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Missingness in variables that define study eligibility criteria is a seldom addressed challenge in electronic health record (EHR)-based settings. It is typically the case that patients with incomplete eligibility information are excluded…

Observational studies provide the only evidence on the effectiveness of interventions when randomized controlled trials (RCTs) are impractical due to cost, ethical concerns, or time constraints. While many methodologies aim to draw causal…

Missing data, inaccuracies in medication lists, and recording delays in electronic health records (EHR) are major limitations for target trial emulation (TTE), the process by which EHR data are used to retrospectively emulate a randomized…

Applications · Statistics 2025-01-16 Max Sunog , Colin Magdamo , Marie-Laure Charpignon , Mark Albers

Although increasingly used for research, electronic health records (EHR) often lack gold-standard assessment of key data elements. Linking EHRs to other data sources with higher-quality measurements can improve statistical inference, but…

Methodology · Statistics 2025-03-05 Jenny Shen , Dane Isenberg , Kristin A. Linn , Rebecca A. Hubbard

Randomised controlled trials (RCTs) are regarded as the gold standard for estimating causal treatment effects on health outcomes. However, RCTs are not always feasible, because of time, budget or ethical constraints. Observational data such…

Methodology · Statistics 2024-02-20 Li Su , Roonak Rezvani , Shaun R. Seaman , Colin Starr , Isaac Gravestock

Causal inference on the average treatment effect (ATE) using non-probability samples, such as electronic health records (EHR), faces challenges from sample selection bias and high-dimensional covariates. This requires considering a…

Methodology · Statistics 2024-03-28 Jiacong Du , Xu Shi , Donglin Zeng , Bhramar Mukherjee

We consider the problem of estimating the average treatment effect (ATE) in a semi-supervised learning setting, where a very small proportion of the entire set of observations are labeled with the true outcome but features predictive of the…

Methodology · Statistics 2020-10-27 David Cheng , Ashwin Ananthakrishnan , Tianxi Cai

Background: Target trial emulation (TTE) that applies trial design principles to improve the analysis of non-randomized studies is increasingly being used. Applications of TTE to emulate cluster randomized trials (RCTs) have been limited.…

Applications · Statistics 2025-12-17 Shomoita Alam , Nathaniel Dyrkton , Susan Shepherd , Ibrahim Sana , Kevin Phelan , Jay JH Park

Causal inference methods based on electronic health record (EHR) databases must simultaneously handle confounding and missing data. Vast scholarship exists aimed at addressing these two issues separately, but surprisingly few papers attempt…

Methodology · Statistics 2025-07-28 Luke Benz , Alexander Levis , Sebastien Haneuse

Many real-world Electronic Health Record (EHR) data contains a large proportion of missing values. Leaving substantial portion of missing information unaddressed usually causes significant bias, which leads to invalid conclusion to be…

Machine Learning · Computer Science 2020-11-04 Lucas J. Liu , Hongwei Zhang , Jianzhong Di , Jin Chen

Using administrative patient-care data such as Electronic Health Records (EHR) and medical/ pharmaceutical claims for population-based scientific research has become increasingly common. With vast sample sizes leading to very small standard…

Methodology · Statistics 2023-08-21 Ritoban Kundu , Xu Shi , Jean Morrison , Jessica Barrett , Bhramar Mukherjee

We study randomized experiments in bipartite systems where only a subset of treatment-side units are eligible for assignment while all units continue to interact, generating interference. We formalize eligibility-constrained bipartite…

Methodology · Statistics 2025-11-17 Albert Tan , Mohsen Bayati , James Nordlund , Roman Istomin

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

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…

Methodology · Statistics 2026-04-16 Qinwei Yang , Jingyi Li , Peng Wu , Shu Yang

Target trial emulation (TTE) enables causal questions to be studied with observational data when randomized controlled trials (RCTs) are infeasible. Yet treatment-effect methods often address causal estimation, missingness, and temporal…

Electronic health records (EHR) data provide a cost and time-effective opportunity to conduct cohort studies of the effects of multiple time-point interventions in the diverse patient population found in real-world clinical settings.…

Quantifying the heterogeneity of treatment effect is important for understanding how a commercial product or medical treatment affects different population subgroups. While much of treatment effect heterogeneity analysis focuses on the…

Understanding whether and how treatment effects vary across subgroups is crucial to inform clinical practice and recommendations. Accordingly, the assessment of heterogeneous treatment effects (HTE) based on pre-specified potential effect…

Methodology · Statistics 2023-12-04 Bryan S. Blette , Scott D. Halpern , Fan Li , Michael O. Harhay

The ability to predict individualized treatment effects (ITEs) based on a given patient's profile is essential for personalized medicine. We propose a hypothesis testing approach to choosing between two potential treatments for a given…

Methodology · Statistics 2020-08-11 Tianxi Cai , Tony Cai , Zijian Guo

We study the problem of selecting the best heterogeneous treatment effect (HTE) estimator from a collection of candidates in settings where the treatment effect is fundamentally unobserved. We cast estimator selection as a multiple testing…

Machine Learning · Statistics 2025-11-25 Jiayi Guo , Zijun Gao
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