Brett Norman, Politico Pro: September 16, 2016

Drug companies and research institutions will have to publicly report more clinical trial data, including results that show their products or experiments failed, under new policies rolled out Friday by HHS.

The new rules address the fact that researchers have routinely flouted requirements to report data to ClinicalTrials.gov. A final rule from FDA and a complementary policy from NIH clarify which trials must be reported and project a new federal commitment to crack down on violators – threatening fines of $10,000 per day or disqualification from NIH funding.

The widespread failure by institutions to report the data is “simply unacceptable,” NIH Director Francis Collins said on a call with reporters.

“This is fundamentally an ethical issue,” FDA Commissioner Robert Califf said. “This is about maintaining the trust that we have with [clinical trial] participants … that if it doesn’t benefit them, it will benefit others.”

A 2014 review of the results of 400 clinical studies found that 30 percent had not been published or shared with ClinicalTrials.gov four years after they had been completed. A study earlier this year found that 43 percent of the trials conducted at 51 academic medical centers went unpublished two years after the trial was completed.

The new requirements, which will take effect in January, clarify that all NIH-funded studies must be published on ClinicalTrials.gov. The FDA will also require publication of studies being done on drugs and devices even if they have not yet been approved. Most studies must be reported within a year of completion, but those involving unapproved products could wait up to three years.

FDA and NIH are also expanding the range of information that must be published, including the demographic data on study participants, any adverse events and the original plan for statistical analysis. The latter is meant to combat so-called p-hacking – when the original hypothesis of an experiment fails and researchers massage the data to come up with some other positive finding that is statistically significant.

“We’ve had a problem where people do the trial and don’t like the result, so they do another analysis,” Califf said.

FDA and NIH decided not to require a lay summary of trial results that would enable the public – not trained in statistics – to more easily digest the information that is posted, angering some consumer advocates.

“That is an outrageous decision,” said Diana Zuckerman, president of the National Center for Health Research. “Without it, clinicaltrials.gov will not be providing useful information to patients.” The data won’t be useful to physicians either unless it is summarized, she said.

Collins and Califf said they had been uncertain how to keep “bias” from creeping into the summaries, adding that advocacy groups should be able to harness the relevant data and make it available in a way that would be more useful to patients. […]

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Anna Edney, Bloomberg: September 13, 2016

The U.S. Food and Drug Administration has a message for doctors: The money you’re taking from pharmaceutical companies may be clouding your judgment.

Research sites where Pfizer Inc. had paid doctors at least $25,000 in speaking, consulting or other fees reported sunnier results for its smoking-cessation drug Chantix, the FDA disclosed Monday. At those sites, doctors studying the drug’s possible link to suicide risk and other behavior changes reported fewer side effects than at locations where colleagues accepted lower or no payments.

The FDA’s findings — part of an agency review of Pfizer’s proposal to drop the most severe consumer warning on the drug’s label — demonstrate the federal government’s concern about the influence of consulting and speaking fees on medical decisions. President Obama’s 2010 health law requires drug makers to report such payments for posting to a public database. The law followed years of efforts by U.S. Senator Charles Grassley, an Iowa Republican, to make drugmakers publicly disclose financial ties to doctors. […]

Diana Zuckerman, president of the National Center for Health Research, part of a coalition of consumer and other groups that petitioned to keep the warning on Chantix, said the FDA staff report shows that it doesn’t trust the “integrity of the data.”

“FDA clearly seems to be saying we can’t trust the results of this study — the way it was coded, the way it was analyzed, and by the way there’s conflicts of interest,” Zuckerman said. […]

Outside experts are scheduled to meet Wednesday to advise the FDA about the necessity of the warning on Chantix. The pill’s “black box” label — the agency’s strongest — currently cites risk of “serious neuropsychiatric events” such as suicidal thoughts or behavior. Chantix generated $671 million in sales last year,according to Pfizer. […]

To see the original article, click here

Michael D. Williamson, Bloomberg BNA: September 8, 2016

High-risk medical devices are sometimes approved using low-quality clinical data and this increases Medicare costs, a member of a Medicare congressional advisory panel said Sept. 8.

The Medicare Payment Advisory Commission’s meeting session reflected its first major examination of the medical device industry. The session may portend a deeper examination of the device industry’s practices, which could cause Congress and/or the Department of Health and Human Services to change payment rates for devices and related services, as the commission’s recommendations are fairly influential among policy makers.

Commissioner Rita Redberg, a cardiologist at the University of California San Francisco Medical Center, called for further MedPAC examination of how to push the Centers for Medicare & Medicaid Services to more broadly consider quality over an entire episode of care when deciding whether to cover a device.

Commissioners didn’t vote on recommendations. However, they debated how MedPAC staff should further research device company practices and their role in driving Medicare costs. Results of that research could prompt the commission to draft recommendations in the future.

In a separate session, commissioners discussed MedPAC staff progress on developing a uniform set of quality measures that could be used under a unified Medicare payment system for all post-acute care providers.

Tie to Rising Medicare Costs

Devices with a high risk to patients are sometimes approved by the Food and Drug Administration using poor quality data, Redberg said. This can result in low-quality devices being implanted into patients. These low-quality devices then need to be deactivated or removed, which drives up Medicare costs. […]

Redberg’s stance received praise from the leader of a patient safety group. “I completely agree with Dr. Redberg,” Diana Zuckerman, president of National Center for Health Research, told Bloomberg BNA Sept. 8. The National Center for Health Research is a nonprofit that encourages new and more effective programs and medical treatments.

In addition, the CMS should advise the FDA about the lack of data and safety and efficacy analysis specifically on patients over age 65, Zuckerman said. The lack of data “is a major problem for drugs and devices,” she told Bloomberg BNA. […]

To see the original article, click here

Diana Zuckerman, Chicago Tribune: July 21, 2016
This article was published in the Chicago Tribune, Baltimore Sun, Orlando Sentinel, Sacramento Bee, and syndicated in many other newspapers across the country.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

To see original article, click here.

Kristen Fischer, HealthZette: June 28, 2016

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.