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Precision medicine is an emerging scientific topic for disease treatment and prevention that takes into account individual patient characteristics. It is an important direction for clinical research, and many statistical methods have been…

Methodology · Statistics 2017-02-17 Jingxiang Chen , Haoda Fu , Xuanyao He , Michael R. Kosorok , Yufeng Liu

Identifying patients who benefit from a treatment is a key aspect of personalized medicine, which allows the development of individualized treatment rules (ITRs). Many machine learning methods have been proposed to create such rules.…

Although there is now a large literature on policy evaluation and learning, much of the prior work assumes that the treatment assignment of one unit does not affect the outcome of another unit. Unfortunately, ignoring interference can lead…

Methodology · Statistics 2025-04-02 Yi Zhang , Kosuke Imai

An optimal individualized treatment rule (ITR) is a function that takes a patient's characteristics, such as demographics, biomarkers, and treatment history, and outputs a treatment that is expected to give the best outcome for that…

Methodology · Statistics 2026-02-05 Augustine Wigle , Erica E. M. Moodie

A treatment regime formalizes personalized medicine as a function from individual patient characteristics to a recommended treatment. A high-quality treatment regime can improve patient outcomes while reducing cost, resource consumption,…

Methodology · Statistics 2015-04-30 Yichi Zhang , Eric B. Laber , Anastasios Tsiatis , Marie Davidian

We develop a mathematical framework to define an optimal individualized treatment rule (ITR) within the context of prioritized outcomes in a randomized controlled trial. Our optimality criterion is based on the framework of generalized…

Methodology · Statistics 2025-06-17 François Petit , Gérard Biau , Raphaël Porcher

To maximize clinical benefit, clinicians routinely tailor treatment to the individual characteristics of each patient, where individualized treatment rules are needed and are of significant research interest to statisticians. In the…

Methodology · Statistics 2021-11-23 Trinetri Ghosh , Yanyuan Ma , Rui Song , Pingshou Zhong

Individualized treatment rules aim to identify if, when, which, and to whom treatment should be applied. A globally aging population, rising healthcare costs, and increased access to patient-level data have created an urgent need for…

Methodology · Statistics 2019-01-04 Ying-Qi Zhao , Eric B. Laber , Yang Ning , Sumona Saha , Bruce Sands

With the emergence of precision medicine, estimating optimal individualized decision rules (IDRs) has attracted tremendous attention in many scientific areas. Most existing literature has focused on finding optimal IDRs that can maximize…

Methodology · Statistics 2022-06-28 Zhengling Qi , Jong-Shi Pang , Yufeng Liu

Dynamic Treatment Regimes (DTRs) provide a systematic framework for optimizing sequential decision-making in chronic disease management, where therapies must adapt to patients' evolving clinical profiles. Inverse probability weighting (IPW)…

Methodology · Statistics 2026-03-26 Chloe Si , David A. Stephens , Erica E. M. Moodie

In many important applications of precision medicine, the outcome of interest is time to an event (e.g., death, relapse of disease) and the primary goal is to identify the optimal individualized decision rule (IDR) to prolong survival time.…

Methodology · Statistics 2022-04-11 Yu Zhou , Lan Wang , Rui Song , Tuoyi Zhao

Recent statistical and reinforcement learning methods have significantly advanced patient care strategies. However, these approaches face substantial challenges in high-stakes contexts, including missing data, inherent stochasticity, and…

Machine Learning · Computer Science 2024-04-02 Harsh Parikh , Quinn Lanners , Zade Akras , Sahar F. Zafar , M. Brandon Westover , Cynthia Rudin , Alexander Volfovsky

Recurrent events, characterized by the repeated occurrence of the same event in an individual, are a common type of data in medical research. Motivated by cancer recurrences, we aim to estimate the optimal individualized treatment regime…

Methodology · Statistics 2025-02-18 Zi-Shu Zhan , Jin-Lun Zhang , Chen Shi , Xiao-Han Xu , Chun-Quan Ou

Estimating dynamic treatment regimes (DTRs) from retrospective observational data is challenging as some degree of unmeasured confounding is often expected. In this work, we develop a framework of estimating properly defined "optimal" DTRs…

Methodology · Statistics 2021-04-19 Shuxiao Chen , Bo Zhang

Most existing interpretable methods explain a black-box model in a post-hoc manner, which uses simpler models or data analysis techniques to interpret the predictions after the model is learned. However, they (a) may derive contradictory…

Machine Learning · Computer Science 2020-01-22 Mengzhuo Guo , Qingpeng Zhang , Xiuwu Liao , Daniel Dajun Zeng

The goal of precision medicine is to provide individualized treatment at each stage of chronic diseases, a concept formalized by Dynamic Treatment Regimes (DTR). These regimes adapt treatment strategies based on decision rules learned from…

Methodology · Statistics 2025-06-09 Sophia Yazzourh , Nicolas Savy , Philippe Saint-Pierre , Michael R. Kosorok

A novel functional additive model is proposed which is uniquely modified and constrained to model nonlinear interactions between a treatment indicator and a potentially large number of functional and/or scalar pretreatment covariates. The…

Methodology · Statistics 2021-01-26 Hyung Park , Eva Petkova , Thaddeus Tarpey , R. Todd Ogden

We propose a nonparametric additive model for estimating interpretable value functions in reinforcement learning, with an application in optimizing postoperative recovery through personalized, adaptive recommendations. While reinforcement…

Machine Learning · Statistics 2025-06-02 Patrick Emedom-Nnamdi , Timothy R. Smith , Jukka-Pekka Onnela , Junwei Lu

Small additive ensembles of symbolic rules offer interpretable prediction models. Traditionally, these ensembles use rule conditions based on conjunctions of simple threshold propositions $x \geq t$ on a single input variable $x$ and…

Machine Learning · Computer Science 2025-06-27 Shahrzad Behzadimanesh , Pierre Le Bodic , Geoffrey I. Webb , Mario Boley

Adaptive therapy is a dynamic cancer treatment protocol that updates (or "adapts") treatment decisions in anticipation of evolving tumor dynamics. This broad term encompasses many possible dynamic treatment protocols of patient-specific…