Think Tank: Exploring Novel Trial Designs and Innovative Statistical Tools in Rare Disease Drug Development

Duke-Margolis, in cooperation with the U.S. Food and Drug Administration, convened a small workshop focused on finding solutions to some of the challenges rare disease settings pose for treatment development and regulatory decision-making. Invited experts explored topics such as the use of external control data in single-arm trials, utilization of early phase data in phase three trials, and best practices for leveraging adaptive study designs. This workshop is the second in a series of small workshops tackling development challenges in rare disease settings.