Two-phase treatment with noncompliance: identifying the cumulative average treatment effect via multisite instrumental variables

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Authors: Guanglei Hong, Qin Hua Xu, Zhengyan Xu, Fan Yang
Cited by: 0

Abstract

Abstract When evaluating a two-phase intervention, the cumulative average treatment effect (ATE) is often the primary causal estimand of interest. However, some individuals who do not respond well to the Phase I treatment may subsequently display noncompliant behaviours. At the same time, exposure to the Phase I treatment is expected to directly influence an individual’s potential outcomes, thereby violating the exclusion restriction. Building on an instrumental variable strategy for multisite trials, we clarify the conditions under which the cumulative ATE of a two-phase treatment can be identified by employing the random assignment of the Phase I treatment as the instrument. Our strategy relaxes both the conventional exclusion restriction and sequential ignorability assumptions. We assess the performance of the new strategy through simulation studies. Additionally, we reanalyse data from the Tennessee class size study, in which students and teachers were randomly assigned to either small or regular class types in kindergarten (Phase I) with noncompliance emerging in Grade 1 (Phase II). Applying our new strategy, we estimate the cumulative ATE of receiving two consecutive years of instruction in a small versus regular class.

Fields of Study & Subjects

Advanced Causal Inference Techniques Statistical Methods in Clinical Trials Pharmacy and Medical Practices

Publication details

Journal / source
Journal of the Royal Statistical Society Series A (Statistics in Society)
Publication date
28 May 2026
Language
EN