Estimation in optimal treatment regimes based on mean residual lifetimes with right‐censored data

Abstract

AbstractAn optimal individualized treatment regime (ITR) is a decision rule in allocating the best treatment to each patient and, hence, maximizing overall benefits. In this paper, we propose a novel framework based on nonparametric inverse probability weighting (IPW) and augmented inverse probability weighting (AIPW) estimators of the value function when the data are subject to right censoring. In contrast to most existing approaches that are designed to maximize the expected survival time under a binary treatment framework, the proposed method targets maximizing the mean residual lifetime of patients. Specifically, the proposed IPW method searches the optimal ITR by maximizing an estimator for the overall population outcome directly, without specifying the regression model for the conditional mean residual lifetime, whereas the AIPW method integrates the model information of the mean residual lifetime to improve the robustness. Furthermore, to overcome the computational difficulty in a nonsmooth value estimator, smoothed IPW and AIPW estimators are constructed. In theory, we establish the asymptotic properties of the proposed method under suitable regularity conditions. The empirical performances of the proposed IPW and AIPW estimators are evaluated using simulation studies and are further illustrated with an application to the real‐world data set from the Acquired Immunodeficiency Syndrome Clinical Trial Group Protocol 175 (ACTG175).