Randomized controlled trials 5: Determining the sample size and power for clinical trials and cohort studies.

Abstract

Performing well-powered randomized controlled trials is of fundamental importance in clinical research. The goal of sample size calculations is to assure that statistical power is acceptable while maintaining a small probability of a type I error. This chapter overviews the fundamentals of sample size calculation for standard types of outcomes for two-group studies. It considers (1) the problems of determining the size of the treatment effect that the studies will be designed to detect, (2) the modifications to sample size calculations to account for loss to follow-up and nonadherence, (3) the options when initial calculations indicate that the feasible sample size is insufficient to provide adequate power, and (4) the implication of using multiple primary endpoints. Sample size estimates for longitudinal cohort studies must take account of confounding by baseline factors.