Adaptive Clinical Trials

Scott S. Emerson, MD, PhD

November, 2010

Abstract: Increasingly, clinical trials are conducted using group sequential methods and, more recently, adaptive clinical trial designs in order to address the ethical and efficiency issues that arise when performing experiments with human volunteers. The design, conduct, and analysis of a sequential clinical trial is necessarily more involved than that for a clinical trial in which the data would only be analyzed at the end of the study. However, the basic tenets of careful consideration of the scientific question and clinical setting remains the same. In this short course we present an overview of the issues that must be considered when conducting any sequential trial, and contrast the more commonly used group sequential approach to the various forms of adaptive designs that have been described in the statistical literature.