Efficient nonparametric inference on the effects of stochastic interventions under two‐phase sampling, with applications to vaccine efficacy trials

DOI10.1111/BIOM.13375zbMath1520.62224arXiv2003.13771OpenAlexW3014731829WikidataQ99572895 ScholiaQ99572895MaRDI QIDQ6079255

Unnamed Author, Nima S. Hejazi, Peter B. Gilbert, Unnamed Author

Publication date: 30 October 2023

Published in: Biometrics (Search for Journal in Brave)

Abstract: The advent and subsequent widespread availability of preventive vaccines has altered the course of public health over the past century. Despite this success, effective vaccines to prevent many high-burden diseases, including HIV, have been slow to develop. Vaccine development can be aided by the identification of immune response markers that serve as effective surrogates for clinically significant infection or disease endpoints. However, measuring immune response marker activity is often costly, which has motivated the usage of two-phase sampling for immune response evaluation in clinical trials of preventive vaccines. In such trials, the measurement of immunological markers is performed on a subset of trial participants, where enrollment in this second phase is potentially contingent on the observed study outcome and other participant-level information. We propose nonparametric methodology for efficiently estimating a counterfactual parameter that quantifies the impact of a given immune response marker on the subsequent probability of infection. Along the way, we fill in theoretical gaps pertaining to the asymptotic behavior of nonparametric efficient estimators in the context of two-phase sampling, including a multiple robustness property enjoyed by our estimators. Techniques for constructing confidence intervals and hypothesis tests are presented, and an open source software implementation of the methodology, the txshift R package, is introduced. We illustrate the proposed techniques using data from a recent preventive HIV vaccine efficacy trial.

Full work available at URL: https://arxiv.org/abs/2003.13771

zbMATH Keywords

two-phase samplingcausal inferencevaccine efficacytargeted minimum loss estimationstochastic interventions

Mathematics Subject Classification ID

Applications of statistics to biology and medical sciences; meta analysis (62P10)

Cites Work

• Unnamed Item
• Population Intervention Causal Effects Based on Stochastic Interventions
• External validity: from do-calculus to transportability across populations
• Asymptotic expansions for general statistical models. With the assist. of W. Wefelmeyer
• Consistent estimation of the influence function of locally asymptotically linear estimators
• Large sample theory for semiparametric regression models with two-phase, outcome dependent sampling.
• Targeted learning in data science. Causal inference for complex longitudinal studies
• Toward Causal Inference With Interference
• Logistic regression for two-stage case-control data
• The Estimation of Choice Probabilities from Choice Based Samples
• Estimation of Regression Coefficients When Some Regressors Are Not Always Observed
• Causal Mediation Analysis for Stochastic Interventions
• Double/debiased machine learning for treatment and structural parameters
• Nonparametric Causal Effects Based on Incremental Propensity Score Interventions
• Non-parametric Methods for Doubly Robust Estimation of Continuous Treatment Effects
• Super Learner
• Contribution to the Theory of Sampling Human Populations

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