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Related papers: Personalized Dynamic Treatment Regimes in Continuo…

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In developing products for rare diseases, statistical challenges arise due to the limited number of patients available for participation in drug trials and other clinical research. Bayesian adaptive clinical trial designs offer the…

Applications · Statistics 2019-09-19 Xiao Wu , Yi Xu , Bradley P. Carlin

Dynamic treatment regimens (DTRs), also known as treatment algorithms or adaptive interventions, play an increasingly important role in many health domains. DTRs are motivated to address the unique and changing needs of individuals by…

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

Treatment effect estimation in continuous time is crucial for personalized medicine. However, existing methods for this task are limited to point estimates of the potential outcomes, whereas uncertainty estimates have been ignored. Needless…

Machine Learning · Computer Science 2024-04-04 Konstantin Hess , Valentyn Melnychuk , Dennis Frauen , Stefan Feuerriegel

Individualized treatment rules (ITRs) are deterministic decision rules that recommend treatments to individuals based on their characteristics. Though ubiquitous in medicine, ITRs are hardly ever evaluated in randomized controlled trials.…

Methodology · Statistics 2023-08-22 François Grolleau , Francois Petit , Raphaël Porcher

The field of precision medicine aims to tailor treatment based on patient-specific factors in a reproducible way. To this end, estimating an optimal individualized treatment regime (ITR) that recommends treatment decisions based on patient…

In clinical practice, physicians make a series of treatment decisions over the course of a patient's disease based on his/her baseline and evolving characteristics. A dynamic treatment regime is a set of sequential decision rules that…

Methodology · Statistics 2015-02-04 Phillip J. Schulte , Anastasios A. Tsiatis , Eric B. Laber , Marie Davidian

This paper studies the problem of performing a sequence of optimal interventions in a causal dynamical system where both the target variable of interest and the inputs evolve over time. This problem arises in a variety of domains e.g.…

Machine Learning · Statistics 2021-10-27 Virginia Aglietti , Neil Dhir , Javier González , Theodoros Damoulas

It is crucial to design Phase II cancer clinical trials that balance the efficiency of treatment selection with clinical practicality. Sargent and Goldberg proposed a frequentist design that allow decision-making even when the primary…

Methodology · Statistics 2025-05-15 Moka Komaki , Satoru Shinoda , Haiyan Zheng , Kouji Yamamoto

Stabilized dynamic treatment regimes are sequential decision rules for individual patients that not only adaptive throughout the disease progression but also remain consistent over time in format. The estimation of stabilized dynamic…

Methodology · Statistics 2019-03-05 Ying-Qi Zhao , Ruoqing Zhu , Guanhua Chen , Yingye Zheng

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

In randomized trials involving multiple treatments, bivariate survival outcomes present significant analytical challenges for making decisions. This paper addresses the problem of deriving optimal individualized treatment rules to maximize…

Machine Learning · Statistics 2026-05-29 Kun Ren , Yifan Cui , Wen Su

Clinical decision making regarding treatments based on personal characteristics leads to effective health improvements. Machine learning (ML) has been the primary concern of diagnosis support according to comprehensive patient information.…

Machine Learning · Computer Science 2021-06-09 Kazuki Nakamura , Ryosuke Kojima , Eiichiro Uchino , Koichi Murashita , Ken Itoh , Shigeyuki Nakaji , Yasushi Okuno

Optimal treatment regimes (OTR) are individualised treatment assignment strategies that identify a medical treatment as optimal given all background information available on the individual. We discuss Bayes optimal treatment regimes…

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

Methodology · Statistics 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

Background. Designing trials to reduce treatment duration is important in several therapeutic areas, including TB and antibiotics. We recently proposed a new randomised trial design to overcome some of the limitations of standard two-arm…

Phase I dose-finding trials in oncology seek to find the maximum tolerated dose (MTD) of a drug under a specific schedule. Evaluating drug-schedules aims at improving treatment safety while maintaining efficacy. However, while we can…

Randomized clinical trials are considered the gold standard for estimating causal effects. Nevertheless, in studies that are aimed at examining adverse effects of interventions, such trials are often impractical because of ethical and…

Methodology · Statistics 2020-01-20 Anthony D. Scotina , Andrew R. Zullo , Robert J. Smith , Roee Gutman

Existing strategies for determining the optimal treatment or monitoring strategy typically assume unlimited access to resources. However, when a health system has resource constraints, such as limited funds, access to medication, or…

Applications · Statistics 2019-03-18 Ellen C Caniglia , Eleanor J Murray , Miguel A Hernan , Zach Shahn

The goal of personalized decision making is to map a unit's characteristics to an action tailored to maximize the expected outcome for that unit. Obtaining high-quality mappings of this type is the goal of the dynamic regime literature. In…

Machine Learning · Computer Science 2018-10-01 Razieh Nabi , Phyllis Kanki , Ilya Shpitser