Confirmatory Enrichment Design: Adequate and Well-Controlled Trials with Population Selection

• Activity Number: 339
• Type: Topic Contributed
• Date/Time: Tuesday, August 6, 2013 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Scott M. Berry*+
• Companies: Berry Consultants
• Keywords: Enrichment Designs ; Adaptive Designs ; Bayesian Statistics ; Trial Simulation

Abstract

An enrichment design is one that addresses the patient population allowing the possibility that adaptive decisions can winnow the population to the subgroup that benefits from the treatment. This enables smaller more efficient trials to be conducted in the appropriate population. This also insures that subjects that are likely to benefit from the treatment will be treated -- those that will not will not be enrolled.

This talk addresses the use of enrichment designs in a confirmatory setting. The trial may start with the largest population studied, but as the trial progresses the population may be winnowed down to those most likely to respond. This is all done in an adequate and well controlled trial. The general concept will be discussed as well as specific (real) examples.