In an important editorial in Science, Andy Grove, former Chief Executive Officer of Intel Corporation, advocates restricting the FDA to safety-only trials. Instead of FDA required efficacy trials patients would be tracked using a very large, open database.
The biomedical industry spends over $50 billion per year on research and development and produces some 20 new drugs….A breakthrough in regulation is needed to create a system that does more with fewer patients.
While safety-focused Phase I trials would continue under their [FDA] jurisdiction, establishing efficacy would no longer be under their purview. Once safety is proven, patients could access the medicine in question through qualified physicians. Patients’ responses to a drug would be stored in a database, along with their medical histories. Patient identity would be protected by biometric identifiers, and the database would be open to qualified medical researchers as a “commons.” The response of any patient or group of patients to a drug or treatment would be tracked and compared to those of others in the database who were treated in a different manner or not at all. These comparisons would provide insights into the factors that determine real-life efficacy: how individuals or subgroups respond to the drug. This would liberate drugs from the tyranny of the averages that characterize trial information today. The technology would facilitate such comparisons at incredible speeds and could quickly highlight negative results. As the patient population in the database grows and time passes, analysis of the data would also provide the information needed to conduct postmarketing studies and comparative effectiveness research.
Grove cites Boldin and Swamidass and especially Bartley Madden’s important book, Free to Choose Medicine, as inspiration for this proposal. By the way, I also recommend Madden’s book very highly (I am also proud to note my bias in this regard).
I have long advocated restricting the FDA to safety only trials (see, for example, FDAReview.org and my paper on off-label prescribing) and it seems that this idea, once considered outlandish, is now rapidly gathering advocates.
Addendum: Derek Lowe offers useful comments (see also Kevin Outterson below). One point that I think the critics miss is that nothing in Groves’s proposal and certainly not in Madden’s proposal which is somewhat different or in anything that I have advocated precludes randomized clinical trials for efficacy. Indeed, I strongly support such trials and have argued for greater funding so such trials can be done not by the pharmaceutical companies but by more objective third parties.