Aug. 13, 2015
by Rachel Schulze, staff writer
Lawmakers this session are looking to boost biomedical research and help patients access treatment.
The 21st Century Cures Act (HR 6) so far has overwhelming support from lawmakers. In July, the House voted 344-77 to approve it.
However, concerns about the practical effects of the measure surfaced after the House passed it. Could that criticism seal the bill's fate?
Before getting into the criticism, here's an overview of the Cures legislation.
What It Does
The legislation addresses a range of issues, but mostly targets two areas:
- Federal funding for biomedical research; and
- Regulation of drugs and medical devices.
Under the bill, NIH would receive $8.75 billion in additional funding over the course of five years from a temporary "innovation fund."
The bill also calls for codifying a system for submitting, reviewing and qualifying biomarkers -- which can offer predictions about a treatment's effect earlier than a long trial -- and other drug development tools in drug reviews.
In addition, the bill could grant some antibiotics expedited review under certain conditions. Further, FDA would have to create a priority review for breakthrough medical devices. The bill also would help provide incentives for the development of treatments for rare diseases, known as orphan drugs.
The bill also instructs the HHS secretary to create a program for evaluating the potential of using clinical experience evidence for supporting approving new uses for already-approved drugs.
FDA would receive $550 million over five years and direct the agency to implement several mandates pertaining to drug and device approvals, as well as patient engagement.
Funding Could Provide More Certainty, Efficiency
The funding would come after years of budget stagnation at NIH.
Stuart Schweitzer, a professor of health policy and management at the UCLA Fielding School of Public Health, said the bill would give NIH more certainty when making decisions about research commitments. "Most biomedical research takes a fair amount of time, and you can't fund certain kinds of projects if you don't know that you're going to have the money next year or the year after."
"I think the 21st Century Cures Act, at least as it relates to FDA, is viewed as an opportunity to make [the review] process more streamlined and more efficient," Avalere Director Sung Hee Choe said.
Choe said if the bill passes, FDA could face hurdles implementing its mandates.
"I think, on the more challenging side, really is going to be how FDA manages or balances all of the responsibilities that are outlined for them within the bill on top of the responsibilities that they already have," she said.
Meanwhile, the measure could face financing questions. The bill would be funded through sale of crude oil from the Strategic Petroleum Reserve.
While moving through the House, Rep. David Brat (R-Va.) proposed an amendment (HA 656) that would have made funding discretionary, and subject to annual appropriations, rather than mandatory. Although the amendment failed, 141-281, funding could be an issue moving forward.
Further, the Obama administration in a policy statement said it "welcomes the additional support for biomedical research and innovation included in" the bill but "has concerns about providing additional funding for [NIH] and FDA without addressing sequestration more broadly."
Patient Safety Problems
The bill, namely its mandates for FDA regulations, has faced criticism for its potential implications for patient safety.
National Center for Health Research President Diana Zuckerman said, "We share Congress' desire to increase funding for NIH, but there are dangerous provisions in this bill that undermine scientific evidence used to approve medical products."
Writing in the New York Times, Rita Redberg, a cardiologist at the University of California-San Francisco Medical Center, and Sanket Dhruva, a cardiologist and clinical scholar at Yale University, raised concerns about the bill's medical devices provisions. They write that the bill "would make it possible for companies that produce high-risk medical devices to submit evidence of safety and efficacy based on sources other than clinical trials, including case histories," adding, "In other words, anecdotal evidence, rather than the scientific studies, could be used to approve devices."
Continuing Beyond Cures
At the same time, some are calling for lawmakers to go beyond the Cures bill.
For example, a report by the Bipartisan Policy Center lists a series of recommendations on drug development, data sharing, health IT interoperability, regulatory authority and other topics, highlighting which are included in the Cures bill and which are not. The authors note that there are 10,000 known diseases but just 500 treatments, adding, "The extraordinary cost of care for those for whom neither cures nor effective treatments are available threatens to overwhelm America's social safety net and significantly constrain the capacity to address equally pressing challenges at home and abroad."
The Senate Health, Education Labor and Pensions Committee Chair Lamar Alexander (R-Tenn.) said his committee has been working on an effort to parallel the House legislation. However, the committee has not yet put out a bill.
In terms of what could appear in a Senate version, Choe said, "I think that there is similar perspective in terms of the objective and the desire to stimulate more investment in the development of new treatment. I think we could see a lot of things that are parallel, so boosts to the NIH funding as well as certain areas within the FDA." Choe added that Alexander and Rep. Richard Burr (R-N.C.) earlier this year released a white paper, "Innovation for Healthier Americans," which could offer some insight about the Senate's priorities.
Schweitzer said the bill's NIH funding boost "is a clear winner, I think, politically," adding, "I could imagine the political statement that somebody could make: 'Well, my opponent voted against funding for NIH.' And gosh, that sounds like a pretty powerful political argument. Now, of course the story is a lot more complicated than that."
Meanwhile, the FDA changes might face more resistance in the Senate, Schweitzer said. Schweitzer described the situation regarding FDA regulation as a "double-edged sword." He said, "[W]hat we're really talking about is a tradeoff. How safe and effective do we want drugs and device, and how quickly do we want them? Because those two objectives, in fact, conflict with one another."