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Evaluating heterogeneity of treatment effects (HTE) across subgroups is common in both randomized trials and observational studies. Although several statistical challenges of HTE analyses including low statistical power and multiple…

统计方法学 · 统计学 2024-07-10 Noorie Hyun , Abisola E. Idu , Andrea J. Cook , Jennifer F. Bobb

Clustering and dependence are common in trials. For example, in some cluster randomized trials (CRTs), pre-existing clusters are enrolled, randomized, and serve as the basis of intervention delivery. Such CRTs are "fully clustered":…

Stepped-wedge cluster randomised trials (SW-CRTs) increasingly evaluate complex interventions, yet methodological guidance for analysing composite endpoints using generalized pairwise comparisons (GPC)remains limited. This work investigates…

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

In a clinical trial, the random allocation aims to balance prognostic factors between arms, preventing true confounders. However, residual differences due to chance may introduce near-confounders. Adjusting on prognostic factors is…

统计方法学 · 统计学 2024-11-18 Joe de Keizer , Rémi Lenain , Raphaël Porcher , Sarah Zoha , Arthur Chatton , Yohann Foucher

Win statistics have become increasingly popular for analyzing hierarchical composite endpoints in clinical trials, because they summarize treatment benefit through pairwise comparisons that respect the clinical importance order among…

统计方法学 · 统计学 2026-04-21 Xi Fang , Guangyu Tong , Yuan Huang , F. Perry Wilson , Patrick J. Heagerty , Fan Li

In epidemiological cohort studies, the relative risk (also known as risk ratio) is a major measure of association to summarize the results of two treatments or exposures. Generally, it measures the relative change in disease risk as a…

统计方法学 · 统计学 2022-07-05 Gopal Nath , Krishna K. Saha , Suojin Wang

Utilizing Bayesian methods in clinical trials has become increasingly popular, as they can incorporate historical data and expert opinions into the design and allow for smaller sample sizes to reduce costs while providing reliable and…

Background: When planning a cluster randomized trial, evaluators often have access to an enumerated cohort representing the target population of clusters. Practicalities of conducting the trial, such as the need to oversample clusters with…

统计方法学 · 统计学 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

Randomization is a common technique used in clinical trials to eliminate potential bias and confounders in a patient population. Equal allocation to treatment groups is the standard due to its optimal efficiency in many cases. However, in…

应用统计 · 统计学 2020-04-09 Thevaa Chandereng , Xiaodan Wei , Rick Chappell

The association between multidimensional exposure patterns and outcomes is commonly investigated by first applying cluster analysis algorithms to derive patterns and then estimating the associations. However, errors in the underlying…

统计方法学 · 统计学 2023-05-31 Timm Intemann , Iris Pigeot

Cluster-randomized trials (CRTs) are widely used to evaluate interventions delivered at the clinic, practice, or community level. Although standard analyses typically target average treatment effects, such summaries mask potentially…

统计方法学 · 统计学 2026-04-16 Changjun Li , Xi Fang , Michael O. Harhay , Andrew B. Forbes , F. Perry Wilson , Guangyu Tong , Fan Li

Clustered standard errors and approximate randomization tests are popular inference methods that allow for dependence within observations. However, they require researchers to know the cluster structure ex ante. We propose a procedure to…

计量经济学 · 经济学 2022-01-14 Yong Cai

In behavioral and psychiatric research, data consisting of a per-subject proportion of "successes" and "failures" over a finite number of trials often arise. This kind of clustered binary data are usually non-normally distributed, which can…

定量方法 · 定量生物学 2016-05-09 Alberto Ferrari , Mario Comelli

The intra-cluster correlation coefficient (ICC) plays an important role while designing the cluster randomized trials (CRTs). Often optimal CRTs are designed assuming that the magnitude of the ICC is constant across the clusters. However,…

统计计算 · 统计学 2019-02-21 Satya Prakash Singh , Pradeep Yadav

In the analysis of cluster randomized trials (CRTs), previous work has defined two meaningful estimands: the individual-average treatment effect (iATE) and cluster-average treatment effect (cATE) estimand, to address individual and…

Stepped wedge cluster randomized trials (SW-CRTs) with binary outcomes are increasingly used in prevention and implementation studies. Marginal models represent a flexible tool for analyzing SW-CRTs with population-averaged interpretations,…

统计方法学 · 统计学 2021-01-05 Fan Li , Hengshi Yu , Paul J. Rathouz , Elizabeth L. Turner , John S. Preisser

The radiological characterization of contaminated elements (walls, grounds, objects) from nuclear facilities often suffers from a too small number of measurements. In order to determine risk prediction bounds on the level of contamination,…

应用统计 · 统计学 2017-05-30 Géraud Blatman , Thibault Delage , Bertrand Iooss , Nadia Pérot

Determining the number of clusters is a fundamental issue in data clustering. Several algorithms have been proposed, including centroid-based algorithms using the Euclidean distance and model-based algorithms using a mixture of probability…

机器学习 · 计算机科学 2024-07-30 Ryosuke Motegi , Yoichi Seki

Cluster-randomized experiments are increasingly used to evaluate interventions in routine practice conditions, and researchers often adopt model-based methods with covariate adjustment in the statistical analyses. However, the validity of…

统计方法学 · 统计学 2023-12-08 Bingkai Wang , Chan Park , Dylan S. Small , Fan Li