Sample Size for Prospective and Retrospective Studies: The 2 × 2 Table

Abstract

Previous papers in this series have discussed the design of epidemiologic studies and analysis for data that can be presented in a 2 × 2 table. The purpose of this paper is to explain how sample sizes are determined for unmatched prospective and retrospective studies. One of the most common questions asked in planning a project is “What sample size do I need for my study?” Determining the correct sample size is an important consideration that is best resolved prior to data collection. In this paper we assume the goal of the study is to examine the association between a dichotomous risk factor and the presence or absence of disease; therefore, the analysis will be that of a 2 × 2 table. We also assume throughout that sample sizes are to be equal for the two comparison groups (ie, equal numbers of exposed and unexposed for a prospective study, and equal numbers of cases and controls for a retrospective study). Sample size curves used to determine sample sizes will be presented. The results here are based on a paper by Schlesselman that contains sample size formulae for these study designs, and to which the reader may refer for an excellent technical discussion of their derivation.