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Methods for extending -- generalizing or transporting -- inferences from a randomized trial to a target population involve conditioning on a large set of covariates that is sufficient for rendering the randomized and non-randomized groups…

统计方法学 · 统计学 2021-10-04 Sarah E Robertson , Jon A Steingrimsson , Issa J Dahabreh

Treatment-induced confounders complicate analyses of time-varying treatment effects and causal mediation. Conditioning on these variables naively to estimate marginal effects may inappropriately block causal pathways and may induce spurious…

应用统计 · 统计学 2018-08-24 Geoffrey T. Wodtke , Zahide Alaca , Xiang Zhou

Adjusting for covariates in randomized controlled trials can enhance the credibility and efficiency of treatment effect estimation. However, handling numerous covariates and their complex (non-linear) transformations poses a challenge.…

统计方法学 · 统计学 2025-10-22 Yi Liu , Ke Zhu , Larry Han , Shu Yang

Cluster-randomized experiments are increasingly used to evaluate interventions in routine practice conditions, and researchers often adopt model-based methods with covariate adjustment in the statistical analyses. However, the validity of…

统计方法学 · 统计学 2023-12-08 Bingkai Wang , Chan Park , Dylan S. Small , Fan Li

Estimation of temporal counterfactual outcomes from observed history is crucial for decision-making in many domains such as healthcare and e-commerce, particularly when randomized controlled trials (RCTs) suffer from high cost or…

机器学习 · 计算机科学 2024-02-13 Chuizheng Meng , Yihe Dong , Sercan Ö. Arık , Yan Liu , Tomas Pfister

Concerns have been expressed over the validity of statistical inference under covariate-adaptive randomization despite the extensive use in clinical trials. In the literature, the inferential properties under covariate-adaptive…

统计方法学 · 统计学 2022-07-05 Li Yang , Wei Ma , Yichen Qin , Feifang Hu

In recent years, there has been growing interest in causal machine learning estimators for quantifying subject-specific effects of a binary treatment on time-to-event outcomes. Estimation approaches have been proposed which attenuate the…

统计方法学 · 统计学 2026-03-30 Matthew Pryce , Karla Diaz-Ordaz , Ruth H. Keogh , Stijn Vansteelandt

In 2023, the U.S. Food and Drug Administration issued guidance for adjustment of covariates in randomized clinical trials, emphasizing its role in enhancing precision and power through prognostic baseline variables. Despite its potential,…

统计方法学 · 统计学 2026-05-28 Kelly Van Lancker , Iván Díaz , Stijn Vansteelandt

Adjusting for (baseline) covariates with working regression models becomes standard practice in the analysis of randomized clinical trials (RCT). When the dimension $p$ of the covariates is large relative to the sample size $n$,…

统计方法学 · 统计学 2025-12-24 Yujia Gu , Lin Liu , Wei Ma

We propose a novel approach for estimating mean survival time in the presence of censored data, in which we divide the population under study into survival-ordered fractions defined by a set of proportions, and compute the mean survival…

统计方法学 · 统计学 2018-10-18 Celia García-Pareja , Matteo Bottai

Randomization is a basis for the statistical inference of treatment effects without strong assumptions on the outcome-generating process. Appropriately using covariates further yields more precise estimators in randomized experiments. R. A.…

统计理论 · 数学 2020-01-03 Xinran Li , Peng Ding

Randomized experiments have become important tools in empirical research. In a completely randomized treatment-control experiment, the simple difference in means of the outcome is unbiased for the average treatment effect, and covariate…

统计理论 · 数学 2021-01-01 Lihua Lei , Peng Ding

We study regression discontinuity designs with the use of additional covariates for estimation of the average treatment effect. We provide a detailed proof of asymptotic normality of the covariate-adjusted estimator under minimal…

统计理论 · 数学 2023-10-16 Patrick Kramer , Alexander Kreiß

In a completely randomized experiment, the variances of treatment effect estimators in the finite population are usually not identifiable and hence not estimable. Although some estimable bounds of the variances have been established in the…

统计理论 · 数学 2022-09-20 Ruoyu Wang , Qihua Wang , Wang Miao , Xiaohua Zhou

Binary endpoints are common in clinical trials and conditional odds ratios have traditionally been used to assess treatment effects. However, the interpretation of odds ratios is difficult, they are non-collapsible and rely on strong…

统计方法学 · 统计学 2026-05-20 Martin Schnuerch , Alex Ocampo , Klaus Kähler Holst , Christian Stock

Sparse additive modeling is a class of effective methods for performing high-dimensional nonparametric regression. This paper develops a sparse additive model focused on estimation of treatment effect-modification with simultaneous…

统计方法学 · 统计学 2020-06-02 Hyung Park , Eva Petkova , Thaddeus Tarpey , R. Todd Ogden

Randomized controlled trials generate experimental variation that can credibly identify causal effects, but often suffer from limited scale, while observational datasets are large, but often violate desired identification assumptions. To…

计量经济学 · 经济学 2023-12-27 George Z. Gui

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

This article considers the joint modeling of longitudinal covariates and partly-interval censored time-to-event data. Longitudinal time-varying covariates play a crucial role in obtaining accurate clinically relevant predictions using a…

统计方法学 · 统计学 2024-12-05 Annabel Webb , Nan Zou , Serigne Lo , Jun Ma

Many rare diseases offer limited established treatment options, leading patients to switch therapies when new medications emerge. To analyze the impact of such treatment switches within the low sample size limitations of rare disease…