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Congress to begin voting on sweeping biomedical innovation bill

Congress is poised to begin voting on a sweeping biomedical innovation bill that includes nearly $5 billion in dedicated funding for a trio of major research initiatives at the National Institutes of Health (NIH). The bill also includes measures to speed the approval of new drugs and medical devices by the Food and Drug Administration (FDA), and it would create a mechanism for catalyzing efforts to streamline federal regulations that universities and academic researchers regard as burdensome.

The bipartisan bill, known as the 21st Century Cures Act, is the culmination of more than 2 years of lobbying by research, patient, and industry groups, and extensive negotiations between members of the Senate and the House of Representatives. The House is expected to approve the bill Wednesday. Its fate in the Senate is unclear, with some Senators objecting to funding mechanisms and other provisions. But late on Tuesday lawmakers announced changes to the bill designed to win over skeptics. Senate Majority Leader Mitch McConnell (R–KY) has said completing Cures and sending it to President Barack Obama for signing is one of his highest priorities before Congress adjourns for the year, and the White House has said it supports the bill.

Biomedical research groups are cautiously optimistic that Cures will finally cross the finish line. The bill includes a long list of largely uncontroversial provisions, including calls for NIH to produce a comprehensive strategic plan, set up a special initiative for young scientists, establish a prize to incentivize certain kinds of research, and take new steps to encourage data sharing and ensure the reproducibility of NIH-funded research. And research lobbyists are delighted with provisions that set aside $4.8 billion over the next 10 years for three NIH initiatives: $1.4 billion for Obama’s Precision Medicine Initiative, $1.8 billion for Vice President Joe Biden’s cancer moonshot, and $1.6 billion for the White House’s Brain Research through Advancing Innovative Neurotechnologies initiative. The bill also provides $30 million over 3 years for regenerative medicine research using adult stem cells.

 Funding concerns

The NIH total is less than the $8.8 billion envisioned in earlier versions, but research advocates aren’t complaining. They say Cures would put Congress on record as supporting sustained funding for key research areas at NIH, which currently spends more than $30 billion annually, and specifies how the spending boost would be paid for. “We’re absolutely thrilled,” says Jon Retzlaff, director of science policy in the Washington, D.C., office of the American Association for Cancer Research, which has lobbied for Biden’s cancer moonshot.

A 25 November Cures draft calls for raising the money by selling oil from the U.S. Strategic Petroleum Reserve and redirecting funds from a public health fund established by the Affordable Care Act (Obamacare). The money would flow into a new NIH Innovation Account that congressional appropriators would control. But that plan has drawn opposition from some Senators, including those concerned about the public health programs, and is a source of angst in the biomedical research community. The arrangement disappoints those who sought a dedicated stream of money for NIH not subject to the vagaries of the annual appropriations process, but satisfies some lawmakers’ desire to keep Congress in control of spending.

“Our hope is that this fund will add a layer of security” for NIH’s budget, says Tannaz Rasouli, a policy specialist at the Association of American Medical Colleges in Washington, D.C. But “there are no guarantees,” warns Howard Garrison, a policy expert at the Federation of American Societies for Experimental Biology (FASEB) in Bethesda, Maryland. He and others want Congress to use the fund as a supplement to NIH’s regular budget, but fear it could become an excuse to curb spending in other parts of the agency. Garrison also worries about other provisions that could burden NIH. In a bid to boost accountability, for instance, Congress wants the director of each institute to personally review and sign off on grants. “It’s awkward, it’s unnecessary, and it’s burdensome,” Garrison says. And FASEB worries a plan to impose renewable, 5-year term limits on institute directors could make it harder for NIH to recruit talent.

New Research Policy Board

Getting a warmer welcome are provisions aimed at reducing the regulatory burden on institutions that get federal research dollars. Reporting requirements on grants have grown over the past few decades, for instance, as have rules meant to safeguard research subjects. The bill creates a Research Policy Board within the White House Office of Management and Budget. The board, which would be made up of representatives from up to 10 federal agencies and a similar number of research institutions, would study emerging regulatory problems as well as recommend how to harmonize existing policies.(For more on the research board, read this ScienceInsider story.) The idea came from a 2015 report by a committee of the National Academies of Sciences, Engineering, and Medicine. Another provision calls for a review of 5-year-old rules that tightened conflict of interest reporting by NIH-funded researchers. Institutions complain the rules have imposed big costs with little benefit.

The bill’s FDA provisions aim to accelerate the agency’s review of some new drugs and medical devices. In certain cases, FDA would allow companies to run smaller clinical trials or rely on evidence collected outside of trials to support approval. In an apparent bid to boost stem cell and other experimental therapies, the current draft also directs FDA to give special attention to treatments designated as “regenerative advanced therapy.” If a treatment meets the criteria—for example, if it is based on stem cells or other tissues and addresses an unmet medical need—regulators can offer a company faster review, or more flexibility in setting trial endpoints.

A set of potentially controversial provisions would expand FDA’s priority review system, which attempts to create an incentive for companies to develop drugs for neglected tropical and pediatric diseases by doling out tradeable vouchers that entitle the companies to speedier agency reviews. Cures would add medical countermeasures—designed to respond to a chemical or biological attack, for example—to the treatments that would win vouchers for their developers. But critics say there is little evidence the vouchers have worked, and worry that creating even more of them will only reduce their potential value.

If Cures stalls in the Senate, where critics have argued it gives too much leeway to the drug industry, backers fear it could be a lengthy wait for another chance to lock in NIH funding and tweak FDA rules. The bill, says Representative Fred Upton (R–MI), a leading sponsor, is a “once-in-a-generation, transformational opportunity to change the way we treat disease.