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With increasing data availability, causal effects can be evaluated across different data sets, both randomized controlled trials (RCTs) and observational studies. RCTs isolate the effect of the treatment from that of unwanted (confounding)…

Longitudinal studies are frequently used in medical research and involve collecting repeated measures on individuals over time. Observations from the same individual are invariably correlated and thus an analytic approach that accounts for…

Existing statistical methods for the analysis of micro-randomized trials (MRTs) are designed to estimate causal excursion effects using data from a single MRT. In practice, however, researchers can often find previous MRTs that employ…

Methodology · Statistics 2025-05-13 Easton Huch , Inbal Nahum-Shani , Lindsey Potter , Cho Lam , David W. Wetter , Walter Dempsey

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

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…

The World Health Organization defines cardio-vascular disease (CVD) as "a group of disorders of the heart and blood vessels," including coronary heart disease and stroke (WHO 21). CVD is affected by "intermediate risk factors" such as…

Machine Learning · Computer Science 2023-05-04 Harish Chauhan , Nikunj Gupta , Zoe Haskell-Craig

Randomized controlled trials (RCTs) have been the cornerstone of clinical evidence; however, their cost, duration, and restrictive eligibility criteria limit power and external validity. Studies using real-world data (RWD), historically…

Methodology · Statistics 2025-11-26 Shu Yang , Margaret Gamalo , Haoda Fu

Machine learning systems trained on electronic health records (EHRs) increasingly guide treatment decisions, but their reliability depends on the critical assumption that patients follow the prescribed treatments recorded in EHRs. Using EHR…

Machine Learning · Computer Science 2025-04-22 Zhongyuan Liang , Arvind Suresh , Irene Y. Chen

Clinical prediction models must be developed using sufficiently large datasets to minimise overfitting and ensure robust predictive performance. Existing sample size calculations assume complete predictor data for all included participants,…

Missing data is a common concern in health datasets, and its impact on good decision-making processes is well documented. Our study's contribution is a methodology for tackling missing data problems using a combination of synthetic dataset…

Machine Learning · Computer Science 2022-11-08 Gift Khangamwa , Terence L. van Zyl , Clint J. van Alten

We evaluate the performance of targeted maximum likelihood estimation (TMLE) for estimating the average treatment effect in missing data scenarios under varying levels of positivity violations. We employ model- and design-based simulations,…

Methodology · Statistics 2026-05-12 Christoph Wiederkehr , Christian Heumann , Michael Schomaker

Evaluating treatment effects is critical in clinical trials but sometimes involves lengthy, invasive, or costly follow-up procedures. In these cases, surrogate markers, which provide intermediate measures of the long-term treatment effect,…

Methodology · Statistics 2026-03-24 Sarah C. Lotspeich , P. D. Anh. Nguyen , Layla Parast

Introduction: Accounting for missing data by imputing or weighting conditional on covariates relies on the variable with missingness being observed at least some of the time for all unique covariate values. This requirement is referred to…

Applications · Statistics 2025-10-16 Paul N Zivich , Bonnie E Shook-Sa , Stephen R Cole , Eric T Lofgren , Jessie K Edwards

Missing data is a ubiquitous problem. It is especially challenging in medical settings because many streams of measurements are collected at different - and often irregular - times. Accurate estimation of those missing measurements is…

Machine Learning · Computer Science 2017-11-27 Jinsung Yoon , William R. Zame , Mihaela van der Schaar

Missing data poses a significant challenge in data science, affecting decision-making processes and outcomes. Understanding what missing data is, how it occurs, and why it is crucial to handle it appropriately is paramount when working with…

Methodology · Statistics 2024-04-09 Youran Zhou , Sunil Aryal , Mohamed Reda Bouadjenek

Understanding causality should be a core requirement of any attempt to build real impact through AI. Due to the inherent unobservability of counterfactuals, large randomised trials (RCTs) are the standard for causal inference. But large…

The ICH E9(R1) Addendum (International Council for Harmonization 2019) suggests treatment-policy as one of several strategies for addressing intercurrent events such as treatment withdrawal when defining an estimand. This strategy requires…

Methodology · Statistics 2023-08-28 Suzie Cro , James H Roger , James R Carpenter

Methods that infer causal dependence from observational data are central to many areas of science, including medicine, economics, and the social sciences. A variety of theoretical properties of these methods have been proven, but empirical…

Methodology · Statistics 2021-07-08 Amanda Gentzel , Purva Pruthi , David Jensen

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

Electronic health records (EHR) consist of longitudinal clinical observations portrayed with sparsity, irregularity, and high-dimensionality, which become major obstacles in drawing reliable downstream clinical outcomes. Although there…

Machine Learning · Computer Science 2020-11-17 Ahmad Wisnu Mulyadi , Eunji Jun , Heung-Il Suk
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