Clinical Trial Design in the Age of Precision Medicine

This course offers an overview of statistical foundation for modern clinical trial design in precision medicine research. Starting from a quick review of traditional clinical development paradigm through Phase I to III clinical trials for medical product approval and Phase IV post-marketing studies for safety evaluation, and challenges in the time and society costs, we will introduce recently developed innovative designs and their statistical methodology across all phases of clinical trials. You expected to learn the statistical considerations for novel phase I-II trial designs, master protocols for umbrella, platform and basket trials, adaptive and enrichment designs including subgroup selections, estimand, surrogate and composite endpoints, integration of real-world evidence and patient-focused medical product development, and meta-analysis of clinical trial endpoints. Prerequisites: Working knowledge of statistics and R.