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In the statistical literature, a number of methods have been proposed to ensure valid inference about marginal effects of variables on a longitudinal outcome in settings with irregular monitoring times. However, the potential biases due to…

统计方法学 · 统计学 2021-12-23 Janie Coulombe , Erica E M Moodie , Robert W Platt

Inferring causal effects from an observational study is challenging because participants are not randomized to treatment. Observational studies in infectious disease research present the additional challenge that one participant's treatment…

统计方法学 · 统计学 2020-12-25 Brian G. Barkley , Michael G. Hudgens , John D. Clemens , Mohammad Ali , Michael E. Emch

In longitudinal studies where units are embedded in space or a social network, interference may arise, meaning that a unit's outcome can depend on treatment histories of others. The presence of interference poses significant challenges for…

统计方法学 · 统计学 2025-08-26 Ye Wang , Michael Jetsupphasuk

In mobile health interventions aimed at behavior change and maintenance, treatments are provided in real time to manage current or impending high risk situations or promote healthy behaviors in near real time. Currently there is great…

统计方法学 · 统计学 2016-08-18 Audrey Boruvka , Daniel Almirall , Katie Witkiewitz , Susan A. Murphy

This article investigates the model-robustness of fixed-effects models for analyzing a broad class of longitudinal cluster trials (CTs) such as stepped-wedge, parallel-with-baseline and crossover designs, encompassing both randomized (CRTs)…

统计方法学 · 统计学 2026-04-10 Kenneth M. Lee , Fan Li

Background: Stepped wedge cluster randomized trials (SW-CRTs) involve sequential measurements within clusters over time. Initially, all clusters start in the control condition before crossing over to the intervention on a staggered…

统计方法学 · 统计学 2026-01-21 Jale Basten , Katja Ickstadt , Nina Timmesfeld

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,…

统计方法学 · 统计学 2021-01-05 Fan Li , Hengshi Yu , Paul J. Rathouz , Elizabeth L. Turner , John S. Preisser

In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

统计方法学 · 统计学 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

The micro-randomized trial (MRT) is an experimental design that can be used to develop optimal mobile health interventions. In MRTs, interventions in the form of notifications or messages are sent through smart phones to individuals,…

统计方法学 · 统计学 2022-02-14 Shuangning Li , Stefan Wager

Mobile health is a rapidly developing field in which behavioral treatments are delivered to individuals via wearables or smartphones to facilitate health-related behavior change. Micro-randomized trials (MRT) are an experimental design for…

统计方法学 · 统计学 2019-05-13 Tianchen Qian , Predrag Klasnja , Susan A. Murphy

In stepped wedge cluster randomized trials (SW-CRTs), observations collected under the control condition are, on average, from an earlier time than observations collected under the intervention condition. In a cohort design, participants…

统计方法学 · 统计学 2023-02-23 Jale Basten , Katja Ickstadt , Nina Timmesfeld

Across research disciplines, cluster randomized trials (CRTs) are commonly implemented to evaluate interventions delivered to groups of participants, such as communities and clinics. Despite advances in the design and analysis of CRTs,…

Causal excursion effect (CEE) characterizes the effect of an intervention under policies that deviate from the experimental policy. It is widely used to study the effect of time-varying interventions that have the potential to be frequently…

统计方法学 · 统计学 2024-06-14 Zhaoxi Cheng , Lauren Bell , Tianchen Qian

In causal inference, it is common to estimate the causal effect of a single treatment variable on an outcome. However, practitioners may also be interested in the effect of simultaneous interventions on multiple covariates of a fixed target…

统计方法学 · 统计学 2022-11-24 Jaime Roquero Gimenez , Dominik Rothenhäusler

Analyses of cluster randomized trials (CRTs) can be complicated by informative missing outcome data. Methods such as inverse probability weighted generalized estimating equations have been proposed to account for informative missingness by…

统计方法学 · 统计学 2023-04-13 Chia-Rui Chang , Rui Wang

In this paper we propose a new template for empirical studies intended to assess causal effects: the outcome-wide longitudinal design. The approach is an extension of what is often done to assess the causal effects of a treatment or…

统计方法学 · 统计学 2018-10-25 Tyler J. VanderWeele , Maya B. Mathur , Ying Chen

In experiments that study social phenomena, such as peer influence or herd immunity, the treatment of one unit may influence the outcomes of others. Such "interference between units" violates traditional approaches for causal inference, so…

统计方法学 · 统计学 2023-08-30 David Choi

Stepped wedge cluster-randomized trial (CRTs) designs randomize clusters of individuals to intervention sequences, ensuring that every cluster eventually transitions from a control period to receive the intervention under study by the end…

统计方法学 · 统计学 2025-02-19 Alessandro Gasparini , Michael J. Crowther , Emiel O. Hoogendijk , Fan Li , Michael O. Harhay

In randomized controlled trials (RCTs) that focus on time-to-event outcomes, intercurrent events can arise in two ways: as semi-competing events, which modify the hazard of the primary outcome events, or as competing events, which make the…

统计方法学 · 统计学 2026-05-26 Yuhao Deng , Shasha Han , Xiao-Hua Zhou

This paper shows how to use a randomized saturation experimental design to identify and estimate causal effects in the presence of spillovers--one person's treatment may affect another's outcome--and one-sided non-compliance--subjects can…