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This paper studies inference in cluster randomized trials where treatment status is determined according to a "matched pairs" design. Here, by a cluster randomized experiment, we mean one in which treatment is assigned at the level of the…

计量经济学 · 经济学 2025-08-14 Yuehao Bai , Jizhou Liu , Azeem M. Shaikh , Max Tabord-Meehan

In cluster-randomized trials (CRTs), entire clusters of individuals are randomized to treatment, and outcomes within a cluster are typically correlated. While frequentist approaches are standard practice for CRT analysis, Bayesian methods…

统计方法学 · 统计学 2025-11-27 Ruyi Liu , Joshua L. Warren , Yuki Ohnishi , Donna Spiegelman , Liangyuan Hu , Fan Li

In cluster-randomized trials (CRTs), there is emerging interest in exploring the causal mechanism in which a cluster-level treatment affects the outcome through an intermediate outcome. The majority of existing causal mediation methods are…

统计方法学 · 统计学 2026-01-12 Chao Cheng , Fan Li

Finding patient subgroups with similar characteristics is crucial for personalized decision-making in various disciplines such as healthcare and policy evaluation. While most existing approaches rely on unsupervised clustering methods,…

机器学习 · 统计学 2026-03-06 Luwei Wang , Nazir Lone , Sohan Seth

Estimating causal effects under interference is pertinent to many real-world settings. Recent work with low-order potential outcomes models uses a rollout design to obtain unbiased estimators that require no interference network…

统计方法学 · 统计学 2025-02-12 Mayleen Cortez-Rodriguez , Matthew Eichhorn , Christina Lee Yu

Heterogeneous treatment effects (HTEs) are commonly identified during randomized controlled trials (RCTs). Identifying subgroups of patients with similar treatment effects is of high interest in clinical research to advance precision…

机器学习 · 计算机科学 2022-12-06 Peniel N. Argaw , Elizabeth Healey , Isaac S. Kohane

Non-adherence to assigned treatment is a common issue in cluster randomised trials (CRTs). In these settings, the efficacy estimand may be also of interest. Many methodological contributions in recent years have advocated using instrumental…

统计方法学 · 统计学 2018-12-05 Schadrac C. Agbla , Bianca De Stavola , Karla DiazOrdaz

Cluster randomized trials (CRTs) with multiple unstructured mediators present significant methodological challenges for causal inference due to within-cluster correlation, interference among units, and the complexity introduced by multiple…

统计方法学 · 统计学 2025-04-21 Yuki Ohnishi , Fan Li

Causal mediation analysis in cluster-randomized trials (CRTs) is complicated by the presence of multiple mediators, intracluster correlation, and within-cluster interference. Existing mediation methods often fall short in accommodating…

统计方法学 · 统计学 2026-04-14 Jiaqi Tong , Chao Cheng , Fan Li

In multicenter randomized trials, when effect modifiers have a different distribution across centers, comparisons between treatment groups that average over centers may not apply to any of the populations underlying the individual centers.…

Subgroup analysis is a frequently used tool for evaluating heterogeneity of treatment effect and heterogeneity in treatment harm across observed baseline patient characteristics. While treatment efficacy and adverse event measures are often…

应用统计 · 统计学 2018-08-14 Nicholas C. Henderson , Ravi Varadhan

Cluster randomized trials are widely used when individual randomization is logistically infeasible or when correlations between observations cannot be ignored, especially in fields such as ophthalmology, infectious disease, vaccine…

统计方法学 · 统计学 2026-05-04 Wanying Shao , Toshimitsu Hamasaki , Scott Evans , Guoqing Diao

Stepped wedge cluster randomized trials (SW-CRTs) have become increasingly popular and are used for a variety of interventions and outcomes, often chosen for their feasibility advantages. SW-CRTs must account for time trends in the outcome…

统计方法学 · 统计学 2024-07-16 Lee Kennedy-Shaffer , Victor De Gruttola , Marc Lipsitch

In stepped wedge cluster randomized trials (SW-CRTs), the intervention is rolled out to clusters over multiple periods. A standard approach for analyzing SW-CRTs utilizes the linear mixed model, where the treatment effect is only present…

In stepped wedge cluster randomized trials (SW-CRTs), observations collected under the control condition are, on average, from an earlier time than observations collected under the intervention condition. In a cohort design, participants…

统计方法学 · 统计学 2023-02-23 Jale Basten , Katja Ickstadt , Nina Timmesfeld

Precision medicine is an emerging field that takes into account individual heterogeneity to inform better clinical practice. In clinical trials, the evaluation of treatment effect heterogeneity is an important component, and recently, many…

统计方法学 · 统计学 2023-02-24 Yuejia Xu , Angela M. Wood , Brian D. M. Tom

Cluster randomized trials (CRTs) offer a practical alternative for addressing logistical challenges and ensuring feasibility in community health, education, and prevention studies, even though randomized controlled trials are considered the…

统计方法学 · 统计学 2025-10-30 Jooyeon Lee , M. S. , Evan Kwiatkowski , Ph. D

Adaptive sample size re-estimation, early stopping, and trial re-design at interim analyses can reduce expected sample sizes in randomised trials. Cluster randomised trials, in which groups of participants are randomly allocated to…

统计方法学 · 统计学 2026-03-09 Samuel I. Watson , James Martin

This article investigates the model-robustness of fixed-effects models for analyzing a broad class of longitudinal cluster trials (CTs) such as stepped-wedge, parallel-with-baseline and crossover designs, encompassing both randomized (CRTs)…

统计方法学 · 统计学 2026-04-10 Kenneth M. Lee , Fan Li

Subgroup selection in clinical trials is essential for identifying patient groups that react differently to a treatment, thereby enabling personalised medicine. In particular, subgroup selection can identify patient groups that respond…