Overview of statistical methods for binary endpoint in immunology clinical trials

Music City Center 

In immunology clinical trials, the primary endpoint to evaluate the drug efficacy comparing to placebo is usually the binary endpoint. Odds ratio, rate difference and rate ratio are three most popular measures to analyze binary endpoint. Which measure to choose for binary endpoint is not only a clinical question but also a statistical question. As odds ratio has good statistical properties, CMH test or logistic regression have been widely used to derive adjusted odds ratio and the corresponding p-value in the immunology clinical trials for binary endpoints. In this work, population-level summary and covariate adjustment for unconditional treatment effect of binary endpoint have been discussed based on recent FDA's guidance, and a comprehensive review through different clinical trials has been conducted and the methods used for binary endpoints in published trials have been summarized. The performance of commonly used statistical methods for binary endpoint has been evaluated with simulations. The recommendations for analysis methods of binary endpoints will be given in the end.

Immunology clinical trial

binary endpoints

CMH analysis

odds ratio

rate difference 

Biopharmaceutical Section