France's New Framework for Regulating Off-Label Drug Use

Abstract

Off-label use of drugs is relatively common in medical practice, even if it's often not supported by strong scientific evidence. Studies in the United States have shown that off-label use may account for approximately 20% of prescriptions, or 150 million prescriptions per year.¹ In addition to its economic effect on the health care system, the inappropriate off-label use of drugs raises major concerns about safety, especially when a drug is widely used regardless of the fact that regulatory agencies have not determined its benefit–risk ratio. In France, a recent scandal involving Mediator (benfluorex) — a fenfluramine derivative approved for treating diabetes but prescribed for indications (weight loss) not included in the “summary of product characteristics” (label), which caused sometimes-fatal valvular heart disease — paved the way for major changes in the regulatory system, including new measures aimed at strongly regulating access to drugs during their postmarketing “real life.”²