By Paul Brown, Tracy Rupp, and Steven Findlay, Health Affairs
July 11, 2016
The full Senate may in the next few days consider companion legislation to the 21st Century Cures Act that passed the House last year. The legislation—currently 19 separate bills—makes substantial changes to the way the Food and Drug Administration (FDA) approves drugs and devices. Set to adjourn for an extended election-year summer recess on July 15, the clock is ticking. The congressional calendar in the fall is full and the Senate may simply not have the time to take up the complex legislation, and reconcile it with the House version, before the November elections.
We believe that’s a good thing. The legislation, while containing some useful measures, is flawed. Instead of hastily agreeing to it, Congress should postpone consideration until 2017 and attach the best of the 19 bills (see below) to must-pass legislation on FDA funding through industry user fees.
At that time, we believe the Senate should reject those bills that lower standards for drug and device approvals and safety, and risk adding to the rising cost of prescription drugs.
This is a story two years in the making. Proponents of the proposed legislation—drug and device companies, and members of both parties in the House and Senate—argue that the FDA stifles innovation and advances in treatment by approving drugs and devices too slowly compared to other countries.
That premise is faulty. Nearly two-thirds of the novel drugs approved in 2015, for example—29 of 45, 64 percent — were approved in the United States before being approved in any other country. The proportion was even higher in 2012 and 2013. The majority of these drugs (60 percent) took advantage of existing FDA expedited review programs—fast track, breakthrough, priority review, and accelerated approval—and nearly half (47 percent) were approved to treat rare or orphan diseases.
As for devices, research shows that “it takes the same amount of time or less for patients to gain access to innovative, high-risk medical devices” in the U.S. as compared to Germany, France, Italy, and Britain.
The House and Senate bills ignore the above facts. They essentially seek to speed-up the approval process by relaxing FDA’s safety and effectiveness standards. And to make that more palatable, sponsors have attached the changes to increases in funding for the National Institutes of Health and the FDA.
But while the public supports increases in biomedical research funding, it is deeply skeptical about lowering the standards for drug and device approval. In the most recent poll on this issue, just under 60 percent of Americans opposed changing federal regulations to speed the development and approval of drugs; 38 percent favored speedier FDA action, according to a STAT-Harvard poll released in May. Respondents’ main concern: faster approval would allow products on the market that don’t work or are unsafe.
The survey echoed a previous poll by Consumers Union that found 82 percent of Americans believe that preventing safety problems is more important than limiting safety testing to speed the clearance or approval of devices or promote innovation.
The 21st Century Cures Act received broad bipartisan support in the House in part because it put a significant amount of money for research on the table. The House bill pledges a $9 billion increase in mandatory funding for the National Institutes of Health over five years, gaining the support of universities and medical schools. It also promises $550 million to the FDA (which, according to the CBO’s analysis, would not fully pay for the additional workload the Act assigns the FDA).
The drug and device industries intensely lobbied House members to pass the legislation. The Pharmaceutical Research and Manufacturing Association (PhRMA) increased its lobbying from $4 million to $5.4 million in the quarter before the 21st Century Cures Act passed. The Advanced Medical Technology Association upped its spending from $550,000 to $740,000 in the same quarter. The Senate’s lobbying database listed more than 1,100 lobbyists working on the legislation. Many patient groups have also voiced their support for the legislation, based on their belief that the bill would result in more treatment options. An analysis of public comments for the House bill completed by Avalere noted that 43 percent were from patient advocacy groups.
The Senate bills are an improvement over the House’s 21st Century Cures Act. But, on balance, they are still weighted heavily in favor of speeding medical products to market by weakening FDA approval standards.
New drugs and devices can, of course, be an improvement over existing products. And when they clearly are, FDA has established pathways to get them to market and patients as fast as possible. The agency, for example, grants more than one-third of requests from industry for “breakthrough” designation for new drugs. But the history of medicine is replete with examples of drugs and devices that caused more harm than good, some of which were approved too hastily — such as Avastin for breast cancer, Vioxx for arthritis, metal-on-metal hip implants, and power morcellators. Innovative drugs and devices simply must be required to actually work and not harm patients. Methods of testing drugs and devices in patients are improving all the time, with new tools that help detect products that warrant further testing or could be dangerous. The default mode of this technical process must always be “safety first.”
Below are our concerns with some of the Senate bills followed by a list of bills we believe would improve public health.
Senate bills that would, in our opinion, increase risks to patients:
- The MEDTECH Act would prevent the FDA from collecting adverse events due to flawed electronic medical records, and from recalling certain types of defective medical software. Some of this software has had life-threatening flaws in the past, such as oncology electronic medical record systems that calculated and recorded incorrect drug dosages for highly toxic chemotherapy drugs.
- The PATH Act would allow antibiotics to be approved with minimal evidence of safety and effectiveness through a “limited population” approval pathway. But, antibiotics approved in this way are promoted by companies so that they are more widely prescribed in order to increase sales. As they are, we won’t have information about whether they’re actually safe or effective for those groups of patients. For example, fluoroquinolones are today widely overprescribed for urinary tract infections despite guidelines recommending other antibiotics and data linking their overuse to the development of resistance.
- The Advancing Breakthrough Devices for Patients Act would encourage shorter and smaller clinical trials for medical devices. Abbreviated clinical trials will make it difficult, if not impossible, to include sufficient participation from subpopulations such as women, seniors, and racial and ethnic minorities in the analysis of the trials. For example, a recent study of high-risk medical devices found that the median number of participants was only 65 patients. With so few patients, it’s difficult to draw reliable conclusions about safety and effectiveness, especially for subgroups of patients. In an increasingly diverse America, this is unacceptable. The bill also pushes FDA to rely on post-market studies for devices rather than ensuring safety effectiveness before hospitals and patients pay for the products.
- The Advancing Hope Act would continue the existing pediatric priority review voucher program through 2022. The program is currently set to expire at the end of September. The program’s ability to stimulate innovation is questionable: a recent GAO review of the program concluded that the six drugs for which vouchers have been awarded so far were in development before the program existed. By allowing drug makers to buy a priority review, the bill removes FDA’s ability to set its work priorities and resource allocations based on public health needs. In a time of threats such as the Zika virus, our government agencies must be able to prioritize public health, and not be bound by vouchers that were sold to the highest corporate bidder.
Senate bills likely to promote innovation and protect public health:
- The Preventing Superbugs and Protecting Patients Act will help prevent drug-resistant infections from contaminated duodenoscopes and other reusable medical devices that have caused harm and deaths. If enacted, this bill will require certain reusable medical devices to have validated cleaning, disinfection, and sterilization procedures prior to marketing. This will reduce the likelihood that these devices will transmit dangerous infections from patient to patient.
- The Advancing Research for Neurological Diseases Act will help people suffering from neurological diseases by providing critical information to researchers as they work on new treatments and cures. The bill requires the Department of Health and Human Services to collect neurological disease surveillance data, such as demographic information, risk factors, outcomes, and treatments. Providing such information is likely to enhance understanding of these diseases and the development of new treatments.
- The Next Generation Researchers Act recognizes the importance of investing in the brightest young researchers to ensure that the United States remains at the forefront of biomedical research.
- The Promoting Biomedical Research and Public Health for Patients Act reduces unnecessary administrative burdens on researchers and encourages compliance with clinicaltrials.gov reporting requirements.
- The FDA and NIH Workforce Authorities Modernization Act will make it easier for FDA to recruit and retain top scientific and technical experts by making salaries more competitive with those offered by industry. It will also lead to the development of standards for regenerative medicine — such as replacing, engineering, or regenerating human cells, tissues, or organs to restore or establish normal function.
Lawmakers are also considering adding the REGROW Act to the Senate’s package of bills. The bill would allow complex regenerative medicine therapies to be conditionally approved based on preliminary evidence. Since at least half of all drugs fail in the last stage of testing, many patients could end up receiving therapies that are later found to be unsafe or ineffective.
On Saturday June 25, six former FDA commissioners from Democratic and Republican administrations suggested at the Aspen Ideas Festival that Congress make the agency independent of the Department of Health and Human Services — similar to the Securities Exchange Commission, for example. With regulatory purview over products that represent a quarter of the U.S. economy, the group said the FDA is harmed by an unstable federal budget process and persistent political meddling. The group said they would issue a white paper on their proposal for the next administration. That’s another reason why Congress should postpone consideration of these bills until 2017.
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