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Designing Cluster Randomized Trials

A Practical Guide for Investigators

Bibliographic Data

ID9104105
AuthorsKendra Davis‐Plourde (0000-0001-6654-0710, Department of Biostatistics, Yale School of Public Health, New Haven, CT), Kendra Davis-Plourde (Yale University, corresponding author), Xiaoying Yu (0000-0003-1659-2365, Department of Biostatistics and Data Science, University of Texas Medical Branch at Galveston, Galveston, TX), Jeanne A Teresi (Columbia University Stroud Center at New York State Psychiatric Institute, New York, NY)
Year2026
Volume64
Issue4
Pages179-191
Publication date2026-04-01
Peer ReviewedYes
Open AccessNo
TypeARTICLE
VenueMedical Care (JOURNAL)
Journal identifiersISSN: 0025-7079 • E-ISSN: 1537-1948
PublisherOvid Technologies (Wolters Kluwer Health) (PUBLISHER)
DOI10.1097/mlr.0000000000002283
PMID41733160
OpenAlexW7131270729
LanguageEN
Citations received1
References cited38

BACKGROUND: An important consideration in the design stage of randomized controlled trials is whether individuals within each site should or can be randomized to study arms (an individually randomized controlled trial) or whether entire sites should be randomized (a cluster randomized trial) where the clusters are sites. Recently, cluster randomized trials have grown in popularity; however, investigators have expressed a need for guidelines related to selection of the best design-especially when considering more complex designs such as stepped wedge. This commentary attempts to provide such guidance. METHODS: In this commentary, we address common misconceptions surrounding the appropriate use of cluster randomization and crossover designs (such as the stepped wedge design). The intracluster correlation coefficients for different levels of clustering are presented, and the impact of different designs on sample size discussed. We provide practical recommendations that are accessible to investigators from all disciplines, regardless of their level of statistical training. On the basis of these recommendations, we present a flowchart to help researchers identify a suitable trial design for their study. In addition, we provide a table of commonly used statistical definitions related to cluster randomized trials and a table of design considerations for more complex designs to support effective consultation with a statistician during the design phase. CONCLUSION: On the basis of these guidelines, it is recommended that researchers prioritize the use of individual randomization and a parallel trial design when the goal of the study is to assess the efficacy or effectiveness of an intervention

Cluster (spacecraft) · Cluster randomised controlled trial · MEDLINE · Randomization · Randomized controlled trial · Research design · Meta-analysis and systematic reviews · Optimal Experimental Design Methods · Statistical Methods in Clinical Trials

  • Development, validation, and cost-effectiveness analysis of an AI-assisted three-tiered glaucoma screening model in a community-based setting

    Open Access•Xiangyu Fu, Jiaying Zhang et al.•Frontiers in Public Health•2026

  • The stepped wedge cluster randomised trial

    Open Access•Karla Hemming, Timothy Haines et al.•BMJ•2015

  • Consort 2010 statement

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  • The PRECIS-2 tool

    Open Access•Kirsty Loudon, Shaun Treweek et al.•BMJ•2015

  • Intraclass Correlation Coefficients for Planning Cluster Randomized Trials in Community-Dwelling Older Adults

    Open Access•Subashan Perera, Neelesh K Nadkarni et al.•Journal of Aging and Health•2018

  • Pitfalls of and Controversies in Cluster Randomization Trials

    Allan Donner, Neil Klar•American Journal of Public Health•2004

  • Individually Randomized Group Treatment Trials

    Sherri L Pals, Sherri Pals et al.•American Journal of Public Health•2008

Unique citing works1
Citations per year1
Citation span2026 - 2026 (1)
Citation velocitycurrent
Highly citedNo
Citation typesNeutral: 1
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