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Main Authors: Spicker, Dylan, Wallace, Michael P., Yi, Grace Y.
Format: Preprint
Published: 2024
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Online Access:https://arxiv.org/abs/2402.12555
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author Spicker, Dylan
Wallace, Michael P.
Yi, Grace Y.
author_facet Spicker, Dylan
Wallace, Michael P.
Yi, Grace Y.
contents 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 were they 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 that 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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publishDate 2024
record_format arxiv
spellingShingle Optimal Dynamic Treatment Regime Estimation in the Presence of Nonadherence
Spicker, Dylan
Wallace, Michael P.
Yi, Grace Y.
Methodology
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 were they 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 that 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.
title Optimal Dynamic Treatment Regime Estimation in the Presence of Nonadherence
topic Methodology
url https://arxiv.org/abs/2402.12555