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.

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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.

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.