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Increased availability of electronic health records (EHR) has enabled researchers to study various medical questions. Cohort selection for the hypothesis under investigation is one of the main consideration for EHR analysis. For uncommon…

Machine Learning · Computer Science 2020-05-14 Mohamed Ghalwash , Zijun Yao , Prithwish Chakrabotry , James Codella , Daby Sow

Given an (optimal) dynamic treatment rule, it may be of interest to evaluate that rule -- that is, to ask the causal question: what is the expected outcome had every subject received treatment according to that rule? In this paper, we study…

Methodology · Statistics 2021-10-27 Lina Montoya , Jennifer Skeem , Mark van der Laan , Maya Petersen

Background --The objective of this study was to examine the association of routine blood test results with coronary heart disease (CHD) risk, to incorporate them into coronary prediction models and to compare the discrimination properties…

Medical Physics · Physics 2018-09-26 Ning Meng , Peng Zhang , Junfeng Li , Jun He , Jin Zhu

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

In observational studies, covariates with substantial missing data are often omitted, despite their strong predictive capabilities. These excluded covariates are generally believed not to simultaneously affect both treatment and outcome,…

Methodology · Statistics 2024-02-23 Shanshan Luo , Mengchen Shi , Wei Li , Xueli Wang , Zhi Geng

In observational studies, adjusting for confounders is required if a treatment comparison is planned. A crude comparison of the primary endpoint without covariate adjustment will suffer from biases, and the addition of regression models…

Methodology · Statistics 2026-02-11 Shiyu Shu , Toshimitsu Hamasaki , Scott Evans , Lauren Komarow , David van Duin , Guoqing Diao

Estimating causal effects from observational data is challenging due to selection bias, which leads to imbalanced covariate distributions across treatment groups. Propensity score-based weighting methods are widely used to address this…

Machine Learning · Computer Science 2025-08-08 Ahmad Saeed Khan , Erik Schaffernicht , Johannes Andreas Stork

Outcome Reporting Bias (ORB) poses significant threats to the validity of meta-analytic findings. It occurs when researchers selectively report outcomes based on the significance or direction of results, potentially leading to distorted…

Methodology · Statistics 2025-07-17 Alessandra Gaia Saracini , Leonhard Held

Collaboration between different data centers is often challenged by heterogeneity across sites. To account for the heterogeneity, the state-of-the-art method is to re-weight the covariate distributions in each site to match the distribution…

Machine Learning · Statistics 2024-04-25 Tianyu Guo , Sai Praneeth Karimireddy , Michael I. Jordan

Model selection/optimization in conformal inference is challenging, since it may break the exchangeability between labeled and unlabeled data. We study this problem in the context of conformal selection, which uses conformal p-values to…

Methodology · Statistics 2024-11-28 Tian Bai , Ying Jin

Combining observational and experimental data for causal inference can improve treatment effect estimation. However, many observational data sets cannot be released due to data privacy considerations, so one researcher may not have access…

Methodology · Statistics 2024-08-26 Charlotte Z. Mann , Adam C. Sales , Johann A. Gagnon-Bartsch

Optical coherence tomography (OCT) is a non-invasive imaging technique with extensive clinical applications in ophthalmology. OCT enables the visualization of the retinal layers, playing a vital role in the early detection and monitoring of…

We introduce a statistical procedure that integrates survival data from multiple biomedical studies, to improve the accuracy of predictions of survival or other events, based on individual clinical and genomic profiles, compared to models…

Applications · Statistics 2020-07-20 Steffen Ventz , Rahul Mazumder , Lorenzo Trippa

In this paper, we develop a multiply robust inference procedure of the average treatment effect (ATE) for data with high-dimensional covariates. We consider the case where it is difficult to correctly specify a single parametric model for…

Methodology · Statistics 2025-09-03 Xintao Xia , Yumou Qiu

Generation of realistic synthetic data has garnered considerable attention in recent years, particularly in the health research domain due to its utility in, for instance, sharing data while protecting patient privacy or determining optimal…

Other Statistics · Statistics 2025-01-30 Niki Z. Petrakos , Erica E. M. Moodie , Nicolas Savy

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

Dataset distillation extracts a small set of synthetic training samples from a large dataset with the goal of achieving competitive performance on test data when trained on this sample. In this work, we tackle dataset distillation at its…

Machine Learning · Computer Science 2023-11-14 Yunzhen Feng , Ramakrishna Vedantam , Julia Kempe

In clinical settings, we often face the challenge of building prediction models based on small observational data sets. For example, such a data set might be from a medical center in a multi-center study. Differences between centers might…

While randomised controlled trials (RCTs) are the gold standard for estimating causal treatment effects, their limited sample sizes and restrictive criteria make it difficult to extrapolate to a broader population. Observational data, while…

Methodology · Statistics 2025-09-09 Stephanie Riley , Ricardo Silva , Matthew Sperrin

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