Rerandomization and optimal matching

Author:

Kalbfleisch John D.1ORCID,Xu Zhenzhen2

Affiliation:

1. Department of Biostatistics University of Michigan Ann Arbor Michigan MI 48109 U.S.A

2. Division of Biostatistics Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Avenue Silver Spring Maryland MD 20993 U.S.A.

Abstract

AbstractOn average, randomization achieves balance in covariate distributions between treatment groups; yet in practice, chance imbalance exists post randomization, which increases the error in estimating treatment effects. This is an important issue, especially in cluster randomized trials, where the experimental units (the clusters) are highly heterogeneous and relatively few in number. To address this, several restricted randomization designs have been proposed to balance on a few covariates of particular interest. More recently, approaches involving rerandomization have been proposed that aim to achieve simultaneous balance on several important prognostic factors. In this article, we comment on some properties of rerandomized designs and propose a new design for comparing two or more treatments. This design combines optimal nonbipartite matching of the subjects together with rerandomization, both aimed at minimizing a measure of distance between elements in blocks to achieve reductions in the mean squared error of estimated treatment effects. Compared with the existing alternatives, the proposed design can substantially reduce the mean squared error of the estimated treatment effect. This enhanced efficiency is evaluated both theoretically and empirically, and robustness properties are also noted. The design is generalized to three or more treatment arms.

Funder

U.S. Food and Drug Administration

Publisher

Wiley

Subject

Statistics, Probability and Uncertainty,Statistics and Probability

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