The purpose of this guidance is to assist industry in developing enrichment strategies that can be used in clinical investigations intended to demonstrate the effectiveness of drug and biological products. Enrichment is the prospective use of any patient characteristic to select a study population in which detection of a drug effect (if one is in fact present) is more likely than it would be in an unselected population. Although this guidance focuses on enrichment directed at improving the ability of a study to detect a drug’s effectiveness, similar strategies can be used in safety assessments.
The enrichment strategies described in this guidance are intended to increase the efficiency of drug development and support precision medicine, i.e., tailoring treatments to those patients who will benefit based on clinical laboratory, genomic, and proteomic factors. This guidance also discusses design options for enrichment strategies and discusses the interpretation of the results of studies that use enrichment strategies.