Keywords: Adaptive Design, Enrichment, Subgroup, Guidance, Case study
By incorporating the population enrichment feature into the adaptive design frameworks, adaptive enrichment designs typically allow the investigator to modify the enrollment criteria at the interim looks based on subgroup efficacy analysis according to some baseline characteristics (e.g. biomarkers, risk scores, age groups). Adaptive enrichment designs may provide opportunities and evidence to identify which subpopulations benefit from the investigational product, especially when benefit for overall population is not expected. In this talk, we will review up-to-date regulatory guidances involving adaptive enrichment designs. Current statistical methodologies of adaptive enrichment designs as well as their strengths and limitations will also be discussed. A couple of case studies will be presented to illustrate the regulatory applications of adaptive enrichment designs.