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Multivariate outcomes are common in pragmatic cluster randomized trials. While sample size calculation procedures for multivariate outcomes exist under parallel assignment, none have been developed for a stepped wedge design. In this…

统计方法学 · 统计学 2022-12-05 Kendra Davis-Plourde , Monica Taljaard , Fan Li

In this article, we develop methods for sample size and power calculations in four-level intervention studies when intervention assignment is carried out at any level, with a particular focus on cluster randomized trials (CRTs). CRTs…

统计方法学 · 统计学 2022-09-07 Xueqi Wang , Elizabeth L. Turner , John S. Preisser , Fan Li

Composite endpoints are increasingly used in clinical trials to capture treatment effects across multiple or hierarchically ordered outcomes. Although inference procedures based on win statistics, such as the win ratio, win odds, and net…

统计方法学 · 统计学 2025-10-28 Xi Fang , Zhiqiang Cao , Fan Li

Cluster randomized trials with measurements at baseline can improve power over post-test only designs by using difference in difference designs. However, subjects may be lost to follow-up between the baseline and follow-up periods. While…

统计方法学 · 统计学 2019-03-26 Jonathan Moyer , Ken Kleinman

Hybrid studies allow investigators to simultaneously study an intervention effectiveness outcome and an implementation research outcome. In particular, type 2 hybrid studies support research that places equal importance on both outcomes…

统计方法学 · 统计学 2024-11-15 Melody A. Owen , Geoffrey M. Curran , Justin D. Smith , Yacob Tedla , Chao Cheng , Donna Spiegelman

Power and sample size calculations for Wald tests in generalized linear models (GLMs) are often limited to specific cases like logistic regression. More general methods typically require detailed study parameters that are difficult to…

统计方法学 · 统计学 2026-01-21 Amy L Cochran , Shijie Yuan , Paul J Rathouz

A stepped wedge cluster randomized trial is a type of longitudinal cluster design that sequentially switches clusters to intervention over time until all clusters are treated. While the traditional posttest-only parallel design requires…

应用统计 · 统计学 2021-01-05 Fan Li

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

统计方法学 · 统计学 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

Marginal structural models fit via inverse probability of treatment weighting are commonly used to control for confounding when estimating causal effects from observational data. When planning a study that will be analyzed with marginal…

应用统计 · 统计学 2020-03-16 Bonnie E. Shook-Sa , Michael G. Hudgens

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

Generalized linear mixed models (GLMM) are commonly used to analyze clustered data, but when the number of clusters is small to moderate, standard statistical tests may produce elevated type I error rates. Small-sample corrections have been…

统计方法学 · 统计学 2023-11-07 Hongxiang Qiu , Andrea J. Cook , Jennifer F. Bobb

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

Simulation offers a simple and flexible way to estimate the power of a clinical trial when analytic formulae are not available. The computational burden of using simulation has, however, restricted its application to only the simplest of…

统计方法学 · 统计学 2020-12-04 Duncan T. Wilson , Rebecca E. A. Walwyn , Richard Hooper , Julia Brown , Amanda J. Farrin

A stepped wedge design is a unidirectional crossover design where clusters are randomized to distinct treatment sequences. While model-based analysis of stepped wedge designs is standard practice to evaluate treatment effects accounting for…

统计方法学 · 统计学 2024-09-13 Bingkai Wang , Xueqi Wang , Fan Li

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

When observations are organized into groups where commonalties exist amongst them, the dependent random measures can be an ideal choice for modeling. One of the propositions of the dependent random measures is that the atoms of the…

机器学习 · 统计学 2016-06-28 Cheng Luo , Richard Yi Da Xu , Yang Xiang

Power analyses are an important aspect of experimental design, because they help determine how experiments are implemented in practice. It is common to specify a desired level of power and compute the sample size necessary to obtain that…

统计方法学 · 统计学 2022-12-09 Zach Branson , Xinran Li , Peng Ding

This paper describes an approach to simultaneously identify clusters and estimate cluster-specific regression parameters from the given data. Such an approach can be useful in learning the relationship between input and output when the…

统计金融 · 定量金融 2024-01-02 Udai Nagpal , Krishan Nagpal

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

Selective inference aims at providing valid inference after a data-driven selection of models or hypotheses. It is essential to avoid overconfident results and replicability issues. While significant advances have been made in this area for…

统计方法学 · 统计学 2025-03-14 Matteo D'Alessandro , Magne Thoresen
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