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Not only does mobile health technology enable researchers to track changes in multiple longitudinal outcomes of interest and to record the occurrence of health-related events over time, but it also allows for the delivery of repeated…

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,…

Methodology · Statistics 2022-02-14 Shuangning Li , Stefan Wager

Empirical studies using Regression Discontinuity (RD) designs often explore heterogeneous treatment effects based on pretreatment covariates, even though no formal statistical methods exist for such analyses. This has led to the widespread…

Randomized clinical trials typically aim to estimate a marginal treatment effect. While covariate adjustment can improve precision, it may change the estimand in nonlinear models due to noncollapsibility, leading to conditional rather than…

Methodology · Statistics 2026-05-25 Leticia Wuethrich , Torsten Hothorn

In longitudinal studies, time-varying covariates are often endogenous, meaning their values depend on both their own history and that of the outcome variable. This violates key assumptions of Generalized Linear Mixed Effects Models (GLMMs),…

The micro-randomized trial (MRT) is a sequential randomized experimental design to empirically evaluate the effectiveness of mobile health (mHealth) intervention components that may be delivered at hundreds or thousands of decision points.…

Methodology · Statistics 2021-12-14 Jieru Shi , Zhenke Wu , Walter Dempsey

Joint models for a wide class of response variables and longitudinal measurements consist on a mixed-effects model to fit longitudinal trajectories whose random effects enter as covariates in a generalized linear model for the primary…

Methodology · Statistics 2014-07-03 Rolando De la Cruz , Cristian Meza , Ana Arribas-Gil , Raymond J. Carroll

There is a growing interest in leveraging the prevalence of mobile technology to improve health by delivering momentary, contextualized interventions to individuals' smartphones. A just-in-time adaptive intervention (JITAI) adjusts to an…

Other Statistics · Statistics 2018-12-31 Nicholas J. Seewald , Shawna N. Smith , Andy Jinseok Lee , Predrag Klasnja , Susan A. Murphy

Multi-Regional Clinical Trials (MRCTs) play a central role in the development of new therapies by enabling the simultaneous evaluation of drug efficacy and safety across diverse global populations. Assessing the consistency of treatment…

Applications · Statistics 2026-02-10 Kunhai Qing , Xinru Ren , Jin Xu , Menggang Yu

To optimize mobile health interventions and advance domain knowledge on intervention design, it is critical to understand how the intervention effect varies over time and with contextual information. This study aims to assess how a push…

Applications · Statistics 2024-10-22 Jiaxin Yu , Tianchen Qian

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

Methodology · Statistics 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes

A dynamic treatment regimen (DTR) is a pre-specified sequence of decision rules which maps baseline or time-varying measurements on an individual to a recommended intervention or set of interventions. Sequential multiple assignment…

Methodology · Statistics 2019-10-23 Brook Luers , Min Qian , Inbal Nahum-Shani , Connie Kasari , Daniel Almirall

This paper studies treatment effect models in which individuals are classified into unobserved groups based on heterogeneous treatment rules. Using a finite mixture approach, we propose a marginal treatment effect (MTE) framework in which…

Econometrics · Economics 2022-05-24 Tadao Hoshino , Takahide Yanagi

This paper investigates the identification and inference of treatment effects in randomized controlled trials with social interactions. Two key network features characterize the setting and introduce endogeneity: (1) latent variables may…

Econometrics · Economics 2024-12-04 Mengsi Gao

Advances in wearables and digital technology now make it possible to deliver behavioral mobile health interventions to individuals in their everyday life. The micro-randomized trial (MRT) is increasingly used to provide data to inform the…

Methodology · Statistics 2020-07-30 Tianchen Qian , Hyesun Yoo , Predrag Klasnja , Daniel Almirall , Susan A. Murphy

In randomized trials, repeated measures of the outcome are routinely collected. The mixed model for repeated measures (MMRM) leverages the information from these repeated outcome measures, and is often used for the primary analysis to…

Methodology · Statistics 2023-07-20 Bingkai Wang , Yu Du

Construction of just-in-time adaptive interventions, such as prompts delivered by mobile apps to promote and maintain behavioral change, requires knowledge about time-varying moderated effects to inform when and how we deliver intervention…

Methodology · Statistics 2022-12-06 Jieru Shi , Zhenke Wu , Walter Dempsey

Mixed Models for Repeated Measures (MMRMs) are ubiquitous when analyzing outcomes of clinical trials. However, the linearity of the fixed-effect structure in these models largely restrict their use to estimating treatment effects that are…

Methodology · Statistics 2023-01-23 Lars Lau Raket

Precision medicine incorporates patient-level covariates to tailor treatment decisions, seeking to improve outcomes. In longitudinal studies with time-varying covariates and sequential treatment decisions, precision medicine can be…

Methodology · Statistics 2020-08-05 Dylan Spicker , Michael Wallace

Multi-regional clinical trials (MRCTs) enable efficient global drug development by assessing treatment effects across regions within a single protocol. While powered for overall efficacy, MRCTs are typically not designed to provide…

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