Causal Inference under Data Restrictions

TL;DR

This dissertation develops new methods for causal inference under data restrictions, addressing challenges in clinical trials, distributed data, and individualized decision making, with applications demonstrating improved estimation and robustness.

Contribution

It introduces novel approaches for causal effect estimation, model averaging across heterogeneous sites, and robust decision rules considering sensitive variables, advancing the field under data limitations.

Findings

Effective estimation of treatment effects in clinical trials with censored data.

Improved causal effect estimation using model averaging across sites.

Robust individualized decision rules with fairness considerations.

Abstract

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.