Confidence intervals for the difference in the success rates of two treatments in the analysis of correlated binary responses.

Abstract

In clinical studies, we often compare the success rates of two treatment groups where post-treatment responses of subjects within clusters are usually correlated. To estimate the difference between the success rates, interval estimation procedures that do not account for this intraclass correlation are likely inappropriate. To address this issue, we propose three interval procedures by direct extensions of recently proposed methods for independent binary data based on the concepts of design effect and effective sample size used in sample surveys. Each of them is then evaluated with four competing variance estimates. We also extend three existing methods recommended for complex survey data using different weighting schemes required for those three existing methods. An extensive simulation study is conducted for the purposes of evaluating and comparing the performance of the proposed methods in terms of coverage and expected width. The interval estimation procedures are illustrated using three examples in clinical and social science studies. Our analytic arguments and numerical studies suggest that the methods proposed in this work may be useful in clustered data\xa0analyses.