Off-label prescribing: best practice or malpractice?

‘‘Labels’’ for medicines are also known as the ‘‘package inserts’’ that list the details of the approval granted by regulatory authorities such as the FDA (U.S. Food and Drug Administration) for the sale of medicines by the pharmaceutical industry. ‘‘Off-label’’ means utilizing an approved medicine in a manner not contained in the approved ‘‘label.’’ Although it is illegal for employees of the pharmaceutical industry to promote ‘‘off label’’ use of their medicine, it is not illegal for licensed practitioners to prescribe off-label. In fact, the majority of the practice of psychiatry, neurology, pain medicine, oncology, and of many other specialties whose clinicians practice in the real world (rather than in clinical trial settings) is off-label, and in one of five categories listed in Table 1. It has been said that more than 80% of psychiatric diagnoses have no FDA-approved medical treatments, a number that is likely to be even greater now with the recent launch of the new DSM 5 (Diagnostic and Statistical Manual of Mental Disorders of the American Psychiatric Association, 5th edition). Thus, it is more important than ever for prescribers to know the rules, the risks (Table 2), and the benefits (Table 3) of off-label prescribing.