Post-marketing studies of drug efficacy: why?

In order to quantify the need for post-marketing studies of drug efficacy, the 100 drug uses (i.e., drug-indication pairs) that were most common in 1978 were reviewed. Of the 100 most common drug uses, 31 were not Food and Drug Administration (FDA)-approved at the time of initial marketing and 18 had not become FDA-approved, even subsequently. In 13, the use was based on the drug's secondary effect rather than on the primary effects investigated before marketing. The efficacy of all was subject to modification by incompletely explored factors, including other drugs, patient age, other illnesses, pregnancy, etc. Specific examples are provided of important modifiers of the efficacy of these drugs that were not investigated prior to marketing. Thus, despite the existence of sufficient data for the regulatory process to permit the marketing of these commonly used drugs, considerable gaps remain in the information needed for their optimal clinical use. Post-marketing studies of drug efficacy will be needed to fill these gaps.