Principal causal effect identification and surrogate end point evaluation by multiple trials

DOI10.1111/RSSB.12135zbMATH Open1414.62048arXiv1507.05935OpenAlexW951453072MaRDI QIDQ5378371FDOQ5378371

Authors: Zhichao Jiang, Peng Ding, Zhi Geng 

Publication date: 12 June 2019

Published in: Journal of the Royal Statistical Society Series B: Statistical Methodology (Search for Journal in Brave)

Abstract: Principal stratification is a causal framework to analyze randomized experiments with a post-treatment variable between the treatment and endpoint variables. Because the principal strata defined by the potential outcomes of the post-treatment variable are not observable, we generally cannot identify the causal effects within principal strata. Motivated by a real data set of phase III adjuvant colon clinical trials, we propose approaches to identifying and estimating the principal causal effects via multiple trials. For the identifiability, we remove the commonly-used exclusion restriction assumption by stipulating that the principal causal effects are homogeneous across these trials. To remove another commonly-used monotonicity assumption, we give a necessary condition for the local identifiability, which requires at least three trials. Applying our approaches to the data from adjuvant colon clinical trials, we find that the commonly-used monotonicity assumption is untenable, and disease-free survival with three-year follow-up is a valid surrogate endpoint for overall survival with five-year follow-up, which satisfies both the causal necessity and the causal sufficiency. We also propose a sensitivity analysis approach based on Bayesian hierarchical models to investigate the impact of the deviation from the homogeneity assumption.

Full work available at URL: https://arxiv.org/abs/1507.05935

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zbMATH Keywords

causal inferenceclinical trialcausal necessitycausal sufficiencycriterion for surrogate end points

Mathematics Subject Classification ID

Nonparametric hypothesis testing (62G10) Censored data models (62N01) Applications of statistics to biology and medical sciences; meta analysis (62P10) Sampling theory, sample surveys (62D05)

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• Principal stratification in causal inference
• Identification and estimation of principal causal effects in randomized experiments with treatment switching
• Surrogate outcomes and transportability
• Using missing types to improve partial identification with application to a study of HIV prevalence in Malawi
• A causal framework for surrogate endpoints with semi-competing risks data
• Causal measures of the treatment effect captured by candidate surrogate endpoints
• Causal effects of treatments for informative missing data due to progression/death
• Defining estimands in clinical trials: a unified procedure
• Evaluating Candidate Principal Surrogate Endpoints
• Statistical identifiability and the surrogate endpoint problem, with application to vaccine trials
• A Bayesian approach to improved estimation of causal effect predictiveness for a principal surrogate endpoint
• Identifiability and estimation of causal effects by principal stratification with outcomes truncated by death
• Identifiability of causal effects on a binary outcome within principal strata
• Identification of causal effects within principal strata using auxiliary variables
• Related Causal Frameworks for Surrogate Outcomes
• Policy Learning with Asymmetric Counterfactual Utilities
• Identification and estimation of causal effects in the presence of confounded principal strata
• Identifying and estimating principal causal effects in a multi-site trial of early college high schools

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