Off-label or unlabeled drug use is the use of a drug approved by the U.S. Food and Drug Administration (FDA) for other uses or in treatment regimens or patient populations that are not included in approved labeling.
The FDA approves drugs for specific indications that are included in the drug’s labeling. When a drug is used for an indication other than those specifically included in the labeling, it is referred to as an off-label use. Many off-label uses are effective, well-documented in the literature, and widely used.
Unapproved or unlabeled uses of drugs include a variety of situations ranging from completely unstudied to thoroughly investigated drug uses where the FDA has not been asked for approval, whereas approved uses of drugs have been shown to be safe and effective by the FDA after the review of adequate and controlled clinical trials that have documented their uses.
A compendium is a comprehensive listing of FDA-approved drugs and biologics. In some cases, compendia specialize in a particular subset of drugs, such as those used for anti-cancer treatment. Compendia include a summary of how each drug works in the body, as well as information for health care practitioners about proper dosing and whether the drug is recommended or endorsed for use in treating a specific disease. A compendium may be used as one of several tools to determine whether a drug should be covered.