Thomas M. Burton, The Wall Street Journal: March 9, 2016

WASHINGTON—A Senate committee Wednesday approved a slate of bills that would relax requirements for approval of medical devices by the Food and Drug Administration, part of a larger effort aimed at speeding up the regulatory process and boosting medical research.

Most of the measures were approved with bipartisan support, but there are indications of discord on the package. Some patient-safety advocates said the legislation would weaken the FDA’s ability to ensure the dependability and safety of medical devices.

[…]

At issue is how aggressively the FDA will regulate medical products. The “breakthrough” [products] bill, for example, would allow the use of shorter or smaller clinical studies and quicker measures of success, and medical-device safety experts have already expressed concern that the standards for device approval are significantly lower than those for drugs.

[…]

The “breakthrough” bill “sets a low bar to qualify for ‘breakthrough’ status’ ” and “lowers standards for safety and effectiveness,” said Diana Zuckerman, president of the National Center for Health Research in Washington, a medical research and advocacy group.

[…]

“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,” Dr. Zuckerman wrote on behalf of her group and 13 other medical safety groups in a letter to the senators on the HELP committee.

To read the full article, click here.

Peter Korn, Portland Tribune: March 3, 2016

If he were playing it safe, Dr. Vinay Prasad might be among the last to turn into a medical provocateur.

Prasad works at Oregon Health & Science University’s Knight Cancer Institute as a cancer physician, and OHSU has gone all-in trying to make it a flagship department.

[…]

Prasad’s outspokenness and willingness to criticize industrywide assumptions place him in the ranks of a small but influential group of physicians and researchers who are either having an impact on the way medicine is practiced in the United States, or tilting at windmills. That depends on whom you ask.

Prasad’s voice is starting to get noticed on a national stage, says Diana Zuckerman, president of the Washington, D.C.-based National Center for Health Research. When Prasad published a study last year showing new cancer drugs were no more effective than cheaper drugs already on the market, Zuckerman had the work distributed to members of Congress, the White House and science reporters around the country.

“To me, that’s huge news,” Zuckerman says of Prasad’s findings. “Why wasn’t that in every media outlet in the country? Why wasn’t it on every TV news program?”

Zuckerman says she knows why: “It’s not funded by the (pharmaceutical) industry.”

She calls Prasad an iconoclast, and says the work he and others perform detailing conflicts of interest is more important than ever, because there is more money involved today and more lives affected when bad drugs are put into the marketplace.

“It’s one thing when there’s only one cancer drug and it’s not very good, but maybe it’s better than nothing,” Zuckerman says. “But it’s another thing when there are 10 different cancer drugs for the same type of cancer and eight of them have exaggerated their effectiveness in the research, and the two that are perhaps the best are the oldest generic drugs that nobody is advertising or promoting because they don’t cost much.”

[…]

Many of the treatments Prasad assails turn out to have unintended consequences. Ironically, it is just such an impact that has him worried about his own advocacy work, something he calls “the third harm.”

If people lose trust in the medical system, he says, science suffers. He’s aware that fewer than 10 percent of adult cancer patients enroll in clinical trials designed to reveal the best therapies. Still, third harm or not, he says he has to speak out.

Zuckerman agrees.

“It’s hugely important that they’re doing this,” she says. “If they weren’t, nobody would have any idea of what’s going on.”

Read the full article here.

Joe Williams, Inside Health Policy: February 16, 2016

FDA’s device center chief Jeff Shuren, in an interview with Inside Health Policy, highlighted the benefit a robust National Device Evaluation System could have in bolstering the agency’s ability to monitor devices in real-time to better inform the risk-benefit profile of approved products. Such a system, he said, could also help in the premarket approval phase to reduce the clinical trial burden on manufacturers and get products to market faster.

The program is a high priority for the Center for Devices and Radiological Health within FDA. It was recently listed in CDRH’s 2016-2017 priority agenda, and FDA in the fiscal 2017 budget requested $1.8 million in funding to help establish the system as part of President Barack Obama’s Precision Medicine Initiative. The agency will hold a public workshop in March on the effort.

The goal for FDA, according to Shuren, is to move past the traditional scope of a national surveillance system and launch a program that would allow the agency to more accurately track and analyze the use of medical devices in a real-world setting and better understand the benefits and risks of specific products outside of the clinical trial setting. […]

“I think more money is problem number one and without more money the agency is never going to do a good job,” Diana Zuckerman, president of the National Center for Health Research, told IHP.

Stakeholders have suggested the medical device user fee process is more difficult to navigate than the sister program in the drug center, given the financial disparity in the medical device industry between the large corporations and smaller start-ups. Zuckerman, however, argued that no one is expecting a small device company to pay millions in user fees to cover something like postmarket surveillance.

“There are many small companies and, yes, small companies should have user fees that are affordable, but the medium, large and extra-large sized device companies could be paying a lot more,” she said.

Johnson & Johnson, for example, pays roughly $2.4 million in user fees for a new drug application, sources say, and only $5,000 for a 510(k) submission. A spokesperson for J&J did not respond to inquiries.

Increasing the amount of user fees for the large companies, sources say, could help fund FDA’s postmarket surveillance goals. Sources familiar with the user fee discussions tell IHP, however, that the medical device industry, in particular the Advanced Medical Technology Association, has rejected the notion that industry should have to pay more for faster approvals. […]

To read the full article, click here.

Tinker Ready, HealthLeaders Media: February 11, 2016

Hospitals don’t have much to gain from the moonshot, at least in the short run. There are lots of other pressing, fixable problems with cancer care that the Obama administration’s effort won’t address. […]

Let’s assume billions for research are well spent. Everyone is happy when we fund cancer research. Why be a buzz kill? Because, in reality, there are a whole lot of other problems with the way we develop and deliver the treatments we already have.

Prevention and Care Delivery

The Cancer Moonshot might do a lot more to improve the prospects for cancer patients by looking beyond cures and paying attention to problems with cancer care delivery, costs, access and prevention. With hospitals’ fortunes now tied to outcomes and population health, they should be looking for breakthroughs in those areas, not toward another marginally effective $100,000 drug.

Diana Zuckerman is a former Congressional aide and long-time DC-based women’s health advocate. She is one of the founders of the Cancer Prevention and Treatment Fund, a non-profit group that promotes cancer risk reduction and helps patients “in choosing the safest and most effective” treatments.

Zuckerman supports additional research funding. She’s just not sure the moonshot will change much.

“This administration has a year left,” she said “What are they going to accomplish? It takes more than a year. We’ve had so many wars on cancer and we’ve had a lot of progress. But If you want to make meaningful progress, you don’t [just] throw money at a problem for a year.”

Her wish list: Zuckerman would like to see more FDA scrutiny, both before and after approval, of marginally effective cancer drugs. Costs are another issue and one way to keep them down would be to prevent drug makers from charging so much for treatments made possible by publically funded NIH research. Like the moonshot. […]

To read the full article, click here.

By Robert Lowes, Medscape

December 31, 2015

Hammered for its regulation of medical device safety, the US Food and Drug Administration (FDA) today proposed telling the public about “emerging signals” of possible device risk before it determines whether the risk actually exists.

Such early warnings already are issued for drugs through the FDA Adverse Event Reporting System (FAERS), a database on adverse event and medication error reports submitted to the agency. A dramatic fall-off in the number of drugs flagged for potential risk signals in FAERS since 2012 prompts FDA critics to wonder whether a similar system for medical devices will benefit the public.

In today’s proposal, the FDA explained that it historically has alerted the public about safety concerns that crop up after a device goes on the market, and usually after it has determined what to do about them. Agency responses include recommendations for the “device user community” and possible regulatory action.

However, the FDA said there is a need to notify the public “about emerging signals that the agency is monitoring or analyzing, and for which the agency does not yet have specific recommendations.” […]

Diana Zuckerman, PhD, president of the National Center for Health Research, a think tank focused on children and adults, said she does not believe that the downward trend line means that the FDA has been receiving fewer adverse event reports. Rather, the process of disseminating possible risk signals has become “moribund.”

“We think FAERS information is not being made public in a timely manner,” Dr Zuckerman toldMedscape Medical News. She cites lack of regulatory and political will as the reason. FDA user fees collected from drug manufacturers “are dependent on speed of review for drug approvals, not on timely FAERS information,” she said. “Similarly, Congress has complained about the speed of drug and device approvals, not on the speed of warning safety signals.

“Unfortunately, safety is not currently a priority at the FDA or Congress,” Dr Zuckerman said. […]

To see original article, click here.

Carolyn Johnson, Washington Post

November 24, 2015

To patients grappling with incurable diseases, new therapies can’t come quickly enough. A bill that hopes to address this need sailed through the House in the summer with the goal of getting more “21st Century Cures” through the drug approval pipeline. But a pair of new studies found that speeding up this process could put drugs that are ineffective or harmful on the market.

At least for complex neurological diseases, such as Alzheimer’s, Parkinson’s and depression, recent history suggests that approving drugs faster based on biomarkers — early signs that a drug could be working — might make drugs that ultimately don’t work available to patients, two teams of researchers found.

These early biomarkers are one of several ways the House bill could speed up the drug approval process. A Senate version of the bill is expected to be presented soon, with many wondering how similar it will be to the House version. The Food and Drug Administration has said the House legislation wouldn’t override its ability to approve safe and effective drugs.

[…] A second study published in the British Medical Journal this week examined three Alzheimer’s drugs that progressed to late-stage trials based on positive data in earlier stage trials that suggested the drugs were working. In each case, the team found that the phase two trials suggested the drugs would work — two showed encouraging decreases in measurements of beta amyloid, a protein that builds up in the brains of Alzheimer’s patients. One drug, semagacestat, was then tested in 1,537 patients and was found to cause skin cancer and even worsen patients’ ability to perform activities of daily life. The other two drugs looked promising in intermediate trials, but also failed once tested in large numbers of patients.

The researchers projected that if the semagacestat had been approved based on its encouraging results in intermediate trials, the treatment would likely have cost thousands of dollars a year, treated 234,000 patients, and caused 19,000 cases of skin cancer.

“This is just yet another example where, if you look at these very reasonable surrogate endpoints …  you can get results that are completely different when you look at things you really care about, which, in this case, is memory loss and the ability to function, day-to-day,” said Diana Zuckerman, president of the National Center for Health Research.

[…] Read full article here.

Susan F. Wood and Diana Zuckerman, The Washington Post

November 19, 2015

Precision medicine is the next big thing in health care, and it’s also one of the few health goals that Congress and the White House agree on. But while we await treatments targeting the precise genetic makeups of individuals and diseases, medical researchers still are not paying enough attention to the most important kinds of differences among patients: those of sex, age and race.

A clear example of this disconnect is the 21st Century Cures Act, which was passed overwhelmingly by the House of Representatives and is being scrutinized by the Senate. The stated goal of the bill is wonderful: to stimulate the development of new cures for a range of diseases. Many medical schools and patient organizations are supporters, since the proposal would provide almost $9 billion more for the National Institutes of Health — including a boost for precision medicine. The 360-page bill offers other potential benefits as well. But an immediate impact would be to ignore how differences between men and women and younger and older patients influence the safety and effectiveness of many medical products.

Throughout the 20th century, most medical research was conducted on relatively young, healthy men. In recent years, researchers have realized that treatments often affect women and older patients differently than men or younger patients. These differences can affect safety and effectiveness. The sleeping pill Ambien, for example, makes women drowsier for longer periods than it does men, putting them at risk if they drive the next morning. Since most medications are taken by people older than 65 and women of all ages, it makes sense to analyze the effects of age and sex on the drugs’ safety and effectiveness before they can be sold.

But the 21st Century Cures Act is based on the assumption that there will be more cures if drugs and devices are studied more quickly by testing them on fewer patients — in some cases, on just a handful. Unfortunately, such studies would be too small to allow safety and effectiveness findings to be broken down for subgroups such as men, women, young adults and seniors.

This embrace of smaller, more preliminary studies could drastically lower scientific standards. When fewer people are studied, it is more likely that a drug will seem safe and effective even if it has dangerous side effects for many patients — who may not have been included in those small studies.

In addition to allowing smaller studies, the House bill would encourage the Food and Drug Administration to determine a drug or device’s effectiveness based on “clinical experience,” which the bill defines to include the experience of one or more doctors or patients. Scientists call these anecdotes and note that just because one doctor has had success treating a few patients with a particular drug does not prove it is either safe or effective. Worse, the bill specifies that after studying only small groups of patients, drug manufacturers could sell a new treatment to anyone, even if the patient was not among the types studied. In fact, hospitals would be paid extra to make it financially feasible to prescribe more expensive new drugs to Medicare patients, even if the drugs were never studied on patients older than 65 (the age of most Medicare patients).

Similarly, lifesaving medical devices, such as heart valves, could be approved based on case histories, which are written descriptions of the experiences of just one or two patients. They are unlikely to be good predictors of how a treatment helps or harms most patients.

The recalls of drugs such as Vioxx and devices such as metal-on-metal hips in recent years have made clear that inadequate testing can produce ineffective and harmful products. And since new drugs tend to be much more expensive than older ones, the costs of widely used, unproven medical products can be enormous in both human and economic terms.

These sections in the 21st Century Cures Act go in the opposite direction of the push for precision medicine and what we’ve learned about differences between male and female and older and younger patients. The General Accountability Office has concluded that the NIH needs to make a priority of analyzing data related to sex differences. Just three years ago, the House and Senate overwhelmingly passed legislation that directed the FDA to ensure that men and women, old and young, are studied, with results analyzed to see which treatments are safest and most effective for whom. Why is Congress undermining that law?

Congress should surely increase funding for research to find 21st-century cures, but the price should not be returning to early-20th-century standards, when unproven medical products were widely available and often put all Americans at risk.

See the article here.