Clinical Trials for Personalized Medicine: Design and Data Analysis

Abstract

In this article, we provide an overview of statistical methods for design and analysis of clinical trials for personalized medicine. In a basic setting where marker-defined subgroups are well defined based on a baseline biomarker, enrichment, markerstratified, and strategy-based clinical trial designs, as well as related data analysis methods, are discussed. Clinical trials with development and validation of predictive signatures or systems are also discussed. Lastly, we provide a brief overview for some recent topics, master protocol clinical trials, the development of dynamic treatment regimens and AI-based diagnostics, as active fields in which further contribution of biometrics is warranted.