Estimation of Optimal Individualized Treatment Rules Using a Covariate-Specific Treatment Effect Curve With High-Dimensional Covariates
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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.
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Instrumental variable (IV) methods address treatment endogeneity, but with non-compliance and heterogeneous treatment effects a binary instrument generally identifies the local average treatment effect (LATE) among compliers rather than the population average treatment effect (ATE). When treatment effects and compliance probabilities are heterogeneous and dependent through latent factors, the ATE need not be identified by IV variation alone. We develop a proximal…
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Principal stratification provides a foundational framework for causal inference with intermediate outcomes by defining causal effects within subpopulations, yet existing work has largely focused on average effects across strata rather than treatment effect heterogeneity within strata. Such within-stratum heterogeneity informs individualized treatment decisions but the associated methods are sparse. We address this gap by studying the identification and estimation of the…
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We study target-population distributional and quantile treatment effects when a source study observes treatment and post-treatment surrogates for all source units but observes a long-run primary outcome only for a validation subset, while the target population contributes only baseline covariates. The target estimands are transported counterfactual distribution functions $ψ_a(y)=P(Y^a\le y\mid R=0)$, their quantiles $q_a(τ)$, and the quantile treatment effect $Δ(τ)=q_1(τ)-q_0(τ)$…
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We propose methods for estimation and uniform inference for a broad class of causal functions, such as conditional average treatment effects and continuous treatment effects, under multi-way clustering. The causal function is identified as the conditional expectation of a Neyman-orthogonal signal that depends on high-dimensional nuisance parameters. We introduce a two-step procedure: the first step uses machine learning to estimate the nuisance parameters, and the second…
多维聚类数据下因果函数的估计与推断
This paper proposes methods of estimation and uniform inference for a general class of causal functions, such as the conditional average treatment effects and the continuous treatment effects, under multiway clustering. The causal function is identified as a conditional expectation of an adjusted (Neyman-orthogonal) signal that depends on high-dimensional nuisance parameters. We propose a two-step procedure where the first step uses machine learning to estimate the…
可微 Hilbert 空间值参数的一步估计
rate estimators and corresponding confidence sets. These estimators correspond to generalizations of cross-fitted one-step estimators based on Hilbert-valued efficient influence functions. We give theoretical guarantees even when arbitrary estimators of nuisance functions are used, including those based on machine learning techniques. We show that these results naturally extend to Hilbert spaces that lack a reproducing kernel, as long as the parameter has an efficient…