Chair: Ginger Haynes, PhD

In a number of diseases that are primary CNS clinical research targets, such as MDD and schizophrenia, there are a substantial number of effective medications already available globally, many at generic prices. Superiority to placebo is often the threshold for regulatory approval; however, patients, physicians, and payers are interested in how newer medications compare to those already in existence. Historically, the studies to generate answers to these important questions would lag regulatory approval for years, if they were executed at all. One method to explore comparative effectiveness is to identify a targeted subpopulation in which a new agent demonstrates advantage over standard of care. Adaptive population enrichment designs can efficiently identify subpopulations in which newly approved medications are superior to other medications. The ISCTM Adaptive Design Working Group will walk through an adaptive population enrichment design for a Phase IIIB/IV study in a CNS disorder. During the workshop, the group will discuss the performance of the design via simulation and the business and practical implications of various design element decisions.