’s health is President Obama’s Precision Medicine Initiative. Should presidential candidates jump on that bandwagon? The goal of precision medicine is to tailor treatments to small groups of patients, rather than the one-size-fits all approach of traditional drug development and medical guidelines. Ironically, however, President Obama’s FDA, like the FDA of every president before him, has continued to focus on one-size-fits-all analysis, usually ignoring even the best established, basic physiological differences that often affect the safety and effectiveness of screening tests, diagnostics, and treatments. For example, experts of all political persuasions agree that women, people older than 65 years, and some racial/ethnic groups tend to metabolize drugs differently or react differently to certain treatments.

As a result of the FDA’s failure to take even those first steps toward a more precise approach, cardiac implants are approved based on their safety and effectiveness in clinical trials constituted disproportionately of White men, diabetes drugs are tested primarily on relatively young White men and women, and it took more than 20 years for the FDA to issue warnings that the typical dosages for many popular prescription sleeping pills were unsafe for most women.

Rather than just waiting for a new government-funded Precision Medicine Initiative to be designed and implemented, wouldn’t it be politically popular for presidential candidates to demand that the FDA immediately require subgroup analyses of more diverse samples? Candidates could explain that this is the quickest, simplest way to start determining which tests and treatments are best for women (as well as all voters older than 65 years and people of color). Since women are more than half the US population and constitute 57% of people older than 65 years, that would be an easy way to improve the health of most Americans and make immediate progress toward the goals of precision medicine. And, it would not cost taxpayers a dime, because it would merely require companies to analyze their data differently. Meanwhile, the more complex aspects of the Precision Medicine Initiative should also be developed, but they will take years to become reality.

WHAT ABOUT THE REST OF ME? THE REST OF US?

It is difficult to imagine “subgroup analysis” and other technical research terms coming out of the mouths of presidential candidates. But, if the GlassSteagall provisions of the US Banking Act of 1933 (which protected consumers by regulating bank activities) are worthy of discussion at Democratic presidential primary debates, perhaps evidence-based prevention and treatment, comparative effectiveness research, or even ineffective surrogate endpoints could be the next big topic.

All voters deserve to have presidential candidates focus on health policy issues that could improve our health—even if they aren’t easily translated to sound bites. If women are asked what we want from our health care system, we will tell the candidates that our health concerns extend beyond our reproductive organs, and even beyond our own personal health needs. We want a health care system that works for us and for the people we care about, and that enables us to choose prevention and treatment strategies that are proven to work and that we can afford. With the right messaging, these public health issues could resonate with all voters—and especially women, who are the majority of US patients and their family caregivers.

To see the original article, click here.

By Victoria Stern, General Surgery News

April 12, 2016

The FDA approves or clears thousands of new medical devices each year. This highly complex process of review has garnered criticism in recent years. In this series of articles, General Surgery News navigates different aspects of the FDA medical device approval process to better understand the various pathways and help unravel the current debates and concerns. This final article in the series explores medical device recalls. […]

In 2014, the FDA’s Center for Devices and Radiological Health (CDRH) published an in-depth analysis of medical device recalls over the past 10 years. What the FDA discovered suggests that there may be cause for concern: Between 2003 and 2012, the annual number of medical device recalls had increased by 97%. Recalls almost doubled over the 10-year study period, increasing from 604 to 1,190, while the total number of products recalled in that time increased from 1,044 to 2,475. […]

So what might account for this rise in recalls? Although the evidence is limited, some experts have voiced concern that the lack of premarket clinical testing may increase our uncertainty about device safety, which may in turn lead to recalls after devices reach the market. […]

As a result, most medical devices do not undergo any safety testing before reaching the market (Milbank Q 2014;92:114-150). The majority of medical devices are cleared through the 510(k) pathway, which typically requires companies to show a device is “substantially equivalent” to an already approved device. Thus, 510(k)-cleared devices often bypass clinical testing, even when the predicate device was recalled or never assessed for safety and effectiveness,explained Rita Redberg, MD, MSc, a cardiologist in the Department of Medicine at the University of California, San Francisco.

Overall, less than 2% of approved medical devices go through the most rigorous FDA regulatory pathway, premarket approval (PMA), which requires companies to perform clinical tests. But even PMA-approved medical devices may not be examined rigorously enough. […]

Inconsistencies in the quality of clinical trials and lack of testing altogether may increase the likelihood that unsafe or subpar devices are approved. In a 2011 study, Diana Zuckerman, MD, and her colleagues evaluated recalls of high-risk medical devices between 2005 and 2009 (Arch Intern Med 2011;171:1006-1011). In that period, the FDA recalled 113 high-risk devices, 80 (71%) of which had been cleared through 510(k) and 21 (19%) of which had been approved via PMA. An additional eight (7%) were exempt from FDA review. According to the authors, 13 of the 80 510(k) recalled devices should have gone through PMA, given the severe risk they posed to patients if a malfunction occurred.

Overall, Dr. Zuckerman and her colleagues found that most medical devices recalled for life-threatening or serious hazards were cleared through 510(k), and concluded that the 510(k) pathway poses greater dangers to patients because it is less likely to uncover design or manufacturing flaws. […]

To see the full article, click here.

By AAMI, Medical Design Technology

April 4, 2016

Advances in technology and data collection can help regulators and other parties keep better track of the safety and performance of medical devices once they are on the market, opening the door to potentially faster product development. […]

This focus on faster, less expensive product development coincides with a move by the U.S. Senate to create a “breakthrough pathway” for FDA approval of medical devices. The Advancing Breakthrough Devices for Patients Act of 2015 would allow shorter or smaller clinical studies and quicker measures of success to serve as sufficient premarket evidence for the approval of devices with the potential to “reduce or eliminate the need for hospitalization, improve patient quality of life, facilitate patients’ ability to manage their own care (such as through self-directed personal assistance), or establish long-term clinical efficiencies.”

Following approval by a bipartisan Senate committee, some medical-device safety experts have expressed concern that the bill “sets a low bar to qualify for ‘breakthrough’ status’” and “lowers standards for safety and effectiveness,” as articulated by Diana Zuckerman, president of the National Center for Health Research (NCHR) in Washington, a medical research and advocacy group, in The Wall Street Journal.

“We are concerned that the focus of these bills is on getting medical products to market more quickly, instead of making sure that they are safe and effective,” NCHR and 13 other medical safety groups wrote in a letter to the members of the Senate Committee on Health, Education, Labor, and Pensions Committee. […]

The FDA’s goal is to gain access to 25 million electronic patient records from national and international clinical registries, claims data, and EHRs by the end of 2016. It is also aiming to increase the number of pre- and postmarket decisions that leverage real-world evidence by 40% during that same timeframe.

To see original article, click here