Off-label Drug Use and Adverse Drug Events: Turning up the Heat on Off-label Prescribing

Yelling “Fire!” in a crowded theater without any imminent danger is an oft-cited example of free speech that is not protected. For many years, the US Food and Drug Administration (FDA) has prohibited the promotion of unapproved uses of drugs. This restriction has recently been challenged under the guise of free speech. In a potential landmark decision in August 2015, a federal judge ruled against the FDA’s restrictions on off-label drug promotion, referencing protection by the First Amendment.¹ Critics of off-label drug promotion point to dietary supplements as an example of the kinds of claims that are commonplace when regulation is lax and worry about an erosion of the authority of the FDA to ensure safety and efficacy of drugs.² Are these concerns about safety warranted? Does evidence show that off-label prescribing might be less safe than on-label prescribing? In light of these concerns, the study of off-label drug use and adverse drug events by Eguale and colleagues³ in this issue of JAMA Internal Medicine is particularly timely.