Statistics in Medicine has just published a special issue from the 2023 NHLBI Biostatistics Workshop on Clinical Trials Designs, guest edited by Colin Wu, James Troendle, and Dong-Yun Kim.

Clinical trials are essential for evaluating medical interventions, but it is an ongoing challenge to design trials that are both statistically sound and clinically relevant. This workshop brought together statisticians and clinicians to discuss two critical aspects of trial design: choice of endpoints and futility monitoring. These topics are pivotal in assuring that trials yield actionable insights while balancing the safety, effectiveness, and ethical considerations. This paper aims to capture the essence of the workshop’s panel discussion and synthesize the shared insights, with a focus on practical recommendations and open questions for future investigation.

The Workshop was divided into two major topics: choice of endpoints and monitoring for futility. A panel discussion after the talks allowed for discussion among the speakers on both topics, and the two combined.