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This paper addresses patient heterogeneity associated with prediction problems in biomedical applications. We propose a systematic hypothesis testing approach to determine the existence of patient subgroup structure and the number of…

Methodology · Statistics 2021-01-08 Xu Gao , Weining Shen , Jing Ning , Ziding Feng , Jianhua Hu

We propose a framework for building patient-specific treatment recommendation models, building on the large recent literature on learning patient-level causal models and inspired by the target trial paradigm of Hernan and Robins. We focus…

Machine Learning · Statistics 2025-07-17 Rom Gutman , Shimon Sheiba , Omer Noy Klein , Naama Dekel Bird , Amit Gruber , Doron Aronson , Oren Caspi , Uri Shalit

Introduction: There have been many warnings that inappropriate dichotomisation of results into positive or negative, high, or normal etc., during medical research could be very damaging. The aim of this paper is to argue that this is the…

Other Quantitative Biology · Quantitative Biology 2023-10-06 Huw Llewelyn

Modelling disease progression of iron deficiency anaemia (IDA) following oral iron supplement prescriptions is a prerequisite for evaluating the cost-effectiveness of oral iron supplements. Electronic health records (EHRs) from the Clinical…

Applications · Statistics 2021-07-30 Li Su , Yafeng Cheng , Dora I. A. Pereira , Jonathan J. Powell

This paper studies the identification of causal effects of a continuous treatment using a new difference-in-difference strategy. Our approach allows for endogeneity of the treatment, and employs repeated cross-sections. It requires an…

Econometrics · Economics 2023-04-18 Xavier D'Haultfoeuille , Stefan Hoderlein , Yuya Sasaki

Dynamic treatment regimes operationalize the clinical decision process as a sequence of functions, one for each clinical decision, where each function takes as input up-to-date patient information and gives as output a single recommended…

Methodology · Statistics 2012-08-08 Eric B. Laber , Daniel J. Lizotte , Bradley Ferguson

In the field of cardio-thoracic surgery, valve function is monitored over time after surgery. The motivation for our research comes from a study which includes patients who received a human tissue valve in the aortic position. These…

Estimating treatment effects conditional on observed covariates can improve the ability to tailor treatments to particular individuals. Doing so effectively requires dealing with potential confounding, and also enough data to adequately…

The health effects of environmental exposures have been studied for decades, typically using standard regression models to assess exposure-outcome associations found in observational non-experimental data. We propose and illustrate a…

Applications · Statistics 2017-09-20 Marie-Abele C. Bind , Donald B. Rubin

Evaluating the influence of continuous covariates, like exposure time or dose, on a response variable is a pivotal objective in the assessment of a compound's effect, particularly when determining toxicity in pre-clinical research or…

Methodology · Statistics 2026-04-16 Lucia Ameis , Niklas Hagemann , Kathrin Möllenhoff

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 clinical studies, the risk of the primary (terminal) event may be modified by intermediate events, resulting in semicompeting risks. To study the treatment effect on the terminal event mediated by the intermediate event, researchers wish…

Methodology · Statistics 2026-05-26 Yuhao Deng , Rui Wang , Tao Zhang , Xiang Zhan

Many policies involve dynamics in their treatment assignments, where individuals receive sequential interventions over multiple stages. We study estimation of an optimal dynamic treatment regime that guides the optimal treatment assignment…

Econometrics · Economics 2024-09-04 Shosei Sakaguchi

Pain management in intensive care usually involves complex trade-offs, since both inadequate and excessive treatment can compromise patient safety. Prior work on reinforcement learning for sedation and analgesia has explored how to optimize…

Machine Learning · Computer Science 2026-05-19 Joel Romero-Hernandez , Oscar Camara

Precision medicine involves developing individualized treatment regimes (ITRs) which allow for treatment decisions to be tailored to patient characteristics. Naturally, the identification of the optimal regime, that is, the rule which…

Methodology · Statistics 2025-09-30 Misha Dolmatov , Erica E. M. Moodie , David A. Stephens , Dipankar Bandyopadhyay

Long-term adherence to medication is a critical factor in preventing chronic diseases, such as cardiovascular disease. To address poor adherence, physicians may recommend adherence-improving interventions; however, such interventions are…

Optimization and Control · Mathematics 2026-02-24 Daniel Otero-Leon , Mariel Lavieri , Brian Denton , Jeremy Sussman , Rodney Hayward

Modeling dose-response relationships of drugs is essential to understanding their effect on patient outcomes under realistic circumstances. While intention-to-treat analyses of clinical trials provide the effect of assignment to a…

Applications · Statistics 2018-02-15 Jacob Spertus , Marcela Horvitz-Lennon , Sharon-Lise Normand

Background: Clinical trials are designed to prove the efficacy of an intervention by means of model-based approaches involving parametric hypothesis testing. Issues arise when no effect is observed in the study population. Indeed, an effect…

Chronic kidney disease (CKD) represents a slowly progressive disorder that can eventually require renal replacement therapy (RRT) including dialysis or renal transplantation. Early identification of patients who will require RRT (as much as…

Machine Learning · Computer Science 2022-09-07 Daniel Lopez-Martinez , Christina Chen , Ming-Jun Chen

An objective of phase I dose-finding trials is to find the maximum tolerated dose; the dose with a particular risk of toxicity. Frequently, this risk is assessed across the first cycle of therapy. However, in oncology, a course of treatment…

Applications · Statistics 2021-05-03 Helen Barnett , Oliver Boix , Dimintris Kontos , Thomas Jaki