Regulating Off-Label Prescribing: The Good, the Bad and the Uncertain

Abstract

Off-label prescribing is a double-edged sword that offers both benefits and risks depending on a range of different factors such as whether the off-label use is new or well-established; the strength of the evidence supporting the off-label use; the urgency of the patient’s situation; and the availability of alternative treatment options. In this memorandum, prepared for a expert witness hearing of the Canadian Standing Senate Committee on Social Affairs, Science and Technology for its study of Prescription Pharmaceuticals in Canada, we discuss the rationale for permitting off-label prescribing and the problems associated with the practice in the Canadian context. We argue that reform efforts should focus on improving the monitoring of off-label use of drug products and in promoting an expanded post-market research agenda for off-label prescribing. In particular, better and more systematic adverse event reporting is essential for a proper assessment of the risks and benefits of medication use. Improving our knowledge of patient outcomes from off-label prescribing in real-world settings may support new beneficial uses of drug products, but will also help to prevent harmful off-label use. Supporting prescribers with access to timely and accurate information on off-label use of drugs will also help to ensure that off-label prescribing decisions are not made off-the-cuff.