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Efficient Estimation of the Complier General Causal Effect in Randomized Controlled Trials with One-Sided Noncompliance

Published: October 15, 2025 | arXiv ID: 2510.14142v1

By: Yin Tang, Yanyuan Ma, Jiwei Zhao

Potential Business Impact:

Finds true drug effects even if people don't follow rules.

Business Areas:
Clinical Trials Health Care

A randomized controlled trial (RCT) is widely regarded as the gold standard for assessing the causal effect of a treatment or intervention, assuming perfect implementation. In practice, however, randomization can be compromised for various reasons, such as one-sided noncompliance. In this paper, we address the issue of one-sided noncompliance and propose a general estimand, the complier general causal effect (CGCE), to characterize the causal effect among compliers. We further investigate the conditions under which efficient estimation of the CGCE can be achieved under minimal assumptions. Comprehensive simulation studies and a real data application are conducted to illustrate the proposed methods and to compare them with existing approaches.

Country of Origin
🇺🇸 United States

Page Count
30 pages

Category
Statistics:
Methodology