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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

With the advent of ubiquitous monitoring and measurement protocols, studies have started to focus more and more on complex, multivariate and heterogeneous datasets. In such studies, multivariate response variables are drawn from a…

统计方法学 · 统计学 2023-03-03 Saverio Ranciati , Veronica Vinciotti , Ernst C. Wit , Giuliano Galimberti

In the design of clinical trials, it is essential to assess the design operating characteristics (e.g., power and the type I error rate). Common practice for the evaluation of operating characteristics in Bayesian clinical trials relies on…

统计方法学 · 统计学 2026-03-17 Luke Hagar , Shirin Golchi

Analyses of cluster randomized trials (CRTs) can be complicated by informative missing outcome data. Methods such as inverse probability weighted generalized estimating equations have been proposed to account for informative missingness by…

统计方法学 · 统计学 2023-04-13 Chia-Rui Chang , Rui Wang

Two-stage randomized experiments are becoming an increasingly popular experimental design for causal inference when the outcome of one unit may be affected by the treatment assignments of other units in the same cluster. In this paper, we…

统计方法学 · 统计学 2022-10-21 Zhichao Jiang , Kosuke Imai , Anup Malani

In cluster-randomized trials, generalized linear mixed models and generalized estimating equations have conventionally been the default analytic methods for estimating the average treatment effect as routine practice. However, recent…

统计方法学 · 统计学 2025-09-19 Fan Li , Jiaqi Tong , Xi Fang , Chao Cheng , Brennan C. Kahan , Bingkai Wang

Multilevel models (mixed-effect models or hierarchical linear models) are now a standard approach to analysing clustered and longitudinal data in the social, behavioural and medical sciences. This review article focuses on multilevel linear…

统计方法学 · 统计学 2019-07-16 George Leckie

We consider experimentation in the presence of non-stationarity, inter-unit (spatial) interference, and carry-over effects (temporal interference), where we wish to estimate the global average treatment effect (GATE), the difference between…

统计理论 · 数学 2025-03-28 Su Jia , Nathan Kallus , Christina Lee Yu

Clinicians and researchers alike are increasingly interested in how best to personalize interventions. A dynamic treatment regimen (DTR) is a sequence of pre-specified decision rules which can be used to guide the delivery of a sequence of…

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

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

The Win Ratio has gained significant traction in cardiovascular trials as a novel method for analyzing composite endpoints (Pocock and others, 2012). Compared with conventional approaches based on time to the first event, the Win Ratio…

统计方法学 · 统计学 2024-10-10 Baoshan Zhang , Yuan Wu

In conventional randomized controlled trials, adjustment for baseline values of covariates known to be at least moderately associated with the outcome increases the power of the trial. Recent work has shown particular benefit for more…

统计方法学 · 统计学 2023-11-27 James Willard , Shirin Golchi , Erica EM Moodie

Adaptive approaches, allowing for more flexible trial design, have been proposed for individually randomized trials to save time or reduce sample size. However, adaptive designs for cluster-randomized trials in which groups of participants…

统计方法学 · 统计学 2022-01-10 Junwei Shen , Shirin Golchi , Erica E. M. Moodie , David Benrimoh

The dynamics associated with the time series of the iteration scheme of coupled cluster theory has been analysed. The phase space analysis indicates the presence of a few significant cluster amplitudes, mostly involving valence excitations,…

强关联电子 · 物理学 2021-02-24 Valay Agarawal , Samrendra Roy , Anish Chakraborty , Rahul Maitra

Cluster randomization trials commonly employ multiple endpoints. When a single summary of treatment effects across endpoints is of primary interest, global hypothesis testing/effect estimation methods represent a common analysis strategy.…

统计方法学 · 统计学 2025-05-19 E. Davies Smith , V. Jairath , G. Zou

We describe group sequential tests which efficiently incorporate information from multiple endpoints allowing for early stopping at pre-planned interim analyses. We formulate a testing procedure where several outcomes are examined, and…

统计方法学 · 统计学 2024-05-09 Abigail J. Burdon , Thomas Jaki

We describe a new family of coupling designs, extending the basic principle of stratified randomization to experiments with continuous, constrained multivariate, text/image and other irregular treatment spaces. Our approach is to first…

计量经济学 · 经济学 2026-04-14 Max Cytrynbaum , Fredrik Sävje

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

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