Analyzing Binary Outcome Data with Small Clusters: A Simulation Study

Binary outcome data with small clusters often arise in medical studies and the size of clusters might be informative of the outcome. The authors conducted a simulation study to examine the performance of a range of statistical methods. The simulation results showed that all methods performed mostly comparable in the estimation of covariate effects. However, the standard logistic regression approach that ignores the clustering encountered an undercoverage problem when the degree of clustering was nontrivial. The performance of random-effects logistic regression approach tended to be affected by low disease prevalence, relatively small cluster size, or informative cluster size.