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Cluster-randomized trials (CRTs) are a well-established class of designs for evaluating community-based interventions. An essential task in planning these trials is determining the number of clusters and cluster sizes needed to achieve…

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.…

Methodology · Statistics 2026-03-20 Hao Wang , Jingxia Liu , Drew B. Cameron , Jiaqi Tong , Donna Spiegelman , Daniella Meeker , Fan Li

When designing experimental studies with human participants, experimenters must decide how many trials each participant will complete, as well as how many participants to test. Most discussion of statistical power (the ability of a study…

Neurons and Cognition · Quantitative Biology 2021-08-30 Daniel H. Baker , Greta Vilidaite , Freya A. Lygo , Anika K. Smith , Tessa R. Flack , Andre D. Gouws , Timothy J. Andrews

Stepped wedge designs (SWDs) are designs for cluster randomized trials that feature staggered, unidirectional cross-over, typically from a control to a treatment condition. Existing literature on statistical power for SWDs primarily focuses…

Methodology · Statistics 2020-10-07 Phillip T. Sundin , Catherine M. Crespi

Pragmatic trials evaluating health care interventions often adopt cluster randomization due to scientific or logistical considerations. Previous reviews have shown that co-primary endpoints are common in pragmatic trials but infrequently…

Methodology · Statistics 2022-05-03 Siyun Yang , Mirjam Moerbeek , Monica Taljaard , Fan Li

We present an aggregation scheme that increases power in randomized controlled trials and quasi-experiments when the intervention possesses a robust and well-articulated theory of change. Longitudinal data analyzing interventions often…

Methodology · Statistics 2022-07-27 Timothy Lycurgus , Ben B. Hansen

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…

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

Methodology · Statistics 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

In epidemiological cohort studies, the relative risk (also known as risk ratio) is a major measure of association to summarize the results of two treatments or exposures. Generally, it measures the relative change in disease risk as a…

Methodology · Statistics 2022-07-05 Gopal Nath , Krishna K. Saha , Suojin Wang

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…

Methodology · Statistics 2026-03-09 Samuel I. Watson , James Martin

For randomized controlled trials (RCTs) with a single intervention being measured on multiple outcomes, researchers often apply a multiple testing procedure (such as Bonferroni or Benjamini-Hochberg) to adjust $p$-values. Such an adjustment…

Methodology · Statistics 2023-05-17 Kristen Hunter , Luke Miratrix , Kristin Porter

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

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…

Methodology · Statistics 2019-03-26 Jonathan Moyer , Ken Kleinman

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…

Methodology · Statistics 2022-12-09 Zach Branson , Xinran Li , Peng Ding

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

Hierarchical composite endpoints, such as those analyzed using the Finkelstein-Schoenfeld (FS) statistic, are increasingly used in clinical trials for their ability to incorporate clinically prioritized outcomes. However, adaptive design…

Methodology · Statistics 2025-04-22 Krishna Padmanabhan , Cyrus Mehta

The reproducibility crisis has led to an increasing number of replication studies being conducted. Sample sizes for replication studies are often calculated using conditional power based on the effect estimate from the original study.…

Methodology · Statistics 2022-10-19 Charlotte Micheloud , Leonhard Held

Win statistics have gained increasing popularity as primary analysis methods for clinical trials with hierarchical endpoints (HEs) as primary endpoints. However, existing sample size and power calculation approaches in trial design still…

Methodology · Statistics 2026-05-19 Baoshan Zhang , Huiman X. Barnhart , Yuan Wu , Roland A. Matsouaka

As single-cell gene expression data analysis continues to grow, the need for reliable clustering methods has become increasingly important. The prevalence of heuristic means for method choice could lead to inaccurate reports if…

Quantitative Methods · Quantitative Biology 2026-05-19 Owen Visser , Somnath Datta

The primary goal of randomized trials is to compare the effects of different interventions on some outcome of interest. In addition to the treatment assignment and outcome, data on baseline covariates, such as demographic characteristics or…

Applications · Statistics 2014-01-09 Alisa J. Stephens , Eric J. Tchetgen Tchetgen , Victor De Gruttola