Optimal and lead-in adaptive allocation for binary outcomes: a comparison of Bayesian methodologies

Commun Stat Theory Methods. 2017;46(6):2823-2836. doi: 10.1080/03610926.2015.1053929. Epub 2016 Apr 8.

Abstract

We compare posterior and predictive estimators and probabilities in response-adaptive randomization designs for two- and three-group clinical trials with binary outcomes. Adaptation based upon posterior estimates are discussed, as are two predictive probability algorithms: one using the traditional definition, the other using a skeptical distribution. Optimal and natural lead-in designs are covered. Simulation studies show: efficacy comparisons lead to more adaptation than center comparisons, though at some power loss; skeptically predictive efficacy comparisons and natural lead-in approaches lead to less adaptation but offer reduced allocation variability. Though nuanced, these results help clarify the power-adaptation trade-off in adaptive randomization.

Keywords: Adaptive Randomization; Bayesian Methods; Clinical Trials; Predictive Probability.