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.
翻译:我们考虑了在非对称一般反应模型下对发生(二进制)结果进行双臂随机实验的设计进行评价的问题。我们的两个主要结果是,Greevy等人(2004年)的先行配对匹配设计(1) 在所有区块设计(包括完全随机化)中,以平均正方差衡量的最佳设计。(2) 这种配对设计是微缩式的,即在对称反应模型下提供了最低平均正方差。理论结果得到了模拟和临床试验数据的支持。