Identification and efficient estimation of compliance and network causal effects in cluster-randomized trials

TL;DR

This paper develops a semiparametric framework with efficient estimators to identify and estimate individual compliance and network effects in cluster-randomized trials with noncompliance, enhancing causal inference accuracy.

Contribution

It introduces new structural assumptions for nonparametric point identification and combines machine learning with influence functions for robust estimation.

Findings

Applied methods to a Kenyan deworming trial, revealing detailed effects.

Demonstrated improved estimation efficiency over existing approaches.

Provided sensitivity analysis tools for assumption violations.

Abstract

Treatment noncompliance is pervasive in infectious disease cluster-randomized trials. Although all individuals within a cluster are assigned the same treatment condition, the treatment uptake status may vary across individuals due to noncompliance. We propose a semiparametric framework to evaluate the individual compliance effect and network assignment effect within principal stratum exhibiting different patterns of noncompliance. The individual compliance effect captures the portion of the treatment effect attributable to changes in treatment receipt, while the network assignment effect reflects the pure impact of treatment assignment and spillover among individuals within the same cluster. Unlike prior efforts which either empirically identify or interval identify these estimands, we characterize new structural assumptions for nonparametric point identification. We then develop semiparametrically efficient estimators that combine data-adaptive machine learning methods with efficient influence functions to enable more robust inference. Additionally, we introduce sensitivity analysis methods to study the impact under assumption violations, and apply the proposed methods to reanalyze a cluster-randomized trial in Kenya that evaluated the impact of school-based mass deworming on disease transmission.