ASSESSING TREATMENT EFFECT THROUGH COMPLIANCE SCORE IN RANDOMIZED TRIALS WITH NONCOMPLIANCE

• 成果类型: Article
• 署名作者: Hu, Zonghui; Zhang, Zhiwei; Follmann, Dean
• 署名单位: National Institutes of Health (NIH) - USA; NIH National Institute of Allergy & Infectious Diseases (NIAID); National Institutes of Health (NIH) - USA; NIH National Cancer Institute (NCI)
• 刊物名称: ANNALS OF APPLIED STATISTICS
• ISSN/ISSBN: 1932-6157
• DOI: 10.1214/21-AOAS1590
• 发表日期: 2022
• 页码: 2279-2290
• 关键词: intention-to-treat; Dimension Reduction; principal stratification; Causal Inference

摘要

A randomized trial is the gold standard for assessing the benefit of a treat-ment versus a control. When noncompliance is present, treatment effect de-pends on the tendency to comply-an attribute that is not directly measurable. Though the principal causal effect has been the most important for handling noncompliance, it is not immediately applicable to clinical decision-making as it targets the average effect in the latent strata of potential compliance. In this work, we propose the concept of compliance score, a linear combination of baseline characteristics, that uncovers the inherent attribute of compliance. We then assess the heterogeneous causal effect, namely, the causal effect of treatment as a function of baseline characteristics through the compliance score. A pseudo-response, along with a nonparametric estimation procedure, is proposed to ensure consistent and optimally efficient estimation. Compare to principal causal effect, the proposed effect is actionable and allows pre-diction of treatment effect at individual level. This work is motivated by and applied to a clinical trial to evaluate the benefit of antiretroviral regimens in HIV-infected patients.