Representatives Fred Upton (R–MI) and Diana DeGette (D–CO) last year made a video promoting their 21st Century Cures initiative.

Representatives Fred Upton (R–MI) and Diana DeGette (D–CO) last year made a video promoting their 21st Century Cures initiative.

Offices of representatives Fred Upton and Diana DeGette

Sweeping plan to revamp biomedical innovation includes controversial ideas for NIH

A U.S. House of Representatives panel today released a widely anticipated proposal for speeding the development of new medical treatments. The massive, 393-page document, dubbed the 21st Century Cures Act, has been under development by Fred Upton (R–MI) and Diana DeGette (D–CO) of the House Energy and Commerce Committee since last April. It grew out of a series of hearings and roundtables last year on ways to spur biomedical research innovation and streamline clinical trials and the drug approval process. The result is a long list of ideas summarized in this document.

The document is a "discussion draft," the committee said in a statement. Upton has invited comments via the Twitter handle #Cures2015. “Some things may be dropped, some items may be added, but everything is on the table as we hope to trigger a thoughtful discussion toward a more polished product,” he said in a statement.

DeGette, in a statement, said that she appreciated Upton's effort "to publicly share possible legislative language on a number of proposals that might be included in an eventual bill. … While I don’t endorse the draft document, I know that with continued engagement, we can reach a bipartisan consensus to help advance biomedical research and cures.”

Many parts of the draft aim to persuade drug developers to focus on diseases for which there are few treatments. One section, dubbed the Dormant Therapies Act, takes aim at drugs for complex diseases, such as Alzheimer’s, that are particularly time-consuming to develop and test. By setting a fixed period of patent exclusivity after such treatments win approval (and not when the drug is first identified), the bill would give drug developers more time to recover costs and make profits. Other provisions would extend the period of patent exclusivity on drugs to treat rare diseases, for novel antibiotics, and for improvements to an existing drug—such as a new formulation that reduces side effects.

The draft proposal also aims to speed clinical trials and incorporate new kinds of data into the U.S. Food and Drug Administration’s evaluation process. The agency would be required to set standards for how it will evaluate “surrogate endpoints”—measures of a patient’s biological response to a drug that can indicate benefits more quickly than measures such as survival and disease progression. It would also require the agency to develop a new process for incorporating patient feedback into the evaluation of a new treatment.

The draft also proposes a 21st Century Cures Consortium, involving federal agencies, drug companies, patient groups, and other stakeholders, which would work together on ways to get drugs to patients faster. National Institutes of Health (NIH) grantees would be required to share data, and data from health care facilities would be made available for research.

Some provisions aimed at NIH, which funds most of the nation’s basic biomedical research, were suggested by the agency. For instance, NIH’s drug development branch, the National Center for Advancing Translational Sciences, would no longer be prohibited from funding clinical trials beyond the stage known as phase IIA.

Other proposals from Representative Andy Harris (R–MD) may be more controversial. For example, the draft calls for setting aside a portion of NIH’s Common Fund to fund proposals from early-career scientists. Although that is a scaled back version of an earlier Harris plan that would have required that NIH bring down the average age of a first-time grantee, it could still draw concerns about such a set-aside. Harris also wants NIH to develop an overarching strategic plan. That idea flopped when the agency last attempted it more than 2 decades ago.

Other sections remain to be filled in, including one on “precision medicine,” the topic of an initiative announced by President Barack Obama in his State of the Union address last week.

Biomedical research advocacy groups are offering praise. The draft “represents a promising opportunity,” said Carrie Wolinetz, president of the umbrella group United for Medical Research, in a statement. “Medical research funding has stalled over the past decade. … The 21st Century Cures initiative will help restart its engine.”

“The Initiative could be a game changer for the medical innovation ecosystem,” said Mary Woolley, the president and CEO of the advocacy group Research!America, in a statement. “Among the many beneficial provisions Research!America fought for is a measure to reduce the administrative burden on researchers,” she noted. (Research universities have long complained about regulatory burdens.)

The approach is also attracting interest in the U.S. Senate. A statement from senators Lamar Alexander (R–TN) and Patty Murray (D–WA), the top Republican and Democrat, respectively, on the Senate’s health committee, noted that one of their committee’s top priorities is also “modernizing the Food and Drug Administration, helping to get treatments and medicines to patients as quickly and as safely as possible.”

The committee now plans to collect comments on the draft, with an eye to pushing the legislation forward later this year.

*Update, 27 January, 5:31 p.m.: This story has been updated to clarify that the document is not a formal legislative proposal.

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