Wu Lili, Yang Shu
Department of Statistics, North Carolina State University.
J Comput Graph Stat. 2023;32(3):1036-1045. doi: 10.1080/10618600.2022.2141752. Epub 2022 Nov 30.
Individualized treatment effect lies at the heart of precision medicine. Interpretable individualized treatment rules (ITRs) are desirable for clinicians or policymakers due to their intuitive appeal and transparency. The gold-standard approach to estimating the ITRs is randomized experiments, where subjects are randomized to different treatment groups and the confounding bias is minimized to the extent possible. However, experimental studies are limited in external validity because of their selection restrictions, and therefore the underlying study population is not representative of the target real-world population. Conventional learning methods of optimal interpretable ITRs for a target population based only on experimental data are biased. On the other hand, real-world data (RWD) are becoming popular and provide a representative sample of the target population. To learn the generalizable optimal interpretable ITRs, we propose an integrative transfer learning method based on weighting schemes to calibrate the covariate distribution of the experiment to that of the RWD. Theoretically, we establish the risk consistency for the proposed ITR estimator. Empirically, we evaluate the finite-sample performance of the transfer learner through simulations and apply it to a real data application of a job training program.
个性化治疗效果是精准医学的核心。可解释的个性化治疗规则(ITRs)因其直观性和透明度而受到临床医生或政策制定者的青睐。估计ITRs的金标准方法是随机试验,即将受试者随机分配到不同的治疗组,并尽可能减少混杂偏倚。然而,实验研究由于其选择限制,外部效度有限,因此潜在的研究人群不能代表目标现实世界人群。仅基于实验数据为目标人群学习最优可解释ITRs的传统方法存在偏差。另一方面,真实世界数据(RWD)正变得越来越流行,并提供了目标人群的代表性样本。为了学习可推广的最优可解释ITRs,我们提出了一种基于加权方案的整合迁移学习方法,以将实验的协变量分布校准到RWD的协变量分布。从理论上讲,我们建立了所提出的ITR估计器的风险一致性。从实证角度,我们通过模拟评估了迁移学习器的有限样本性能,并将其应用于一个职业培训项目的真实数据应用中。