The United States can double the number of innovative new medical treatments available in the next 10 or 15 years, but only if important changes are made to how drugs are developed and approved, says a White House report . That was the message in a study released today by the President's Council of Advisors on Science and Technology (PCAST). Speaking at a meeting this morning where the recommendations were released, Margaret Hamburg, commissioner of the U.S. Food and Drug Administration (FDA), said: "Our job really is not complete until we have significant impact on the health of patients and the greater public here in this country and around the world. And I think this report helps move us toward that goal."
PCAST is an illustrious group: Its 21 members include co-chair Eric Lander, president of the Broad Institute in Cambridge, Massachusetts; Eric Schmidt, the executive chair of Google; and the presidents of Yale University and Rensselaer Polytechnic Institute. The changes urged by the group are substantial and would require both additional funding for research and regulation, and possibly action by Congress to grant FDA more leverage. Among other things, the PCAST report argues that FDA ought to make greater use of a special track called accelerated approval, in which drugs can be given a green light based on limited information. Most therapies now granted accelerated approval are cancer drugs, and companies are required to continue large trials of the therapies after approval and report back on results to ensure that products work as expected. (Indeed, one cancer drug, Avastin, recently had its approval for breast cancer therapy revoked  after studies showed it didn't work as effectively as thought.) PCAST suggests that FDA expand this fast track to include other serious diseases and think creatively about measures to assess a drug's effectiveness in this setting. At the same time, the PCAST report admits that such new measures, typically called surrogate markers, represent an "educated guess" about whether a drug will help.
Another way to speed drugs to market, PCAST suggests, would be to limit newly approved drugs to narrow populations of patients. Right now, any therapy that's for sale can legally be used to treat any disease (although it may not be covered by insurance or by federal programs like Medicare). For example, the report asks, what if a weight loss therapy could be limited to patients only with morbid obesity, not those who are only 10 pounds overweight? "Drug sponsors could propose faster and smaller clinical trials for initial market approval," the PCAST authors write, because the people using the drug would be those at greatest risk of health problems or death. The committee proposes a "special medical use" label that would urge physicians to prescribe the drug only to those for whom it's designed. Changes like this, the authors acknowledge, would make it more critical for FDA to have a strong postmarketing surveillance system. And for that, FDA would need something that's very hard to get these days -- more funding.
The committee's members also urge new economic incentives for drug development and more collaboration among stakeholders, including academics, companies, and patient advocacy groups.