Adaptive Clinical Trial Design: An Overview and Potential Applications in Dermatology

Abstract

The challenges of drug development, including increasing costs, late-stage drug failures, and the decline in the number of drugs being approved by the US Food and Drug Administration over time, have generated interest in adaptive study designs that have the potential to address these problems. Adaptive trial designs use interim data analysis to amend trials, and have been recognized for more than a decade as a way to increase trial efficiency, partly by the increased probability of demonstrating a drug effect if one exists. In this article, we define adaptive trials; give examples of the most common types; highlight the pros, cons, and ethical considerations of these designs; and illustrate how these tools can be applied to drug development in dermatology.