Estimation of Optimal Individualized Treatment Rules Using a Covariate-Specific Treatment Effect Curve With High-Dimensional Covariates

Wenchuan Guo, Xiao-Hua Zhou and Shujie Ma

Journal of the American Statistical Association, 2021, vol. 116, issue 533, 309-321

Abstract: With a large number of baseline covariates, we propose a new semiparametric modeling strategy for heterogeneous treatment effect estimation and individualized treatment selection, which are two major goals in personalized medicine. We achieve the first goal through estimating a covariate-specific treatment effect (CSTE) curve modeled as an unknown function of a weighted linear combination of all baseline covariates. The weight or the coefficient for each covariate is estimated by fitting a sparse semiparametric logistic single-index coefficient model. The CSTE curve is estimated by a spline-backfitted kernel procedure, which enables us to further construct a simultaneous confidence band (SCB) for the CSTE curve under a desired confidence level. Based on the SCB, we find the subgroups of patients that benefit from each treatment, so that we can make individualized treatment selection. The innovations of the proposed method are 3-fold. First, the proposed method can quantify variability associated with the estimated optimal individualized treatment rule with high-dimensional covariates. Second, the proposed method is very flexible to depict both local and global associations between the treatment and baseline covariates in the presence of high-dimensional covariates, and thus it enjoys flexibility while achieving dimensionality reduction. Third, the SCB achieves the nominal confidence level asymptotically, and it provides a uniform inferential tool in making individualized treatment decisions. Supplementary materials for this article are available online.

Date: 2021
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DOI: 10.1080/01621459.2020.1865167

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