Maximin optimal designs for cluster randomized trials.
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.
Department of Biostatistics, UCLA Fielding School of Public Health, University of California, Los Angeles, California 90095-1772, U.S.A.
Sheng
S
Wu
Department of Biostatistics, UCLA Fielding School of Public Health, University of California, Los Angeles, California 90095-1772, U.S.A.
Weng Kee
WK
Wong
Department of Biostatistics, UCLA Fielding School of Public Health, University of California, Los Angeles, California 90095-1772, U.S.A.
Catherine M
CM
Crespi
D035843:Biomedical Research; D016000:Cluster Analysis; D006801:Humans; D016032:Randomized Controlled Trials as Topic; D012306:Risk; D012959:Socioeconomic Factors
Balanced design; Binary outcome; Intraclass correlation coefficient; Relative cost efficiency; Robust design; Sampling ratio