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Adaptive sample size re-estimation (SSR) is a well-established strategy for improving the efficiency and flexibility of clinical trials. Its central challenge is determining whether, and by how much, to increase the sample size at an…

Methodology · Statistics 2025-10-20 Rui Jin , Cai Wu , Qiqi Deng

Dynamic treatment regimes (DTRs) are used in medicine to tailor sequential treatment decisions to patients by considering patient heterogeneity. Common methods for learning optimal DTRs, however, have shortcomings: they are typically based…

Machine Learning · Statistics 2023-06-21 Theresa Blümlein , Joel Persson , Stefan Feuerriegel

The sequential treatment decisions made by physicians to treat chronic diseases are formalized in the statistical literature as dynamic treatment regimes. To date, methods for dynamic treatment regimes have been developed under the…

Methodology · Statistics 2022-02-22 Janie Coulombe , Erica E. M. Moodie , Susan M. Shortreed , Christel Renoux

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

Dynamic treatment regimes (DTR) are a statistical paradigm in precision medicine which aim to optimize patient outcomes by individualizing treatments. At its simplest, a DTR may require only a single decision to be made; this special case…

Applications · Statistics 2021-09-06 Larry Dong , Erica E. M. Moodie , Laura Villain , Rodolphe Thiébaut

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

Optimal dynamic treatment regimes (DTRs), as a key part of precision medicine, have progressively gained more attention recently. To inform clinical decision making, interpretable and parsimonious models for contrast functions are…

Methodology · Statistics 2025-12-08 Chunyu Wang , Brian Tom

Public policies and medical interventions often involve dynamic treatment assignments, in which individuals receive a sequence of interventions over multiple stages. We study the statistical learning of optimal dynamic treatment regimes…

Methodology · Statistics 2025-05-21 Shosei Sakaguchi

Adaptive interventions, aka dynamic treatment regimens, are sequences of pre-specified decision rules that guide the provision of treatment for an individual given information about their baseline and evolving needs, including in response…

Methodology · Statistics 2024-05-02 Wenchu Pan , Daniel Almirall , Amy M. Kilbourne , Andrew Quanbeck , Lu Wang

The study of precision medicine involves dynamic treatment regimes (DTRs), which are sequences of treatment decision rules recommended by taking patient-level information as input. The primary goal of the DTR study is to identify an optimal…

Methodology · Statistics 2024-12-11 Dan Liu , Wenqing He

In the management of most chronic conditions characterized by the lack of universally effective treatments, adaptive treatment strategies (ATSs) have been growing in popularity as they offer a more individualized approach, and sequential…

Methodology · Statistics 2021-08-03 Armando Turchetta , Erica E. M. Moodie , David A. Stephens , Sylvie D. Lambert

An optimal dynamic treatment regime (DTR) consists of a sequence of decision rules in maximizing long-term benefits, which is applicable for chronic diseases such as HIV infection or cancer. In this paper, we develop a novel angle-based…

Methodology · Statistics 2020-01-15 Fei Xue , Yanqing Zhang , Wenzhuo Zhou , Haoda Fu , Annie Qu

Evidence supporting the current World Health Organization recommendations of early antiretroviral therapy (ART) initiation for adolescents is inconclusive. We leverage a large observational data and compare, in terms of mortality and CD4…

Applications · Statistics 2019-04-04 Liangyuan Hu , Joseph W. Hogan

The focus of precision medicine is on decision support, often in the form of dynamic treatment regimes (DTRs), which are sequences of decision rules. At each decision point, the decision rules determine the next treatment according to the…

Methodology · Statistics 2023-12-22 Cong Jiang , Mary Thompson , Michael Wallace

An individualized treatment rule (ITR) is a decision rule that aims to improve individual patients health outcomes by recommending optimal treatments according to patients specific information. In observational studies, collected data may…

Methodology · Statistics 2023-10-03 Zeyu Bian , Erica EM Moodie , Susan M Shortreed , Sylvie D Lambert , Sahir Bhatnagar

Longitudinal cluster randomized trials (L-CRTs) are increasingly used to evaluate the cost-effectiveness of healthcare interventions across multiple assessment periods, yet design methods for powering these trials remain underdeveloped.…

Methodology · Statistics 2026-03-20 Hao Wang , Jingxia Liu , Drew B. Cameron , Jiaqi Tong , Donna Spiegelman , Daniella Meeker , Fan Li

Studies often report estimates of the average treatment effect. While the ATE summarizes the effect of a treatment on average, it does not provide any information about the effect of treatment within any individual. A treatment strategy…

Methodology · Statistics 2025-06-13 Nicholas T. Williams , Katherine L. Hoffman Iván Díaz , Kara E. Rudolph

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

Methodology · Statistics 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

Technological advancements have made it possible to deliver mobile health interventions to individuals. A novel framework that has emerged from such advancements is the just-in-time adaptive intervention (JITAI), which aims to suggest the…

Methodology · Statistics 2023-07-10 Jing Xu , Xiaoxi Yan , Caroline Figueroa , Joseph Jay Williams , Bibhas Chakraborty

Personalized medicine seeks to identify the causal effect of treatment for a particular patient as opposed to a clinical population at large. Most investigators estimate such personalized treatment effects by regressing the outcome of a…

Machine Learning · Statistics 2021-09-02 Eric V. Strobl , Shyam Visweswaran