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Understanding whether and how treatment effects vary across subgroups is crucial to inform clinical practice and recommendations. Accordingly, the assessment of heterogeneous treatment effects (HTE) based on pre-specified potential effect…

统计方法学 · 统计学 2023-12-04 Bryan S. Blette , Scott D. Halpern , Fan Li , Michael O. Harhay

We propose a framework for testing the homogeneity of conditional average treatment effects (CATEs) across multiple experimental and observational studies. Our approach leverages multiple randomized trials to assess whether treatment…

计量经济学 · 经济学 2026-02-25 Ana Armendariz , Martin Huber

Sample size determination for cluster randomised trials (CRTs) is challenging as it requires robust estimation of the intra-cluster correlation coefficient (ICC). Typically, the sample size is chosen to provide a certain level of power to…

应用统计 · 统计学 2023-08-23 S. Faye Williamson , Svetlana V. Tishkovskaya , Kevin J. Wilson

Before embarking on data collection, researchers typically compute how many individual observations they should do. This is vital for doing studies with sufficient statistical power, and often a cornerstone in study pre-registrations and…

统计方法学 · 统计学 2023-09-06 Edwin S Dalmaijer

Identifying heterogeneous treatment effects (HTEs) in randomized controlled trials is an important step toward understanding and acting on trial results. However, HTEs are often small and difficult to identify, and HTE modeling methods…

统计方法学 · 统计学 2020-11-24 Erin Craig , Donald A Redelmeier , Robert J Tibshirani

Randomized controlled trials (RCTs) are considered the gold standard for estimating the average treatment effect (ATE) of interventions. One use of RCTs is to study the causes of global poverty -- a subject explicitly cited in the 2019…

机器学习 · 计算机科学 2023-05-26 Connor T. Jerzak , Fredrik Johansson , Adel Daoud

The ability to accurately estimate the sample size required by a stepped-wedge (SW) cluster randomized trial (CRT) routinely depends upon the specification of several nuisance parameters. If these parameters are mis-specified, the trial…

统计方法学 · 统计学 2017-10-10 Michael Grayling , Adrian Mander , James Wason

Cluster algorithms are increasingly popular in biomedical research due to their compelling ability to identify discrete subgroups in data, and their increasing accessibility in mainstream software. While guidelines exist for algorithm…

机器学习 · 统计学 2021-05-26 E. S. Dalmaijer , C. L. Nord , D. E. Astle

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

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

Longitudinal cluster randomized trials (L-CRTs) are increasingly used to evaluate the cost-effectiveness of healthcare interventions across multiple assessment periods, yet design methods for powering these trials remain underdeveloped.…

统计方法学 · 统计学 2026-03-20 Hao Wang , Jingxia Liu , Drew B. Cameron , Jiaqi Tong , Donna Spiegelman , Daniella Meeker , Fan Li

In this study, we focus on estimating the heterogeneous treatment effect (HTE) for survival outcome. The outcome is subject to censoring and the number of covariates is high-dimensional. We utilize data from both the randomized controlled…

统计方法学 · 统计学 2025-03-21 Xin Ye , Shu Yang , Xiaofei Wang , Yanyan Liu

The credibility revolution advances the use of research designs that permit identification and estimation of causal effects. However, understanding which mechanisms produce measured causal effects remains a challenge. The dominant current…

计量经济学 · 经济学 2026-04-08 Jiawei Fu , Tara Slough

Statistical power is often a concern for clustered RCTs due to variance inflation from design effects and the high cost of adding study clusters (such as hospitals, schools, or communities). While covariate pre-specification is the…

统计方法学 · 统计学 2020-05-07 Peter Z. Schochet

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

The heterogeneity of treatment effect (HTE) lies at the heart of precision medicine. Randomized controlled trials are gold-standard for treatment effect estimation but are typically underpowered for heterogeneous effects. In contrast, large…

统计方法学 · 统计学 2024-11-14 Shu Yang , Siyi Liu , Donglin Zeng , Xiaofei Wang

In cluster randomized controlled trials (CRCT) with a finite populations, the exact design-based variance of the Horvitz-Thompson (HT) estimator for the average treatment effect (ATE) depends on the joint distribution of unobserved…

计量经济学 · 经济学 2025-12-17 Yue Fang , Geert Ridder

Robust estimation of heterogeneous treatment effects is a fundamental challenge for optimal decision-making in domains ranging from personalized medicine to educational policy. In recent years, predictive machine learning has emerged as a…

机器学习 · 统计学 2025-06-23 Maximilian Schuessler , Erik Sverdrup , Robert Tibshirani

Stepped wedge cluster randomized controlled trials are typically analyzed using models that assume the full effect of the treatment is achieved instantaneously. We provide an analytical framework for scenarios in which the treatment effect…

统计方法学 · 统计学 2025-09-25 Avi Kenny , Emily Voldal , Fan Xia , Patrick J. Heagerty , James P. Hughes

Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account…

统计方法学 · 统计学 2022-11-29 Angela Y. Zhu , Nandita Mitra , Karla Hemming , Michael O. Harhay , Fan Li