An ambitious effort in the U.S. House of Representatives to jump-start biomedical innovation took another step forward today with the release of a bipartisan draft bill. The so-called 21st Century Cures Act contains huge news for supporters of biomedical research: It recommends substantial budget increases for the National Institutes of Health (NIH), including $10 billion in extra funding over 5 years. Other provisions aimed at speeding the drug approval process at the Food and Drug Administration (FDA) are mostly unchanged from an earlier version, but some incentives for drug developers have been removed.
Although the call for increased NIH funding is aspirational—the bill can only recommend funding levels, not require congressional appropriators to provide the cash—it is still “some of the best news for NIH funding since 2003,” says Patrick White, president of Act for NIH, an advocacy group in Washington, D.C. (A big one-time budget increase as part of the 2009 to 2010 stimulus funding was another high point, he notes.)
United for Medical Research, a Washington, D.C.–based coalition of research, patient, and industry groups, commented that a $10 billion increase would “help put the NIH on a sustainable growth path and ensure the United States remains the world’s medical innovation leader.” Other provisions affecting NIH have drawn concern, however, and the American Society for Biochemistry and Molecular Biology (ASBMB), based in Rockville, Maryland, calls the draft bill “a mixed bag.”
The bill, which focuses on changes to policies at NIH and FDA, has been under development for nearly a year, informed by a series of hearings and roundtables hosted by representatives Fred Upton (R–MI) and Diana DeGette (D–CO) of the House Energy and Commerce Committee. But an initial discussion draft, rolled out by Upton’s office in January, failed to win DeGette’s support. And NIH advocates were disappointed that the initial draft contained no new funding for NIH and that several provisions would dictate how the agency operates.
Today’s 199-page draft (half the length of the last version) is getting a warmer reception. The research community is thrilled that it would authorize $1.5 billion increases for the now-$30.3 billion agency in each of the next 3 years. That is $500 million more than the $1 billion (or 3.3%) raise requested by the White House for 2016.
In addition, the bill creates a new pot of money, called the NIH Innovation Fund, that would provide NIH with $2 billion more each year for 5 years. The money appears to be so-called mandatory funding, White says, which means it would not be part of the spending bills that Congress must approve each year. NIH supporters in Congress have been working for years to find a way to support NIH with mandatory funds so as to protect the agency from the uncertainty and ups and downs of annual appropriations.
The Innovation Fund would come with strings, however: NIH would have to use it for what the bill calls “young emerging scientists,” precision medicine, and a third unspecified category.
Given the current difficulties facing federal budgetmakers, it’s not clear whether Congress will be willing to go along with either the mandatory spending or the annual budget increases called for by 21st Century Cures. The annual NIH increases would have to be approved by House and Senate appropriations committees, which are now operating under tight spending caps. Those constraints “leave many of us wondering how these increases are mathematically possible,” said ASBMB Public Affairs Director Benjamin Corb.
The bill also still contains directives that some onlookers consider micromanaging. It requires NIH to develop an overall 5-year strategic plan; the Association of American Medical Colleges has called this directive unnecessary because NIH’s 27 institutes and centers already develop their own strategic plans. Another point of contention is a set-aside of funding for young investigators. Corb’s group says this preferential treatment will hurt midcareer and senior investigators.
ASBMB also dislikes a provision calling for NIH to create a “capstone award” for senior researchers who are ready to wind down their labs. When NIH floated this idea a couple of months ago, it drew a flood of harsh comments from scientists, many of whom saw it as cutting into the pool of funds for research grants.
“While the committee clearly took advice from the research community following its initial discussion draft, some provisions remain troubling,” Corb said.
FDA provisions tweaked
Many of the priorities the bill sets for FDA remain unchanged from the January document, but lawmakers did make some cuts. As in the earlier draft, the bill directs FDA to create a framework for incorporating patient feedback into its evaluation of new drugs and to issue new guidance on how it will evaluate new measures of a patient's biological response to a drug. Those priorities drew praise from Jim Greenwood, president and CEO of the Biotechnology Industry Organization in Washington, D.C. “We applaud the Committee’s recognition, in this draft, of the need for [FDA’s] involvement in validating and facilitating the use of cutting-edge drug development tools,” he said in a statement.
The draft also emphasizes the need for new antibiotics, establishes a specialized approval pathway, and increases Medicare reimbursement for much-needed antimicrobials—a possible revenue incentive for drug developers.
A section on precision medicine, which contained placeholder language in the previous draft, now calls for FDA to issue guidance on what qualifies as a “precision drug,” acceptable methods for identifying subsets of patients who might benefit from a new therapy, and how to design clinical trials around these limited populations.
But certain provisions designed as incentives for drug developers are notably absent from the new draft. Gone is a section that would offer 15 years of postapproval marketing exclusivity to drugs for complex diseases without effective treatments. Generous exclusivity provisions were one of the sticking points that led DeGette to withhold support for the previous draft, according to a representative from her office. Greenwood said he hopes “to work with the draft’s authors to ensure that additional incentives are included in either the next draft, or the final bill.”
The energy and commerce subcommittee on health will discuss the new draft at a hearing tomorrow morning. And a Senate committee is working on a similar bill and held a hearing this week to discuss therapeutic development challenges with FDA and NIH officials.