Off-Label Drug Risks: Toward a New FDA Regulatory Approach

Abstract

Most off-label uses of prescription drugs are not supported by strong clinical evidence, leading to millions of patients being exposed to poorly understood risks without well-established benefits. The FDA’s approach to limiting these risks has been to bar manufacturers from promoting their drugs for off-label uses. But this strategy has had only limited success and is threatened by courts’ increasing willingness to extend First Amendment protections to drug makers’ speech.

This Article proposes that the FDA should shift its focus from off-label promotion toward off-label prescribing. The Agency should provide information on drug labels about the strength of the evidence that supports common off-label drug uses. Leveraging the FDA’s unique ability to gather and analyze information and physicians’ role as learned intermediaries could alter prescriber practices to reduce the rate of unsupported off-label drug uses.

Keywords: FDA, Off-Label, Drug Label

JEL Classification: 118