Citation

Wu S, Wong WK, Crespi CM. Biometrics 2017; 73(3): 916-926.

Affiliation

Department of Biostatistics, UCLA Fielding School of Public Health, University of California, Los Angeles, California 90095-1772, U.S.A.

Copyright

(Copyright © 2017, Biometric Society, Publisher John Wiley and Sons)

DOI

10.1111/biom.12659

PMID

28182835

Abstract

We consider design issues for cluster randomized trials (CRTs) with a binary outcome where both unit costs and intraclass correlation coefficients (ICCs) in the two arms may be unequal. We first propose a design that maximizes cost efficiency (CE), defined as the ratio of the precision of the efficacy measure to the study cost. Because such designs can be highly sensitive to the unknown ICCs and the anticipated success rates in the two arms, a local strategy based on a single set of best guesses for the ICCs and success rates can be risky. To mitigate this issue, we propose a maximin optimal design that permits ranges of values to be specified for the success rate and the ICC in each arm. We derive maximin optimal designs for three common measures of the efficacy of the intervention, risk difference, relative risk and odds ratio, and study their properties. Using a real cancer control and prevention trial example, we ascertain the efficiency of the widely used balanced design relative to the maximin optimal design and show that the former can be quite inefficient and less robust to mis-specifications of the ICCs and the success rates in the two arms.

© 2017, The International Biometric Society.

Language: en

Keywords

Balanced design; Binary outcome; Intraclass correlation coefficient; Relative cost efficiency; Robust design; Sampling ratio