This dissertation focuses on modern causal inference under uncertainty and data restrictions, with applications to neoadjuvant clinical trials, distributed data networks, and robust individualized decision making. In the first project, we propose a method under the principal stratification framework to identify and estimate the average treatment effects on a binary outcome, conditional on the counterfactual status of a post-treatment intermediate response. Under mild assumptions, the treatment effect of interest can be identified. We extend the approach to address censored outcome data. The proposed method is applied to a neoadjuvant clinical trial and its performance is evaluated via simulation studies. In the second project, we propose a tree-based model averaging approach to improve the estimation accuracy of conditional average treatment effects at a target site by leveraging models derived from other potentially heterogeneous sites, without them sharing subject-level data. The performance of this approach is demonstrated by a study of the causal effects of oxygen therapy on hospital survival rates and backed up by comprehensive simulations. In the third project, we propose a robust individualized decision learning framework with sensitive variables to improve the worst-case outcomes of individuals caused by sensitive variables that are unavailable at the time of decision. Unlike most existing work that uses mean-optimal objectives, we propose a robust learning framework by finding a newly defined quantile- or infimum-optimal decision rule. From a causal perspective, we also generalize the classic notion of (average) fairness to conditional fairness for individual subjects. The reliable performance of the proposed method is demonstrated through synthetic experiments and three real-data applications.
翻译:在第一个项目中,我们提议在主要分层框架下采用一种方法,以确定和估计平均治疗对二进制结果的影响,但以治疗后中间反应的反事实状况为条件;在轻度假设下,可以确定利息的治疗效果;我们扩大处理受审查的结果数据的方法;拟议方法适用于新黄黄麻临床试验,并通过模拟研究对其性能进行评估;在第二个项目中,我们提议以树为基础的平均模型,利用来自其他可能混杂地点的模型,提高目标地点有条件平均治疗效果的估计准确性,但不分享主题一级数据;这一方法的绩效表现体现在对氧气疗法对医院存活率的因果关系的研究中,并得到全面模拟的支持;在第三个项目中,我们提议一个强有力的个性化决定学习框架,其中含有敏感变量,以改善个人最坏的情况;在第二个项目中,我们提出一个基于敏感因素的公平性能模型,通过从敏感的变量来提高目标的准确性能;在确定最低一级决定时,我们采用一个最稳健的模型;在确定性决定中,我们用一个最稳健的模型,我们用一个最稳健的模型研究。