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Risk-adjusted quality measures are used to evaluate healthcare providers while controlling for factors beyond their control. Existing healthcare provider profiling approaches typically assume that the risk adjustment is perfect and the…

Applications · Statistics 2022-07-26 Nicholas Hartman , Joseph M. Messana , Jian Kang , Abhijit S. Naik , Tempie H. Shearon , Kevin He

The application of existing methods for constructing optimal dynamic treatment regimes is limited to cases where investigators are interested in optimizing a utility function over a fixed period of time (finite horizon). In this manuscript,…

Methodology · Statistics 2015-10-22 Ashkan Ertefaie

In biomedical studies, estimating drug effects on chronic diseases requires a long follow-up period, which is difficult to meet in randomized clinical trials (RCTs). The use of a short-term surrogate to replace the long-term outcome for…

Methodology · Statistics 2023-06-27 Wenjie Hu , Xiaohua Zhou , Peng Wu

Recent advances in dynamic treatment regimes (DTRs) facilitate the search for optimal treatments, which are tailored to individuals' specific needs and able to maximize their expected clinical benefits. However, existing algorithms relying…

Machine Learning · Statistics 2024-10-18 Hanwen Ye , Wenzhuo Zhou , Ruoqing Zhu , Annie Qu

Long-term treatment effect estimation is a significant but challenging problem in many applications. Existing methods rely on ideal assumptions, such as no unobserved confounders or binary treatment, to estimate long-term average treatment…

Machine Learning · Computer Science 2025-10-23 Zeqin Yang , Weilin Chen , Ruichu Cai , Yuguang Yan , Zhifeng Hao , Zhipeng Yu , Zhichao Zou , Jixing Xu , Zhen Peng , Jiecheng Guo

Kidney transplantation is the preferred treatment for end-stage renal disease, yet the scarcity of donors and inefficiencies in allocation systems create major bottlenecks, resulting in prolonged wait times and alarming mortality rates.…

Computer Science and Game Theory · Computer Science 2024-10-15 Sean Berry , Berk Gorgulu , Sait Tunc , Mucahit Cevik , Matthew J Ellis

The estimand framework provides guidance on handling intercurrent events, such as treatment discontinuation, in the analysis of clinical trial responses. Under ICH E9(R1), the treatment policy (TP) strategy incorporates post-discontinuation…

Methodology · Statistics 2026-04-07 Myeongjong Kang , Sangyoon Yi

We propose a new method in indefinite-horizon settings for estimating optimal dynamic treatment regimes for time-to-event outcomes. This method allows patients to have different numbers of treatment stages and is constructed using…

Methodology · Statistics 2025-01-31 Jane She , Matthew Egberg , Michael R. Kosorok

Randomized experimentation (also known as A/B testing or bucket testing) is widely used in the internet industry to measure the metric impact obtained by different treatment variants. A/B tests identify the treatment variant showing the…

When devising a course of treatment for a patient, doctors often have little quantitative evidence on which to base their decisions, beyond their medical education and published clinical trials. Stanford Health Care alone has millions of…

Background: Hypertensive kidney disease (HKD) patients in intensive care units (ICUs) face high short-term mortality, but tailored risk prediction tools are lacking. Early identification of high-risk individuals is crucial for clinical…

Machine Learning · Computer Science 2025-07-28 Yong Si , Junyi Fan , Li Sun , Shuheng Chen , Minoo Ahmadi , Elham Pishgar , Kamiar Alaei , Greg Placencia , Maryam Pishgar

Quantile optimal treatment regimes (OTRs) aim to assign treatments that maximize a specified quantile of patients' outcomes. Compared to treatment regimes that target the mean outcomes, quantile OTRs offer fairer regimes when a lower…

Methodology · Statistics 2026-01-07 Junwen Xia , Jingxiao Zhang , Dehan Kong

Objective: To compare different risk-based methods for optimal prediction of treatment effects. Methods: We simulated RCT data using diverse assumptions for the average treatment effect, a baseline prognostic index of risk (PI), the shape…

In personalized medicine, the ability to predict and optimize treatment outcomes across various time frames is essential. Additionally, the ability to select cost-effective treatments within specific budget constraints is critical. Despite…

Machine Learning · Computer Science 2024-10-14 Thomas Schwarz , Cecilia Casolo , Niki Kilbertus

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

Image-based precision medicine aims to personalize treatment decisions based on an individual's unique imaging features so as to improve their clinical outcome. Machine learning frameworks that integrate uncertainty estimation as part of…

Machine Learning · Computer Science 2023-08-11 Joshua Durso-Finley , Jean-Pierre Falet , Raghav Mehta , Douglas L. Arnold , Nick Pawlowski , Tal Arbel

Optimal treatment rules are mappings from individual patient characteristics to tailored treatment assignments that maximize mean outcomes. In this work, we introduce a conditional potential benefit (CPB) metric that measures the expected…

Methodology · Statistics 2024-08-29 Alexander W. Levis , Eli Ben-Michael , Edward H. Kennedy

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

We propose a prognostic stratum matching framework that addresses the deficiencies of Randomized trial data subgroup analysis and transforms ObservAtional Data to be used as if they were randomized, thus paving the road for precision…

Applications · Statistics 2023-11-06 Dimitris Bertsimas , Angelos G. Koulouras , Georgios Antonios Margonis

The burden of diseases is rising worldwide, with unequal treatment efficacy for patient populations that are underrepresented in clinical trials. Healthcare, however, is driven by the average population effect of medical treatments and,…

Machine Learning · Computer Science 2024-02-08 Ghadeer O. Ghosheh , Moritz Gögl , Tingting Zhu