This story is the ninth in ScienceInsider’s After Election 2014 series. Through Election Day on 4 November, we will periodically examine research issues that will face U.S. lawmakers when they return to Washington, D.C., for a lame-duck session and when a new Congress convenes in January. Click here to see all the stories published so far; click here for a list of published and planned stories.
Today, a look at the 21st Century Cures Initiative, which is expected to produce legislative proposals to improve biomedical research and innovation in the new Congress that convenes in January.
Can a congressional odd couple successfully shake up the complex system that transforms research discoveries into new drugs and treatments?
It’s a question that has been rippling through biomedical research circles in recent months, as an unlikely pair of allies—conservative Representative Fred Upton (R–MI) and the relatively liberal Representative Diana DeGette (D–CO)—have pursued a project they’ve dubbed the 21st Century Cures Initiative. The goal: to speed up the lengthy and costly process of developing new treatments for disease.
So far, the 6-month effort has involved more than a dozen hearings and roundtables on Capitol Hill, plus other listening sessions outside of Washington, D.C. Lawmakers have heard from an all-star list of witnesses, including Francis Collins, director of the National Institutes of Health (NIH), senior officials at the Food and Drug Administration (FDA), and advocacy group leaders.
21st Century Cures won’t produce its first major product, however, until after the new Congress takes office in January. That’s when Upton and DeGette—the top Republican and a senior Democrat, respectively, on the U.S. House of Representatives Committee on Energy and Commerce—are expected to unveil draft legislation designed to put the talk into action.
Biomedical research advocates are watching closely. “I think this effort has really gotten everybody’s attention,” says Margaret Anderson, executive director of FasterCures, a think tank in Washington, D.C. “It’s given everyone an outlet to start to funnel their thinking about what’s going on in medical research.”
“I think this is the first time in quite a while where the community has been asked to try and identify specific things that they think Congress can do,” says David Moore, senior director of government relations at the Association of American Medical Colleges, also in Washington, D.C.
Exactly what the bill will call for, however, remains something of a mystery. The hearings and roundtable talks have produced an unwieldy list of insights that Moore describes as “95% diagnosis and only about 5% proposed therapy. … What I haven’t heard—and I think what we’re all struggling with—is what are specific actions that Congress can take.”
Still, staffers on the Energy and Commerce Committee tell ScienceInsider that the listening tour has helped identify some potential targets for streamlining clinical trials and the drug approval process. For instance, the new bill is likely to propose changes to the system of institutional review boards (IRBs) that approve clinical trials. Now, trials that enroll patients at more than one location—such as at several university hospitals—must get IRB approval at each site. Critics say that’s time-consuming and inefficient and would like to see Congress change the rules to create incentives to encourage centralized IRB approvals for multisite trials.
The legislation will also likely take aim at the way FDA evaluates biomarkers—the physiological measurements that indicate whether a drug is acting on its molecular target in the body. Reliable biomarkers could save time and money by serving as surrogates for other clinical trial endpoints that take longer to measure, such as survival or disease progression. But proposals to use biomarkers have created a deadlock in the drug review process: FDA reviewers aren’t confident that the biomarkers submitted in new drug applications are reliable, committee staff members say, while drug sponsors are uncertain that these tools will help them win approval. So the new legislation would set standards for how the agency should evaluate new biomarkers as they are developed.
At the hearings, witnesses have dropped other hints about what they’d like to see in the bill. During a 10 September discussion, for instance, Collins suggested “it would be nice” to end a rule that prevents NIH’s National Center for Advancing Translational Sciences from supporting research beyond phase IIa of clinical testing. FDA Commissioner Margaret Hamburg suggested amending the Food, Drug, and Cosmetic Act so that clinical trials for medical devices no longer require review by a local IRB. (“Done!” Upton replied.) And Moore says Congress is in a good position to help reduce costly and burdensome federal regulations that universities claim are stifling research.
It is possible that lawmakers may use the bill to address another perennial concern—how NIH allocates funding to different disease areas. “There seems to be a real lousy correlation between where we’re spending our dollars and disease burden,” Representative Bill Cassidy (R–LA) told Collins at one roundtable, suggesting that the agency commits too much funding to AIDS research and not enough to Alzheimer’s, relative to the projected impact of the two diseases. Collins defended the scientific reasoning behind NIH’s spending priorities, but also repeated a point made at almost every 21st Century Cures event: “I promise you, this is not just researchers with their hands out, saying ‘Leave us alone.’ We understand the obligation to be incredibly efficient and creative about what we do.”
Even if a 21st Century Cures bill eventually becomes law—and that could be a long process—it would have only a limited impact on agency budgets. That’s because it will be an authorization bill, which can set policy but only suggest funding levels; final spending decisions are up to Congress’s appropriations committees.
Still, biomedical advocates hope the process will help them make the case for research funding and other reforms. If nothing else, Anderson says, the 21st Century Cures process has “offered up the possibility of more holistic conversation about these issues.”