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Stepped wedge designs (SWDs) are increasingly used to evaluate longitudinal cluster-level interventions but pose substantial challenges for valid inference. Because crossover times are randomized, intervention effects are intrinsically…

Methodology · Statistics 2026-05-12 Fan Xia , K. C. Gary Chan , Emily Voldal , Avi Kenny , Patrick J. Heagerty , James P. Hughes

Causal identification of treatment effects for infectious disease outcomes in interconnected populations is challenging because infection outcomes may be transmissible to others, and treatment given to one individual may affect others'…

Methodology · Statistics 2021-05-11 Xiaoxuan Cai , Eben Kenah , Forrest W. Crawford

Randomized saturation designs are a family of designs which assign a possibly different treatment proportion to each cluster of a population at random. As a result, they generalize the well-known (stratified) completely randomized designs…

Methodology · Statistics 2022-03-21 Chencheng Cai , Jean Pouget-Abadie , Edoardo M. Airoldi

Recent advances in engineering technologies have enabled the collection of a large number of longitudinal features. This wealth of information presents unique opportunities for researchers to investigate the complex nature of diseases and…

Methodology · Statistics 2023-11-27 Zihang Lu , Noirrit Kiran Chandra

Background: In settings where proof-of-principle trials have succeeded but the effectiveness of different forms of implementation remains uncertain, trials that not only generate information about intervention effects but also provide…

Quantitative Methods · Quantitative Biology 2017-05-16 Guy Harling , Rui Wang , Jukka-Pekka Onnela , Victor De Gruttola

Controlled experiments are widely used in many applications to investigate the causal relationship between input factors and experimental outcomes. A completely randomized design is usually used to randomly assign treatment levels to…

Methodology · Statistics 2026-05-12 Yiou Li , Lulu Kang , Xiao Huang

We examine study designs for extending (generalizing or transporting) causal inferences from a randomized trial to a target population. Specifically, we consider nested trial designs, where randomized individuals are nested within a sample…

Given two 2-level factors of interest, a 2^2 split-plot design} (a) takes each of the $2^2=4$ possible factorial combinations as a treatment, (b) identifies one factor as `whole-plot,' (c) divides the experimental units into blocks, and (d)…

Methodology · Statistics 2016-02-15 Anqi Zhao , Peng Ding , Tirthankar Dasgupta

Stepped wedge cluster randomized trials (SWCRTs) often face challenges with potential confounding by time trends. Traditional frequentist methods can fail to provide adequate coverage of the intervention's true effect using confidence…

Methodology · Statistics 2024-02-02 Danni Wu , Hyung G. Park , Corita R. Grudzen , Keith S. Goldfeld

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.…

Methodology · Statistics 2025-05-19 E. Davies Smith , V. Jairath , G. Zou

Variance reduction for causal inference in the presence of network interference is often achieved through either outcome modeling, typically analyzed under unit-randomized Bernoulli designs, or clustered experimental designs, typically…

Methodology · Statistics 2026-01-19 Matthew Eichhorn , Samir Khan , Johan Ugander , Christina Lee Yu

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…

Methodology · Statistics 2026-05-04 Wanying Shao , Toshimitsu Hamasaki , Scott Evans , Guoqing Diao

Digital firms routinely run many online experiments on shared user populations. When product decisions are compositional, such as combinations of interface elements, flows, messages, or incentives, the number of feasible interventions grows…

Machine Learning · Statistics 2026-04-13 Xin Wen , Xi Chen , Will Wei Sun , Yichen Zhang

Statisticians increasingly face the problem to reconsider the adaptability of classical inference techniques. In particular, divers types of high-dimensional data structures are observed in various research areas; disclosing the boundaries…

Statistics Theory · Mathematics 2017-06-09 Paavo Sattler , Markus Pauly

In many health policy settings, adaptive interventions target a population of clusters (e.g., schools), with the ultimate intent of impacting outcomes at the level of individuals within the clusters. Health policy researchers can use…

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…

Methodology · Statistics 2024-09-13 Bingkai Wang , Xueqi Wang , Fan Li

Instrumental variable methods are widely used in medical and social science research to draw causal conclusions when the treatment and outcome are confounded by unmeasured confounding variables. One important feature of such studies is that…

Methodology · Statistics 2021-05-25 Bo Zhang , Siyu Heng , Emily J. MacKay , Ting Ye

In pragmatic cluster randomized controlled trials (PCRCTs), healthcare providers are randomized while both providers and patients may deviate from the assigned intervention. In many PCRCTs, cluster-level implementation is measured using…

Applications · Statistics 2026-04-29 Anthony Sisti , Ellen McCreedy , Roee Gutman

While palliative care is increasingly commonly delivered to hospitalized patients with serious illnesses, few studies have estimated its causal effects. Courtright et al. (2016) adopted a cluster-randomized stepped-wedge design to assess…

Many scientific questions in biomedical, environmental, and psychological research involve understanding the effects of multiple factors on outcomes. While factorial experiments are ideal for this purpose, randomized controlled treatment…

Methodology · Statistics 2025-12-03 Ruoqi Yu , Peng Ding
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