FDA Bulletin, April 1982

The appropriateness or the legality of prescribing approved drugs for uses not included in their official labeling is sometimes of concern and confusion among practitioners.

Under the Federal Food, Drug, and Cosmetic (FD&C) Act, a drug approved for marketing may be labeled, promoted, and advertised by the manufacturer only for those uses which the drug’s safety and effectiveness have been established and which FDA has approved. These are commonly referred to as ‘approved uses.” This means that adequate and well-controlled clinical trials have documented these uses, and the results of the trials have been reviewed and approved by the FDA.

The FD&C Act does not, however, limit the manner in which a physician may use an approved drug. Once a product has been approved for marketing, a physician may prescribe it for uses or in treatment regimens or patient populations that are not included in approved labeling. Such “unapproved” or, more precisely, ‘unlabeled’ uses may be appropriate and rational in certain circumstances, and may, in fact, reflect approaches to drug therapy that have been extensively reported in medical literature.

The term “unapproved uses” is, to some extent, misleading. It includes a variety of situations ranging from unstudied to thoroughly investigated drug uses. Valid new uses for drugs already on the market are first discovered through serendipitous observations and therapeutic innovations, subsequently confirmed by well-planned and executed clinical investigations. Before such advances can be added to the approved labeling, however, data substantiating the effectiveness of a new use or regimen must be submitted by the manufacturer to FDA for evaluation. This may take time and, without the initiative of the drug manufacturer whose product is involved, may never occur. For that reason, accepted medical practice often includes drug use that is not reflected in approved drug labeling.

With respect to its role in medical practice, the package insert is informationalonly. FDA tries to assure that prescription drug information in the package insert

accurately and fully reflects the data on safety and effectiveness on which drug

approval is based.

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