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Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

统计方法学 · 统计学 2026-01-22 Xinyuan Chen , Fan Li

This paper provides asymptotically valid tests for the null hypothesis of no treatment effect heterogeneity. Importantly, I consider the presence of heterogeneity that is not explained by observed characteristics, or so-called idiosyncratic…

计量经济学 · 经济学 2023-04-04 Jaime Ramirez-Cuellar

This paper proposes a novel approach for estimating treatment effects in panel data settings, addressing key limitations of the standard difference-in-differences (DID) approach. The standard approach relies on the parallel trends…

计量经济学 · 经济学 2026-01-14 Shoya Ishimaru

Treatment effect heterogeneity refers to the systematic variation in treatment effects across subgroups. There is an increasing need for clinical trials that aim to investigate treatment effect heterogeneity and estimate subgroup-specific…

统计方法学 · 统计学 2026-03-06 Xianglin Zhao , Shirin Golchi , Jean-Philippe Gouin , Kaberi Dasgupta

Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

统计方法学 · 统计学 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

Estimating heterogeneous treatment effects (HTEs) is crucial for precision medicine. While multiple studies can improve the generalizability of results, leveraging them for estimation is statistically challenging. Existing approaches often…

统计方法学 · 统计学 2025-12-22 Cathy Shyr , Boyu Ren , Prasad Patil , Giovanni Parmigiani

Understanding treatment heterogeneity is essential to the development of precision medicine, which seeks to tailor medical treatments to subgroups of patients with similar characteristics. One of the challenges to achieve this goal is that…

统计方法学 · 统计学 2019-08-21 Shujie Ma , Jian Huang , Zhiwei Zhang , Mingming Liu

Multi-Regional Clinical Trials (MRCTs) play a central role in the development of new therapies by enabling the simultaneous evaluation of drug efficacy and safety across diverse global populations. Assessing the consistency of treatment…

应用统计 · 统计学 2026-02-10 Kunhai Qing , Xinru Ren , Jin Xu , Menggang Yu

Randomized trials and observational studies, more often than not, run over a certain period of time. The treatment effect evolves during this period which provides crucial insights into the treatment response and the long-term effects. Many…

统计方法学 · 统计学 2020-04-13 Shu Li , Peter Bühlmann

Many clinical questions involve estimating the effects of multiple treatments using observational data. When using longitudinal data, the interest is often in the effect of treatment strategies that involve sustaining treatment over time.…

统计方法学 · 统计学 2024-05-03 Emily Granger , Gwyneth Davies , Ruth H. Keogh

The average treatment effect (ATE) is popularly used to assess the treatment effect. However, the ATE implicitly assumes a homogenous treatment effect even amongst individuals with different characteristics. In this paper, we mainly focus…

统计方法学 · 统计学 2016-03-10 Yunjian Yin , Lan Liu , Zhi Geng

A new meta-algorithm for estimating the conditional average treatment effects is proposed in the paper. The main idea underlying the algorithm is to consider a new dataset consisting of feature vectors produced by means of concatenation of…

机器学习 · 统计学 2019-09-10 Lev V. Utkin , Mikhail V. Kots , Viacheslav S. Chukanov

We study treatment-effect estimation using panel data. The treatment may be non-binary, non-absorbing, and the outcome may be affected by treatment lags. We make a parallel-trends assumption, and propose event-study estimators of the effect…

计量经济学 · 经济学 2026-05-13 Clément de Chaisemartin , Xavier D'Haultfœuille

Integrating data from multiple heterogeneous sources has become increasingly popular to achieve a large sample size and diverse study population. This paper reviews development in causal inference methods that combines multiple datasets…

统计方法学 · 统计学 2021-10-05 Xu Shi , Ziyang Pan , Wang Miao

Patients often discontinue treatment in a clinical trial because their health condition is not improving. Consequently, the patients still in the study at the end of the trial have better health outcomes on average than the initial patient…

统计方法学 · 统计学 2022-06-06 Alex Ocampo , Heinz Schmidli , Peter Quarg , Francesca Callegari , Marcello Pagano

In a randomized control trial, the precision of an average treatment effect estimator can be improved either by collecting data on additional individuals, or by collecting additional covariates that predict the outcome variable. We propose…

统计方法学 · 统计学 2017-09-27 Pedro Carneiro , Sokbae Lee , Daniel Wilhelm

Evaluating the treatment effects has become an important topic for many applications. However, most existing literature focuses mainly on the average treatment effects. When the individual effects are heavy-tailed or have outlier values,…

统计方法学 · 统计学 2023-05-11 Yongchang Su , Xinran Li

To test scientific theories and develop individualized treatment rules, researchers often wish to learn heterogeneous treatment effects that can be consistently found across diverse populations and contexts. We consider the problem of…

统计方法学 · 统计学 2026-05-01 Yi Zhang , Melody Huang , Kosuke Imai

We study the problem of treatment effect estimation in randomized experiments with high-dimensional covariate information, and show that essentially any risk-consistent regression adjustment can be used to obtain efficient estimates of the…

统计方法学 · 统计学 2022-06-08 Stefan Wager , Wenfei Du , Jonathan Taylor , Robert Tibshirani

Subgroup analyses of randomized controlled trials (RCTs) constitute an important component of the drug development process in precision medicine. In particular, subgroup analyses of early-stage trials often influence the design and…

统计方法学 · 统计学 2025-02-12 Daniel Schwartz , Riddhiman Saha , Steffen Ventz , Lorenzo Trippa