Use of Bayesian Techniques in Randomized Clinical Trials: A CMS Case Study. Technology Assessment Report

摘要

We provide a basic tutorial on Bayesian statistics and the possible uses of such statistics in clinical trial design and analysis. We conducted a synthesis of existing published research focusing on how Bayesian techniques can modify inferences that affect policy-level decisionmaking. Noting that subgroup analysis is a particularly fruitful application of Bayesian methodology, and an area of particular interest to CMS, we focused our efforts there rather on the design of such trials. We used simulation studies and a case study of patient-level data from eight trials to explore Bayesian techniques in the CMS decisional context in the clinical domain of the prevention of sudden cardiac death and the use of the implantable cardioverter defibrillator (ICD). We combined knowledge gained through the literature review, simulation studies, and the case study to provide findings concerning the use of Bayesian approaches specific to the CMS context.