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Response-adaptive randomization (RAR) has been studied extensively in conventional, single-stage clinical trials, where it has been shown to yield ethical and statistical benefits, especially in trials with many treatment arms. However, RAR…

Methodology · Statistics 2024-01-09 Peter Norwood , Marie Davidian , Eric Laber

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…

Methodology · Statistics 2024-09-19 Sarah E. Robertson , Jon A. Steingrimsson , Issa J. Dahabreh

Cluster randomized trials (CRTs) randomly assign an intervention to groups of individuals (e.g., clinics or communities) and measure outcomes on individuals in those groups. While offering many advantages, this experimental design…

There has been significant attention given to developing data-driven methods for tailoring patient care based on individual patient characteristics. Dynamic treatment regimes formalize this through a sequence of decision rules that map…

Methodology · Statistics 2022-02-22 Eric J. Rose , Erica E. M. Moodie , Susan Shortreed

Adaptive interventions (AIs) are increasingly becoming popular in medical and behavioral sciences. An AI is a sequence of individualized intervention options that specify for whom and under what conditions different intervention options…

Applications · Statistics 2018-12-18 Palash Ghosh , Inbal Nahum-Shani , Bonnie Spring , Bibhas Chakraborty

Randomized experiments ensure robust causal inference that are critical to effective learning analytics research and practice. However, traditional randomized experiments, like A/B tests, are limiting in large scale digital learning…

Applications · Statistics 2019-02-04 Timothy NeCamp , Josh Gardner , Christopher Brooks

The sequential multiple assignment randomized trial (SMART) is the gold standard trial design to generate data for the evaluation of multi-stage treatment regimes. As with conventional (single-stage) randomized clinical trials, interim…

Methodology · Statistics 2023-09-13 Cole Manschot , Eric Laber , Marie Davidian

Paired cluster-randomized experiments (pCRTs) are common across many disciplines because there is often natural clustering of individuals, and paired randomization can help balance baseline covariates to improve experimental precision.…

Methodology · Statistics 2024-07-03 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

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…

Methodology · Statistics 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

This paper studies inference in cluster randomized trials where treatment status is determined according to a "matched pairs" design. Here, by a cluster randomized experiment, we mean one in which treatment is assigned at the level of the…

Econometrics · Economics 2025-08-14 Yuehao Bai , Jizhou Liu , Azeem M. Shaikh , Max Tabord-Meehan

Cluster or group randomized trials (CRTs) are increasingly used for both behavioral and system-level interventions, where entire clusters are randomly assigned to a study condition or intervention. Apart from the assigned cluster-level…

Methodology · Statistics 2024-11-19 Shubhadeep Chakraborty , Bo Wang , Ram Tiwari , Samiran Ghosh

Dynamic treatment regimes (DTR) are sequential decision rules corresponding to several stages of intervention. Each rule maps patients' covariates to optional treatments. The optimal dynamic treatment regime is the one that maximizes the…

Methodology · Statistics 2023-10-10 Zhishuai Liu , Zishu Zhan , Jian Liu , Danhui Yi , Cunjie Lin , Yufei Yang

Complete randomization balances covariates on average, but covariate imbalance often exists in finite samples. Rerandomization can ensure covariate balance in the realized experiment by discarding the undesired treatment assignments. Many…

Methodology · Statistics 2022-07-07 Xin Lu , Tianle Liu , Hanzhong Liu , Peng Ding

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":…

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…

Methodology · Statistics 2022-01-10 Junwei Shen , Shirin Golchi , Erica E. M. Moodie , David Benrimoh

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

Numerous publications have now addressed the principles of designing, analyzing, and reporting the results of, stepped-wedge cluster randomized trials. In contrast, there is little research available pertaining to the design and analysis of…

Methodology · Statistics 2018-06-29 Michael Grayling , Adrian Mander , James Wason

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…

Methodology · Statistics 2025-10-10 Xi Fang , Bingkai Wang , Liangyuan Hu , Fan Li

We address estimation of intervention effects in experimental designs in which (a) interventions are assigned at the cluster level; (b) clusters are selected to form pairs, matched on observed characteristics; and (c) intervention is…

Methodology · Statistics 2014-11-24 Zhenke Wu , Constantine E. Frangakis , Thomas A. Louis , Daniel O. Scharfstein

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…

Methodology · Statistics 2026-04-16 Changjun Li , Xi Fang , Michael O. Harhay , Andrew B. Forbes , F. Perry Wilson , Guangyu Tong , Fan Li