Power and sample size calculations for cluster randomized trials with binary outcomes when intracluster correlation coefficients vary by treatment arm.
Lee Kennedy-ShafferMichael D HughesPublished in: Clinical trials (London, England) (2021)
The common approach of fitting generalized estimating equations with an exchangeable working correlation structure with a common intracluster correlation coefficient across arms likely does not substantially reduce the power or efficiency of the analysis in the setting of a large number of small or modest-sized clusters, even if the intracluster correlation coefficient varies by treatment arm. Our formulae, however, allow formal evaluation of this and may identify situations in which variation in intracluster correlation coefficient by treatment arm or another binary covariate may have a more substantial impact on power and hence sample size requirements.