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Using Decision Lists to Construct Interpretable and Parsimonious Treatment Regimes

机译:使用决定列表构建可解释和宽松的治疗制度

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摘要

A treatment regime formalizes personalized medicine as a function from individual patient characteristics to a recommended treatment. A high-quality treatment regime can improve patient outcomes while reducing cost, resource consumption, and treatment burden. Thus, there is tremendous interest in estimating treatment regimes from observational and randomized studies. However, the development of treatment regimes for application in clinical practice requires the long-term, joint effort of statisticians and clinical scientists. In this collaborative process, the statistician must integrate clinical science into the statistical models underlying a treatment regime and the clinician must scrutinize the estimated treatment regime for scientific validity. To facilitate meaningful information exchange, it is important that estimated treatment regimes be interpretable in a subject-matter context. We propose a simple, yet flexible class of treatment regimes whose members are representable as a short list of if-then statements. Regimes in this class are immediately interpretable and are therefore an appealing choice for broad application in practice. We derive a robust estimator of the optimal regime within this class and demonstrate its finite sample performance using simulation experiments. The proposed method is illustrated with data from two clinical trials.
机译:治疗制度将个性化药物形式形式是从个体患者特征到推荐治疗的功能。高质量的治疗制度可以改善患者结果,同时降低成本,资源消耗和治疗负担。因此,对从观察和随机研究的治疗方案估算治疗方案存在巨大兴趣。然而,在临床实践中申请的治疗方案的发展需要长期,共同努力的统计学家和临床科学家。在这一协作过程中,统计名人必须将临床科学整合到治疗制度的统计模型中,临床医生必须审查估计的治疗制度以进行科学有效性。为了便于有意义的信息交流,重要的是在主题背景下可以解释估计的治疗制度。我们提出了一种简单而灵活的一类治疗制度,其成员可以作为if-then语句的简短列表。本课程中的制度立即可解释,因此在实践中具有广泛应用的吸引人选择。我们在该类中获得了最佳制度的强大估算器,并使用模拟实验展示其有限的样本性能。所提出的方法用来自两种临床试验的数据说明。

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