Design of Major Randomized Trials: Part 3 of a 4-Part Series on Statistics for Clinical Trials

Abstract

This paper provides practical guidance on the fundamentals of design for major randomized controlled trials. Topics covered include the choice of patients, choice of treatment and control groups, choice of primary and secondary endpoints, methods of randomization, appropriate use of blinding, and determination of trial size. Insights are made with reference to contemporary major trials in cardiology.

Keywords: avoidance of bias; randomized controlled trial; sample size; selection of patients; study design; treatments and endpoints.