TL13: Surrogate Endpoints in Clinical Trials: A Statistical Perspective
*Zhong Gao, FDA/CBER 

Keywords: Surrogate endpoints, clinical trials, validation

Surrogate endpoints have been playing an increasingly important role in clinical trials in broad medical areas such as vaccine development, cardiovascular diseases, immune system disorders, infectious diseases, etc. Surrogate endpoints may potentially help accelerate clinical trials, improve cost-effectiveness and compliance, and avoid awaiting a severe or even fatal clinical endpoint. Moreover, surrogate endpoints facilitate assessment of clinical outcomes that are difficult to measure or observe. Nevertheless, the assessment of relevance of a biomarker to clinical outcome and validation of surrogate endpoints remain challenging from both clinical and statistical perspectives. In this roundtable discussion, we intend to promote communications, among statisticians from industry, academia, and regulatory agencies, on the key issues in the area of surrogate endpoints. The topics include but are not limited to study designs to evaluate surrogate endpoints, statistical methods for surrogate endpoint validation and their pros and cons, meta-analysis, strategies for multiple surrogates, joint modeling of longitudinal biomarkers and time-to-event clinical endpoints, and biomarker assay development.