A Novel Confidence Interval for a Single Proportion in the Presence of Clustered Binary Outcome Data

• Activity Number: 476 - SPEED: Clinical Trial Design, Longitudinal Analysis, and Other Topics in Biopharmaceutical Statistics
• Type: Contributed
• Date/Time: Wednesday, August 1, 2018 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Meghan Short* and Joseph M. Massaro
• Companies: Boston University School of Public Health and Boston University
• Keywords: clustered binary data; beta-binomial distribution; confidence interval; small sample; coverage; empirical length

Abstract

Estimating a single proportion via a 100(1-?)% confidence interval in the presence of clustered data is an important statistical problem. It is necessary to account for possible under- or over-dispersion, for instance, in animal-based teratology studies with within-litter correlation, epidemiological studies that involve clustered sampling, and clinical trial designs with multiple measurements per subject. Several asymptotic confidence interval methods have been developed, many of which have not been compared with regard to the operational characteristics of coverage probability and empirical length. In addition, these asymptotic intervals have been found via simulation to have under-coverage of the true proportion for small-to-moderate sample sizes. This study uses Monte Carlo simulations to calculate coverage probabilities and empirical lengths of five existing confidence intervals for clustered data across various true correlations, true probabilities of interest, and sample sizes. In addition, we introduce a new score-based confidence interval method, which we find to have better coverage than existing intervals for small sample sizes under a wide range of scenarios.