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Unmeasured confounding and selection bias are often of concern in observational studies and may invalidate a causal analysis if not appropriately accounted for. Under outcome-dependent sampling, a latent factor that has causal effects on…

Methodology · Statistics 2022-08-03 Kendrick Qijun Li , Xu Shi , Wang Miao , Eric Tchetgen Tchetgen

Cluster-randomized trials (CRTs) are widely used to evaluate group-level interventions and increasingly collect multiple outcomes capturing complementary dimensions of benefit and risk. Investigators often seek a single global summary of…

Methodology · Statistics 2026-01-22 Xinyuan Chen , Fan Li

Electronic health records and other sources of observational data are increasingly used for drawing causal inferences. The estimation of a causal effect using these data not meant for research purposes is subject to confounding and…

Methodology · Statistics 2023-04-19 Janie Coulombe , Shu Yang

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…

Methodology · Statistics 2023-04-13 Chia-Rui Chang , Rui Wang

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

Contextual sensing and delivery of digital interventions to improve health outcomes have gained significant traction in behavioral and psychiatric studies. Micro-randomized trials (MRTs) are a common experimental design for obtaining…

Methodology · Statistics 2025-04-01 Jieru Shi , Zhenke Wu , Walter Dempsey

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…

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…

Methodology · Statistics 2016-08-18 Audrey Boruvka , Daniel Almirall , Katie Witkiewitz , Susan A. Murphy

When extending inferences from a randomized trial to a new target population, the transportability condition for conditional difference effect measures is invoked to identify the marginal causal mean difference in the target population.…

Methodology · Statistics 2024-07-18 Guanbo Wang , Alexander Levis , Jon Steingrimsson , Issa Dahabreh

Augmenting the control arm in clinical trials with external data can improve statistical power for demonstrating treatment effects. In many time-to-event outcome trials, participants are subject to truncation by death. Direct application of…

Methodology · Statistics 2025-06-24 Zehao Su , Helene C. W. Rytgaard , Henrik Ravn , Frank Eriksson

Doubly robust estimators of causal effects are a popular means of estimating causal effects. Such estimators combine an estimate of the conditional mean of the outcome given treatment and confounders (the so-called outcome regression) with…

Methodology · Statistics 2019-01-17 David Benkeser , Weixin Cai , Mark J van der Laan

We consider the conditional treatment effect for competing risks data in observational studies. While it is described as a constant difference between the hazard functions given the covariates, we do not assume specific functional forms for…

Applications · Statistics 2021-12-28 Denise Rava , Ronghui Xu

Just-in-time adaptive interventions (JITAIs) are time-varying adaptive interventions that use frequent opportunities for the intervention to be adapted--weekly, daily, or even many times a day. The micro-randomized trial (MRT) has emerged…

Randomized controlled trials (RCT's) allow researchers to estimate causal effects in an experimental sample with minimal identifying assumptions. However, to generalize or transport a causal effect from an RCT to a target population,…

Methodology · Statistics 2022-02-08 Melody Huang

Causal mediation analysis in cluster-randomized trials (CRTs) is complicated by the presence of multiple mediators, intracluster correlation, and within-cluster interference. Existing mediation methods often fall short in accommodating…

Methodology · Statistics 2026-04-14 Jiaqi Tong , Chao Cheng , Fan Li

This paper aims to provide practitioners of causal mediation analysis with a better understanding of estimation options. We take as inputs two familiar strategies (weighting and model-based prediction) and a simple way of combining them…

This paper develops a multiply robust (MR) dose-response estimator for causal inference problems involving multivalued treatments. We combine a family of generalised propensity score (GPS) models and a family of outcome regression (OR)…

Methodology · Statistics 2017-05-18 Cian Naik , Emma J. McCoy , Daniel J. Graham

Treatment effect estimates are often available from randomized controlled trials as a single average treatment effect for a certain patient population. Estimates of the conditional average treatment effect (CATE) are more useful for…

Methodology · Statistics 2023-09-12 Wouter A. C. van Amsterdam , Rajesh Ranganath

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

In this paper, we propose a robust method to estimate the average treatment effects in observational studies when the number of potential confounders is possibly much greater than the sample size. We first use a class of penalized…

Methodology · Statistics 2018-12-21 Yang Ning , Sida Peng , Kosuke Imai