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Difference-in-differences (DiD) is a cornerstone of causal inference, yet extending it to functional outcomes is not a routine scalar generalization; rather, it entails three fundamental challenges in identification, inference, and…

统计方法学 · 统计学 2026-05-29 Junzhu Nie , Chengxiu Ling , Mengfei Ran

Recurrent events often serve as key endpoints in clinical studies but may be prematurely truncated by terminal events such as death, creating selection bias and complicating causal inference. To address this challenge, we develop a Bayesian…

统计方法学 · 统计学 2026-03-18 Yuki Ohnishi , Michael O. Harhay , Guangyu Tong , Fan Li

In situations where it is difficult to enroll patients in randomized controlled trials, external data can improve efficiency and feasibility. In such cases, adaptive trial designs could be used to decrease enrollment in the control arm of…

统计方法学 · 统计学 2020-10-02 Brian D. Segal , W. Katherine Tan

To precisely define the treatment effect of interest in a clinical trial, the ICH E9 estimand addendum describes that relevant so-called intercurrent events should be identified and strategies specified to deal with them. Handling…

统计方法学 · 统计学 2025-02-06 Camila Olarte Parra , Rhian M. Daniel , Jonathan W. Bartlett

Clinical trials traditionally employ blinding as a design mechanism to reduce the influence of placebo effects. In practice, however, it can be difficult or impossible to blind study participants and unblinded trials are common in medical…

应用统计 · 统计学 2016-06-22 Elias Chaibub Neto

Estimating externally valid causal effects is a foundational problem in the social and biomedical sciences. Generalizing or transporting causal estimates from an experimental sample to a target population of interest relies on an overlap…

统计方法学 · 统计学 2024-03-29 Melody Huang

A common practice in clinical trials is to evaluate a treatment effect on an intermediate endpoint when the true outcome of interest would be difficult or costly to measure. We consider how to validate intermediate endpoints in a…

统计方法学 · 统计学 2022-11-30 Emily K. Roberts , Michael R. Elliott , Jeremy M. G. Taylor

Evaluating treatment effect heterogeneity widely informs treatment decision making. At the moment, much emphasis is placed on the estimation of the conditional average treatment effect via flexible machine learning algorithms. While these…

统计方法学 · 统计学 2021-05-07 Lihua Lei , Emmanuel J. Candès

This paper develops new methods for causal inference in observational studies on a single large network of interconnected units, addressing two key challenges: long-range dependence among units and the presence of general interference. We…

统计方法学 · 统计学 2025-11-24 Jizhou Liu , Dake Zhang , Eric J. Tchetgen Tchetgen

Randomized controlled trials (RCTs) are the gold standard for evaluating causal effects but are often costly and difficult to scale; consequently, they are frequently augmented with auxiliary external controls in many applications. Prior…

统计方法学 · 统计学 2026-05-28 Jiawei Shan , Yiteng Tu , Guanbo Wang , Chao Ying , Jiwei Zhao

We develop new methods to integrate experimental and observational data in causal inference. While randomized controlled trials offer strong internal validity, they are often costly and therefore limited in sample size. Observational data,…

计量经济学 · 经济学 2025-11-04 Xuelin Yang , Licong Lin , Susan Athey , Michael I. Jordan , Guido W. Imbens

We propose a doubly robust estimator for the average treatment effect in high dimensional low sample size observational studies, where contamination and model misspecification pose serious inferential challenges. The estimator combines…

统计方法学 · 统计学 2025-11-04 Byeonghee Lee , Sangwook Kang , Ju-Hyun Park , Saebom Jeon , Joonsung Kang

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

Background: The E-value has become widely used for assessing robustness to unmeasured confounding in observational studies, but the original framework was developed for single time-point exposure-outcome settings. This study extends the…

应用统计 · 统计学 2026-03-02 Md. Niamul Islam Sium

Measuring treatment effects in observational studies is challenging because of confounding bias. Confounding occurs when a variable affects both the treatment and the outcome. Traditional methods such as propensity score matching estimate…

统计方法学 · 统计学 2021-12-23 Bevan I. Smith , Charles Chimedza

This paper develops a unified framework for estimating continuous outcomes under multiple treatment levels in observational studies. We integrate the Generalized Propensity Score (GPS), Covariate Balancing Propensity Score (CBPS), and…

统计方法学 · 统计学 2025-09-22 Byeonghee Lee , Joonsung Kang

Recent work has made important contributions in the development of causally-interpretable meta-analysis. These methods transport treatment effects estimated in a collection of randomized trials to a target population of interest. Ideally,…

统计方法学 · 统计学 2023-02-08 Justin M. Clark , Kollin W. Rott , James S. Hodges , Jared D. Huling

In this paper, we develop new methods for estimating average treatment effects in observational studies, focusing on settings with more than two treatment levels under unconfoundedness given pre-treatment variables. We emphasize…

统计方法学 · 统计学 2017-10-11 Shu Yang , Guido W. Imbens , Zhanglin Cui , Douglas Faries , Zbigniew Kadziola

In classical study designs, the aim is often to learn about the effects of a treatment or intervention on a single outcome; in many modern studies, however, data on multiple outcomes are collected and it is of interest to explore effects on…

统计方法学 · 统计学 2017-06-15 Edward H. Kennedy , Shreya Kangovi , Nandita Mitra

Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes…

统计方法学 · 统计学 2021-12-21 Shuxi Zeng , Fan Li , Liangyuan Hu , Fan Li