Assessing the treatment effect heterogeneity with a latent variable

DOI10.5705/SS.202016.0150zbMATH Open1382.62058arXiv1603.02712OpenAlexW2962721263MaRDI QIDQ4602118FDOQ4602118

Authors: Yunjian Yin, Lan Liu, Zhi Geng 

Publication date: 26 January 2018

Published in: STATISTICA SINICA (Search for Journal in Brave)

Abstract: The average treatment effect (ATE) is popularly used to assess the treatment effect. However, the ATE implicitly assumes a homogenous treatment effect even amongst individuals with different characteristics. In this paper, we mainly focus on assessing the treatment effect heterogeneity, which has important implications in designing the optimal individual treatment regimens and in policy making. The treatment benefit rate (TBR) and treatment harm rate (THR) have been defined to characterize the magnitude of heterogeneity for binary outcomes. When the outcomes are continuous, we extend the definitions of the TBR and THR to compare the difference between potential outcomes with a pre-specified level c. Unlike the ATE, these rates involve the joint distribution of the potential outcomes and can not be identified without further assumptions even in randomized clinical trials. In this article, we assume the potential outcomes are independent conditional on the observed covariates and an unmeasured latent variable. Under this assumption, we prove the identification of the TBR and THR in non-separable (generalized) linear models for both continuous and binary outcomes. We then propose estimators and derive their asymptotic distributions. In the simulation studies, we implement our proposed methods to assess the performance of our estimators and carry out a sensitive analysis for different underlying distribution for the latent variable. Finally, we illustrate the proposed methods in two randomized controlled trials.

Full work available at URL: https://arxiv.org/abs/1603.02712

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zbMATH Keywords

causal inferenceheterogeneitylatent variableaverage treatment effect

Mathematics Subject Classification ID

Applications of statistics to biology and medical sciences; meta analysis (62P10) Generalized linear models (logistic models) (62J12)

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• Modern approaches for evaluating treatment effect heterogeneity from clinical trials and observational data
• Identification of causal effects with latent confounding and classical additive errors in treatment
• Latent class mixture models of treatment effect heterogeneity

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