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arXiv · 2008.02341

Bayesian Set of Best Dynamic Treatment Regimes and Sample Size Determination for SMARTs with Binary Outcomes

William J. Artman·Ashkan Ertefaie·Kevin G. Lynch·James R. McKay
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Abstract

One of the main goals of sequential, multiple assignment, randomized trials (SMART) is to find the most efficacious design embedded dynamic treatment regimes. The analysis method known as multiple comparisons with the best (MCB) allows comparison between dynamic treatment regimes and identification of a set of optimal regimes in the frequentist setting for continuous outcomes, thereby, directly addressing the main goal of a SMART. In this paper, we develop a Bayesian generalization to MCB for SMARTs with binary outcomes. Furthermore, we show how to choose the sample size so that the inferior embedded DTRs are screened out with a specified power. We compare log-odds between different DTRs using their exact distribution without relying on asymptotic normality in either the analysis or the power calculation. We conduct extensive simulation studies under two SMART designs and illustrate our method's application to the Adaptive Treatment for Alcohol and Cocaine Dependence (ENGAGE) trial.

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William J. Artman, Ashkan Ertefaie, Kevin G. Lynch, James R. McKay. 2020-08-05. Bayesian Set of Best Dynamic Treatment Regimes and Sample Size Determination for SMARTs with Binary Outcomes. https://arxiv.org/abs/2008.02341

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