The term off-label drug use (OLDU) is used extensively in the medical literature,
continuing medical education exercises, and the media. Yet, we propose that many health
care professionals have an underappreciation of its definition, prevalence, and implications.
This article introduces and answers 10 questions regarding OLDU in an effort to clarify
the practice's meaning, breadth of application, acceptance, and liabilities. Off-label
drug use involves prescribing medications for indications, or using a dosage or dosage
form, that have not been approved by the US Food and Drug Administration. Since the
Food and Drug Administration does not regulate the practice of medicine, OLDU has
become common. It occurs in every specialty of medicine, but it may be more common
in areas of medicine in which the patient population is less likely to be included
in clinical trials (eg, pediatric, pregnant, or psychiatric patients). Pharmaceutical
companies are not allowed to promote their medications for an off-label use, which
has lead to several large settlements for illegal marketing. To limit liability, physicians
should prescribe medications only for indications that they believe are in the best
interest of the patient. In addition, health care professionals should educate themselves
about OLDU to weigh the risks and benefits and provide the best possible care for
their patients.
Copyright © 2012 Mayo Foundation for Medical Education and Research. Published by
Elsevier Inc. All rights reserved.