The authors – CM Wittich, CM Burkle, and WL Lanier – offer a concise review of the topic of off-label drug use including its definition, prevalence, and implications for drug safety. The article format addresses 10 common questions and their answers about off-label drug use. The breadth of application, its acceptance, and the liabilities of off-label use are explored. A history of FDA regulations surrounding the practice is presented, which helps to put its evolution into proper perspective. Off-label use, which occurs in every medical specialty, is more common in patient populations not likely to be included in clinical trials (e.g., pediatric, pregnant, or psychiatric patients). Once a medication is marketed, the FDA does not limit or control how the medication is prescribed by physicians. The pros and cons of the distribution of information regarding the off-label use of medications by pharmaceutical companies, the use of informed consent, and the liability of prescribing physicians are discussed.
Source: Mayo Clinic Proceedings – pdf of full article.