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Marginalized models are in great demand by most researchers in the life sciences particularly in clinical trials, epidemiology, health-economics, surveys and many others since they allow generalization of inference to the entire population…

Methodology · Statistics 2016-10-26 Samuel Iddi , Kwabena Doku-Amponsah

Existing statistical methods for the analysis of micro-randomized trials (MRTs) are designed to estimate causal excursion effects using data from a single MRT. In practice, however, researchers can often find previous MRTs that employ…

Methodology · Statistics 2025-05-13 Easton Huch , Inbal Nahum-Shani , Lindsey Potter , Cho Lam , David W. Wetter , Walter Dempsey

Stepped wedge cluster randomized trials (SW-CRTs) with binary outcomes are increasingly used in prevention and implementation studies. Marginal models represent a flexible tool for analyzing SW-CRTs with population-averaged interpretations,…

Methodology · Statistics 2021-01-05 Fan Li , Hengshi Yu , Paul J. Rathouz , Elizabeth L. Turner , John S. Preisser

Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account…

Methodology · Statistics 2022-11-29 Angela Y. Zhu , Nandita Mitra , Karla Hemming , Michael O. Harhay , Fan Li

Cluster sampling is common in survey practice, and the corresponding inference has been predominantly design-based. We develop a Bayesian framework for cluster sampling and account for the design effect in the outcome modeling. We consider…

Methodology · Statistics 2020-06-24 Susanna Makela , Yajuan Si , Andrew Gelman

The increasing interest in subpopulation analysis has led to the development of various new trial designs and analysis methods in the fields of personalized medicine and targeted therapies. In this paper, subpopulations are defined in terms…

Methodology · Statistics 2020-12-01 Roland Gerard Gera , Tim Friede

We propose a unified probabilistic framework for sparse count tensors with excess zeros, motivated by single-cell Hi-C data. The observed data are naturally represented as a three-way tensor indexed by genomic loci pairs and cells,…

Methodology · Statistics 2026-04-27 Elena Tuzhilina , Yaoming Zhen

Cluster randomized trials (CRTs) often enroll large numbers of participants, but due to logistical and fiscal challenges, only a subset of participants may be selected for measurement of certain outcomes, and those sampled may, purposely or…

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…

In the analysis of cluster randomized trials (CRTs), previous work has defined two meaningful estimands: the individual-average treatment effect (iATE) and cluster-average treatment effect (cATE) estimand, to address individual and…

The generalized estimating equation (GEE) method is a popular tool for longitudinal data analysis. However, GEE produces biased estimates when the outcome of interest is associated with cluster size, a phenomenon known as informative…

Methodology · Statistics 2025-01-03 Yue Ma , Haofeng Wang , Xuejun Jiang

Cluster randomized trials (CRTs) are popular in public health and in the social sciences to evaluate a new treatment or policy where the new policy is randomly allocated to clusters of units rather than individual units. CRTs often feature…

Methodology · Statistics 2019-08-16 Hyunseung Kang , Luke Keele

Evaluating heterogeneity of treatment effects (HTE) across subgroups is common in both randomized trials and observational studies. Although several statistical challenges of HTE analyses including low statistical power and multiple…

Methodology · Statistics 2024-07-10 Noorie Hyun , Abisola E. Idu , Andrea J. Cook , Jennifer F. Bobb

We propose a regression model for count data when the classical generalized linear model approach is too rigid due to a high outcome of zero counts and a nonlinear influence of continuous covariates. Zero-Inflation is applied to take into…

Methodology · Statistics 2013-04-12 T. Opitz , P. Tramini , N. Molinari

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

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

Small sample sizes in clinical studies arises from factors such as reduced costs, limited subject availability, and the rarity of studied conditions. This creates challenges for accurately calculating confidence intervals (CIs) using the…

Methodology · Statistics 2025-11-11 Mulan Wu , Mengyu Xu , Dongyun Kim

Clustering with variable selection is a challenging yet critical task for modern small-n-large-p data. Existing methods based on sparse Gaussian mixture models or sparse K-means provide solutions to continuous data. With the prevalence of…

Machine Learning · Statistics 2020-04-28 Tanbin Rahman , Yujia Li , Tianzhou Ma , Lu Tang , George Tseng

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

In a clinical trial, the random allocation aims to balance prognostic factors between arms, preventing true confounders. However, residual differences due to chance may introduce near-confounders. Adjusting on prognostic factors is…

Methodology · Statistics 2024-11-18 Joe de Keizer , Rémi Lenain , Raphaël Porcher , Sarah Zoha , Arthur Chatton , Yohann Foucher