Balanced designs that balance covariates (especially blocking) are asymptotically variance-optimal for binary ATE under Neyman's nonparametric model, and a CMH-based variance estimator is finite-sample conservative and asymptotically tight under local alternatives.
The Role of Pairwise Matching in Experimental Design for an Incidence Outcome
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abstract
We consider the problem of evaluating designs for a two-arm randomized experiment with an incidence (binary) outcome under a nonparametric general response model. Our two main results are that the priori pair matching design of Greevy et al. (2004) is (1) the optimal design as measured by mean squared error among all block designs which includes complete randomization. And (2), this pair-matching design is minimax, i.e. it provides the lowest mean squared error under an adversarial response model. Theoretical results are supported by simulations and clinical trial data.
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Optimal Designs with Robust Inference for Binary Treatment Effects
Balanced designs that balance covariates (especially blocking) are asymptotically variance-optimal for binary ATE under Neyman's nonparametric model, and a CMH-based variance estimator is finite-sample conservative and asymptotically tight under local alternatives.