Optimal dynamic treatment regime estimation in the presence of nonadherence

Dynamic treatment regimes (DTRs) are sequences of functions that formalize the process of precision medicine. DTRs take as input patient information and output treatment recommendations. A major focus of the DTR literature has been on the estimation of optimal DTRs, the sequences of decision rules that result in the best outcome in expectation, across the complete population if they were to be applied. While there is a rich literature on optimal DTR estimation, to date, there has been minimal consideration of the impacts of nonadherence on these estimation procedures. Nonadherence refers to any process through which an individual's prescribed treatment does not match their true treatment. We explore the impacts of nonadherence and demonstrate that, generally, when nonadherence is ignored, suboptimal regimes will be estimated. In light of these findings, we propose a method for estimating optimal DTRs in the presence of nonadherence. The resulting estimators are consistent and asymptotically normal, with a double robustness property. Using simulations, we demonstrate the reliability of these results, and illustrate comparable performance between the proposed estimation procedure adjusting for the impacts of nonadherence and estimators that are computed on data without nonadherence.

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Publication Details

Journal
Biometrics
Published
2025-04-02
DOI
https://doi.org/10.1093/biomtc/ujaf041
Primary Topic
Advanced Causal Inference Techniques
Type
article
Field-Weighted Citation Impact
0.00

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article

Optimal dynamic treatment regime estimation in the presence of nonadherence

Grace Y. Yi, Dylan Spicker, Michael P. Wallace
Biometrics
Advanced Causal Inference Techniques
article

Optimal dynamic treatment regime estimation in the presence of nonadherence

Grace Y. Yi, Dylan Spicker, Michael P. Wallace
article en

Abstract

Dynamic treatment regimes (DTRs) are sequences of functions that formalize the process of precision medicine. DTRs take as input patient information and output treatment recommendations. A major focus of the DTR literature has been on the estimation of optimal DTRs, the sequences of decision rules that result in the best outcome in expectation, across the complete population if they were to be applied. While there is a rich literature on optimal DTR estimation, to date, there has been minimal consideration of the impacts of nonadherence on these estimation procedures. Nonadherence refers to any process through which an individual's prescribed treatment does not match their true treatment. We explore the impacts of nonadherence and demonstrate that, generally, when nonadherence is ignored, suboptimal regimes will be estimated. In light of these findings, we propose a method for estimating optimal DTRs in the presence of nonadherence. The resulting estimators are consistent and asymptotically normal, with a double robustness property. Using simulations, we demonstrate the reliability of these results, and illustrate comparable performance between the proposed estimation procedure adjusting for the impacts of nonadherence and estimators that are computed on data without nonadherence.

BiometricsVol. 81(2)
University of New Brunswick (CA), Western University (CA), University of Waterloo (CA)
New Brunswick Innovation Foundation, Natural Sciences and Engineering Research Council of Canada
Peace, Justice and strong institutions
Openalex Percentile: Top 91%
Advanced Causal Inference Techniques
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