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Estimating individualized treatment rules - particularly in the context of right-censored outcomes - is challenging because the treatment effect heterogeneity of interest is often small, thus difficult to detect. While this motivates the…

Covariate imbalance between treatment groups makes it difficult to compare cumulative incidence curves in competing risk analyses. In this paper we discuss different methods to estimate adjusted cumulative incidence curves including inverse…

统计方法学 · 统计学 2024-12-04 Patrick van Hage , Saskia le Cessie , Marissa C. van Maaren , Hein Putter , Nan van Geloven

The doubly-robust (DR) estimator is popular for evaluating causal effects in observational studies and is often perceived as more desirable than inverse probability weighting (IPW) or outcome modeling alone because it provides extra…

统计方法学 · 统计学 2026-02-03 Chengxin Yang , Laine E. Thomas , Fan Li

This paper considers the evaluation of discretely distributed treatments when outcomes are only observed for a subpopulation due to sample selection or outcome attrition. For identification, we combine a selection-on-observables assumption…

计量经济学 · 经济学 2021-07-16 Michela Bia , Martin Huber , Lukáš Lafférs

Covariate adjustment is desired by both practitioners and regulators of randomized clinical trials because it improves precision for estimating treatment effects. However, covariate adjustment presents a particular challenge in…

统计方法学 · 统计学 2023-07-20 Yunfan Li , Jessica L. Ross , Aaron M. Smith , David P. Miller

In prevalent cohort studies with follow-up, the time-to-event outcome is subject to left truncation leading to selection bias. For estimation of the distribution of time-to-event, conventional methods adjusting for left truncation tend to…

统计方法学 · 统计学 2025-12-29 Yuyao Wang , Andrew Ying , Ronghui Xu

This paper is devoted to robust estimation based on dual divergences estimators for parametric models in the framework of right censored data. We give limit laws of the proposed estimators and examine their asymptotic properties through a…

统计理论 · 数学 2011-06-15 Mohamed Cherfi

To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying…

统计方法学 · 统计学 2023-08-08 Liangyuan Hu , Jiayi Ji , Himanshu Joshi , Erick Scott , Fan Li

A model for competing (resp. complementary) risks survival data where the failure time can be left (resp. right) censored is proposed. Product-limit estimators for the survival functions of the individual risks are derived. We deduce the…

统计理论 · 数学 2007-06-13 Valentin Patilea , Jean-Marie Rolin

The pseudo-observations approach has been gaining popularity as a method to estimate covariate effects on censored survival data. It is used regularly to estimate covariate effects on quantities such as survival probabilities, restricted…

统计方法学 · 统计学 2024-12-06 Yael Travis-Lumer , Micha Mandel , Rebecca A. Betensky

We consider the problem of estimating the effects of a binary treatment on a continuous outcome of interest from observational data in the absence of confounding by unmeasured factors. We provide a new estimator of the population average…

统计方法学 · 统计学 2020-08-04 James Robins , Mariela Sued , Quanhong Lei-Gomez , Andrea Rotnitzky

Cluster-randomized trials (CRTs) are experimental designs where groups or clusters of participants, rather than the individual participants themselves, are randomized to intervention groups. Analyzing CRT requires distinguishing between…

统计方法学 · 统计学 2025-10-10 Xi Fang , Bingkai Wang , Liangyuan Hu , Fan Li

There is growing interest in a hybrid control design for treatment evaluation, where a randomized controlled trial is augmented with external control data from a previous trial or a real world data source. The hybrid control design has the…

统计方法学 · 统计学 2026-05-06 Zhiwei Zhang , Peisong Han , Wei Zhang

We are interested in the estimation of average treatment effects based on right-censored data of an observational study. We focus on causal inference of differences between t-year absolute event risks in a situation with competing risks. We…

In time-to-event settings, g-computation and doubly robust estimators are based on discrete-time data. However, many biological processes are evolving continuously over time. In this paper, we extend the g-computation and the doubly robust…

统计方法学 · 统计学 2024-11-15 A. Chatton , F. Le Borgne , C. Leyrat , Y. Foucher

Longitudinal data often involve heterogeneity, sparse signals, and contamination from response outliers or high-leverage observations especially in biomedical science. Existing methods usually address only part of this problem, either…

统计方法学 · 统计学 2026-02-26 Yuyao Wang , Yu Lu , Tianni Zhang , Mengfei Ran

Individualized treatment rules can lead to better health outcomes when patients have heterogeneous responses to treatment. Very few individualized treatment rule estimation methods are compatible with a multi-treatment observational study…

统计方法学 · 统计学 2019-11-14 Owen E. Leete , Nathan Kallus , Michael G. Hudgens , Sonia Napravnik , Michael R. Kosorok

Interference occurs when the treatment (or exposure) of one individual affects the outcomes of others. In some settings it may be reasonable to assume individuals can be partitioned into clusters such that there is no interference between…

统计方法学 · 统计学 2018-06-21 Lan Liu , Michael G. Hudgens , Bradley Saul , John D. Clemens , Mohammad Ali , Michael E. Emch

The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…

统计方法学 · 统计学 2025-10-23 Gopal Kotecha , Daniel E. Schwartz , Steffen Ventz , Lorenzo Trippa

The rapid expansion of large-scale electronic health record (EHR) data offers unique opportunities to improve the accuracy and efficiency of clinical risk estimation. Yet, because clinical events may occur outside the recording health…

统计方法学 · 统计学 2026-05-11 Jie Zhou , Enhao Wang , Xuan Wang