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Observational cohort studies are increasingly being used for comparative effectiveness research to assess the safety of therapeutics. Recently, various doubly robust methods have been proposed for average treatment effect estimation by…

统计方法学 · 统计学 2025-03-11 Xiaoqing Tan , Shu Yang , Wenyu Ye , Douglas E. Faries , Ilya Lipkovich , Zbigniew Kadziola

We examine four important considerations in the development of covariate adjustment methodologies for indirect treatment comparisons. Firstly, we consider potential advantages of weighting versus outcome modeling, placing focus on…

统计方法学 · 统计学 2026-05-07 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

In the absence of data from a randomized trial, researchers often aim to use observational data to draw causal inference about the effect of a treatment on a time-to-event outcome. In this context, interest often focuses on the…

统计方法学 · 统计学 2021-06-15 Ted Westling , Alex Luedtke , Peter Gilbert , Marco Carone

Regression analyses based on transformations of cumulative incidence functions are often adopted when modeling and testing for treatment effects in clinical trial settings involving competing and semi-competing risks. Common frameworks…

统计方法学 · 统计学 2024-01-11 Alexandra Bühler , Richard J Cook , Jerald F Lawless

In causal inference, estimating the average treatment effect is a central objective, and in the context of competing risks data, this effect can be quantified by the cause-specific cumulative incidence function (CIF) difference. While…

统计方法学 · 统计学 2026-03-27 Yifei Tian , Ying Wu

A population-averaged additive subdistribution hazards model is proposed to assess the marginal effects of covariates on the cumulative incidence function and to analyze correlated failure time data subject to competing risks. This approach…

统计方法学 · 统计学 2023-08-23 Xinyuan Chen , Denise Esserman , Fan Li

In precision medicine, one of the most important problems is estimating the optimal individualized treatment rules (ITR), which typically involves recommending treatment decisions based on fully observed individual characteristics of…

统计方法学 · 统计学 2025-10-15 Yue Zhang , Shanshan Luo , Zhi Geng , Yangbo He

The predictiveness curve is a valuable tool for predictive evaluation, risk stratification, and threshold selection in a target population, given a single biomarker or a prediction model. In the presence of competing risks, regression…

统计方法学 · 统计学 2025-08-04 Wei Tao , Jing Ning , Wen Li , Wenyaw Chan , Xi Luo , Ruosha Li

Precision medicine aims to tailor treatment decisions according to patients' characteristics. G-estimation and dynamic weighted ordinary least squares (dWOLS) are double robust statistical methods that can be used to identify optimal…

统计方法学 · 统计学 2021-11-10 Denis Talbot , Erica EM Moodie , Caroline Diorio

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment…

统计方法学 · 统计学 2022-10-05 Dasom Lee , Shu Yang , Xiaofei Wang

In survival contexts, substantial literature exists on estimating optimal treatment regimes, where treatments are assigned based on personal characteristics to maximize the survival probability. These methods assume that a set of covariates…

统计方法学 · 统计学 2025-07-24 Junwen Xia , Zishu Zhan , Jingxiao Zhang

There are now many options for doubly robust estimation; however, there is a concerning trend in the applied literature to believe that the combination of a propensity score and an adjusted outcome model automatically results in a doubly…

Covariate balance is crucial in obtaining unbiased estimates of treatment effects in observational studies. Methods based on inverse probability weights have been widely used to estimate treatment effects with observational data. Machine…

统计方法学 · 统计学 2021-04-08 Michele Santacatterina

Treatment specific survival curves are an important tool to illustrate the treatment effect in studies with time-to-event outcomes. In non-randomized studies, unadjusted estimates can lead to biased depictions due to confounding. Multiple…

统计方法学 · 统计学 2023-04-25 Robin Denz , Renate Klaaßen-Mielke , Nina Timmesfeld

This paper constructs a doubly robust estimator for continuous dose-response estimation. An outcome regression model is augmented with a set of inverse generalized propensity score covariates to correct for potential misspecification bias.…

统计理论 · 数学 2015-06-17 Daniel J. Graham , Emma J. McCoy , David A. Stephens

The use of cumulative incidence functions for characterizing the risk of one type of event in the presence of others has become increasingly popular over the past decade. The problems of modeling, estimation and inference have been treated…

统计方法学 · 统计学 2020-11-16 Youngjoo Cho , Annette M. Molinaro , Chen Hu , Robert L. Strawderman

Valid estimation of treatment effects from observational data requires proper control of confounding. If the number of covariates is large relative to the number of observations, then controlling for all available covariates is infeasible.…

统计方法学 · 统计学 2018-01-11 Joseph Antonelli , Matthew Cefalu , Nathan Palmer , Denis Agniel

This article introduces a new estimator of average treatment effects under unobserved confounding in modern data-rich environments featuring large numbers of units and outcomes. The proposed estimator is doubly robust, combining outcome…

计量经济学 · 经济学 2024-10-30 Alberto Abadie , Anish Agarwal , Raaz Dwivedi , Abhin Shah

Doubly robust estimators combine an inverse probability weighting estimator and a mass imputation estimator. Several doubly robust estimators for estimating the population mean (or prevalence) of an outcome have been proposed for…

统计方法学 · 统计学 2025-08-11 Shaun R Seaman , Tommy Nyberg , Anne M Presanis

We consider the conditional treatment effect for competing risks data in observational studies. While it is described as a constant difference between the hazard functions given the covariates, we do not assume specific functional forms for…

应用统计 · 统计学 2021-12-28 Denise Rava , Ronghui Xu
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