Discarding efficacy?

Andrew Grove, former CEO of Intel, writes an editorial in Science:

We might conceptualize an “e-trial” system along similar lines. Drug safety would continue to be ensured by the U.S. Food and Drug Administration. While safety-focused Phase I trials would continue under their 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.

Alex Tabarrok of Marginal Revolution (who is a big advocate for FDA reform, running this site) really likes the idea. I hate it. While the current system has some problems, Grove’s system would be much, much worse than the current system. The biggest problem is that we would have no good data about whether a drug is truly efficacious, because all of the results in the database would be confounded by selection bias. Getting a large sample size and having subgroups tells you nothing about why someone got the treatment in the first place.

Would physicians pay attention to peer-reviewed articles and reviews identifying the best treatments for specific groups? Or would they just run their own analyses? I think there would be a lot of the latter, which is scary since many clinicians can’t even define selection bias or properly interpret statistical tests. The current system has limitations, but Grove’s idea would move us even further from any sort of evidence-based medicine.

Other commenters at Marginal Revolution rightly note that it’s difficult to separate safety from efficacy, because recommending a drug is always based on a balance of risks and benefits. Debilitating nausea or strong likelihood of heart attack would never be OK in a drug for mild headaches, but if it cures cancer the standards are (and should be) different.

Derek Lowe, a fellow Arkansan who writes the excellent chemistry blog In The Pipeline, has more extensive (and informed) thoughts here.

Update (1/5/2012): More criticism, summarized by Derek Lowe.

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