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The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

Matching on covariates is a well-established framework for estimating causal effects in observational studies. The principal challenge stems from the often high-dimensional structure of the problem. Many methods have been introduced to…

Methodology · Statistics 2022-07-12 Florian Gunsilius , Yuliang Xu

We consider the problem of estimating the effects of a binary treatment on a continuous outcome of interest from observational data in the absence of confounding by unmeasured factors. We provide a new estimator of the population average…

Methodology · Statistics 2020-08-04 James Robins , Mariela Sued , Quanhong Lei-Gomez , Andrea Rotnitzky

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,…

Large-scale human mobility exhibits spatial and temporal patterns that can assist policymakers in decision making. Although traditional prediction models attempt to capture these patterns, they often interfered by non-periodic public…

Machine Learning · Computer Science 2025-04-17 Xiaojie Yang , Hangli Ge , Jiawei Wang , Zipei Fan , Renhe Jiang , Ryosuke Shibasaki , Noboru Koshizuka

Recently a new experimental approach, the hybrid experimental design (HED), was introduced to enable investigators to answer scientific questions about building behavioral interventions in which human-delivered and digital components are…

Methodology · Statistics 2026-02-26 Mengbing Li , Inbal Nahum-Shani , Walter Dempsey

In longitudinal studies using routinely collected data, such as electronic health records (EHRs), patients tend to have more measurements when they are unwell; this informative observation pattern may lead to bias. While semi-parametric…

Methodology · Statistics 2024-10-02 Rose Garrett , Brian Feldman , Eleanor Pullenayegum

In this article, we propose novel structural nested models to estimate causal effects of continuous treatments based on mobile health data. To find the treatment regime that optimizes the expected short-term outcomes for patients, we define…

Methodology · Statistics 2020-07-24 Liangyu Zhu , Wenbin Lu , Rui Song

Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

Methodology · Statistics 2018-06-12 Rose Baker , Dan Jackson

Recent research in causal inference has made important progress in addressing challenges to the external validity of trial findings. Such methods weight trial participant data to more closely resemble the distribution of effect-modifying…

Methodology · Statistics 2024-07-18 Justin M. Clark , Kollin W. Rott , James S. Hodges , Jared D. Huling

Marginal structural models (MSMs) are often used to estimate causal effects of treatments on survival time outcomes from observational data when time-dependent confounding may be present. They can be fitted using, e.g., inverse probability…

Methodology · Statistics 2023-12-27 Shaun R Seaman , Ruth H Keogh

Often in follow-up studies intermediate events occur in some patients, such as reinterventions or adverse events. These intermediate events directly affect the shapes of their longitudinal profiles. Our work is motivated by two studies in…

While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision…

Machine Learning · Statistics 2020-04-22 Steve Yadlowsky , Fabio Pellegrini , Federica Lionetto , Stefan Braune , Lu Tian

Machine learning (ML) models are increasingly used as decision-support tools in high-risk domains. Evaluating the causal impact of deploying such models can be done with a randomized controlled trial (RCT) that randomizes users to ML vs.…

Methodology · Statistics 2025-07-17 Jacob M. Chen , Michael Oberst

Motivated by a study about prompt coronary angiography in myocardial infarction, we propose a method to estimate the causal effect of a treatment in two-arm experimental studies with possible non-compliance in both treatment and control…

Statistics Theory · Mathematics 2012-10-26 F. Bartolucci , A. Farcomeni

When estimating causal effects, it is important to assess external validity, i.e., determine how useful a given study is to inform a practical question for a specific target population. One challenge is that the covariate distribution in…

Methodology · Statistics 2025-01-03 Zhenghao Zeng , Edward H. Kennedy , Lisa M. Bodnar , Ashley I. Naimi

We propose a nonlinear difference-in-differences method to estimate multivariate counterfactual distributions in classical treatment and control study designs with observational data. Our approach sheds a new light on existing approaches…

Methodology · Statistics 2024-03-11 William Torous , Florian Gunsilius , Philippe Rigollet

Predicting the effect of interventions with many possible variations, e.g., therapeutic content that affects mental health outcomes or an earnings call transcript that drives movement in share price, is useful across several domains.…

Machine Learning · Computer Science 2026-05-27 Nikita Dhawan , Arnav Paruthi , Andrew Kim , Lovedeep Gondara , Jekaterina Novikova , Chris J. Maddison

We extend Fisher's randomization test (FRT) to test conditional independence between observed outcomes and treatments given covariates in both randomized experiments and observational studies, with no restriction on the variable type of…

Methodology · Statistics 2025-06-12 Zhen Zhong

We recently developed a new method riAFT-BART to draw causal inferences about population treatment effect on patient survival from clustered and censored survival data while accounting for the multilevel data structure. The practical…

Methodology · Statistics 2023-08-14 Liangyuan Hu
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