Brett Norman, POLITICO: April 29, 2016

A bill that would make it easier to bring stem-cell treatments to market is getting a major push from a GOP senator in a tough reelection fight, a deep-pocketed GOP donor and a centrist Washington think tank — and it’s raising alarm among federal regulators and some medical research experts who say the measure could put patients at harm.

The bill’s supporters say it will spur investment in a flourishing field of research and accelerate patients’ access to potential cures. But even some of the most high-profile advocates of stem cell treatments argue the bill would remove key regulatory safeguards for a promising, but largely unproven area of medical research.

Sen. Mark Kirk’s (R-Ill.) REGROW Act would allow companies to sell stem-cell therapies that have been shown to be safe but haven’t yet been proven to work — a lower standard that’s unprecedented for human treatments in the United States but has been recently adopted in Japan and Europe. […]

The bill instead would require a full traditional drug application demonstrating effectiveness after five years, or else FDA could revoke the approval. Companies would be required to monitor patients for side effects and FDA could pull a treatment off the market if any harmful consequences are detected along the way.

Some patient groups, academic centers and companies are supporting the bill, but it’s opposed by two of the largest advocacy organizations in the field: the Alliance for Regenerative Medicine and the International Society for Stem Cell Research.

“What we don’t need at this point are products that go onto the market under some conditional approval process that aren’t rigorously tested and could truly compromise the whole industry,” said ARM chairman Edward Lanphier, also the CEO of Sangamo BioSciences.

FDA declined to comment on the legislation, but lobbyists and Senate aides told POLTICO that senior agency officials expressed concern that the bill would undercut the longstanding standard that medical treatments be proven both safe and effective before they are allowed to be sold to the public. […]

Some patient advocates will fight it all the way. Diana Zuckerman, president of the National Center for Health Research, called the bill an effort to short-circuit the FDA’s “gold standard” for drug approvals that shows “a lack of understanding of the importance of well-designed research.”

“In recent years, Congress has been chipping away at that standard and REGROW is just the latest, and in some ways most egregious example of that,” Zuckerman said.

To see 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.4 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. 

Rachel Schulze, American Health Hotline: April 19, 2016

The Senate’s version of the House-approved 21st Century Cures Act (HR 6) is attracting stakeholder attention — and not just for its different approach. Patient safety experts say the bill has promise, but more changes are needed as the Senate and House look to create a final, joint proposal.

The House passed the 350-plus-page bill aimed at bolstering medical innovation 344-77 in July. The legislation targets two main areas: regulation for drugs and medical devices and federal funding for biomedical research.

At the time it passed in the House, the Cures Act enjoyed overwhelming support from lawmakers, American Health Line reported last year. However, stakeholders have raised concerns about the bill’s practical effects. Namely, parts of the bill pertaining to drug and device regulation drew criticism from patient safety advocates. The bill’s fiscal sustainability is also uncertain. […]

Cross-Examining the Patient Safety Proposals

The Senate HELP Committee’s decision to consider several smaller bills helped to address some patient safety advocates’ concerns regarding the Cures Act.

Diana Zuckerman, president of the National Center for Health Research, in an interview with American Health Line said, “By examining each aspect of the Cures Act piece by piece, the Senate rejected several of the worst sections of the so-called Cures Act.”

For example, she noted that the HELP bills do not include a provision for third-party review of medical devices. Zuckerman explained, “The third-party provision is especially dangerous because it would apply to high-risk devices that have been modified by the manufacturer.” She continued, “Instead of the company notifying the FDA of the change and proving that the modified device is still safe and effective, the company could hire a ‘third party’ to review the device companies’ written policies on quality control … and determine that the company’s policies are adequate and therefore the company can be trusted to make revisions to their high-risk devices.”

However, Zuckerman noted that “none of the Senate bills do much to reassure patients that FDA approval means medical products are truly proven safe and effective, as studied on sufficient numbers of patients, including women, men, people of color, and patients over 65.” She cited Sen. Patty Murray’s (D-Wash.) bill (S 2503) on reusable devices as an exception.

Linda Radach, a charter member of Washington Advocates for Patient Safety who works with Consumers Union Safe Patient Project and the National Center for Health Research, told American Health Line, “[L]egislation from both the House and the Senate has a strong common theme which places industry above what is best for patients.”

Zuckerman also expressed concerns about several of the proposals HELP has advanced. One Senate bill, the PATH Act (S 185), would require FDA to create a program for approving certain antibiotics for limited populations. Zuckerman noted that the PATH Act “is much better than the [Cures Act] version, but it does not protect against the overuse of antibiotics,” which is a major contributor to antibiotic resistance. […]

To see the full article, click here.

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.

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

Robert Lowes, Medscape Medical News: March 29, 2016

The US Food and Drug Administration (FDA) has an early warning system to help catch safety problems with drugs after they reach the market. In the second quarter of 2015, a class of diabetes drugs called SGLT-2 inhibitors showed up on its radar screen.

The public didn’t know about this blip until about 7 months later. By law, it should have known months earlier. FDA critics say the agency’s early warning system needs fixing.

The regulatory radar is built on the FDA Adverse Event Reporting System (FAERS), which receives reports of problems from physicians, nurses, pharmacists, patients and their family members, and attorneys as well as drug manufacturers who pass on complaints they get from the public.

[…]

To one public health expert, publication delays for the FAERS watch lists illustrate how the FDA caters more to drug manufacturers than patients.

“The FDA has many requirements, and Congress keeps passing legislation that adds more mandates without adding funds for staff,” said Diana Zuckerman, PhD, president of the National Center for Health Research, a think tank focused on children and adults. “In the last year, it has become more obvious that the requirements that FDA has as its first priority are the ones that benefit industry — faster approvals.”

The FDA is beholden to pharmaceutical companies, said Dr Zuckerman, because they pay user fees that partially fund the agency. These same companies lobby a Republican Congress for a quicker, less onerous review process, and Congress in turn pressures the FDA to speed things up.

The agency felt a different kind of pressure when GAO [Government Accountability Office, the federal watchdog agency] issued its report on January 14, and Congresswoman DeLauro immediately called the FDA’s data problems “a severe safety risk for American consumers.” Dr Zuckerman said it was no coincidence that roughly 3 weeks later, the FDA posted its FAERS watch lists for the first three quarters of 2015, followed by the fourth-quarter report on March 22.

The watch lists play an important role in patient care, she said.

“Doctors who know there is a possible adverse risk for a drug might be likely to report it themselves if they see it in their patients,” she said. “And they’d be more likely to consider alternatives.”

Likewise, said Dr Zuckerman, some patients may balk at taking a drug appearing on a watch list.

“It’s not that these safety signals mean ‘Never use this drug,’ ” she said. “But they’re warning signals. That’s the whole point.”

[…]

To see original article, click here.