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We consider optimal regimes for algorithm-assisted human decision-making. Such regimes are decision functions of measured pre-treatment variables and, by leveraging natural treatment values, enjoy a "superoptimality" property whereby they…

Methodology · Statistics 2024-02-23 Mats J. Stensrud , Julien Laurendeau , Aaron L. Sarvet

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

The complexity of human cancer often results in significant heterogeneity in response to treatment. Precision medicine offers potential to improve patient outcomes by leveraging this heterogeneity. Individualized treatment rules (ITRs)…

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

In recent years new cancer treatments improved survival in multiple histologies. Some of these therapeutics, and in particular treatment combinations, are often associated with severe treatment-related adverse events (AEs). It is therefore…

Applications · Statistics 2022-03-10 Steffen Ventz , Lorenzo Trippa

The modern workflow for radiation therapy treatment planning involves mathematical optimization to determine optimal treatment machine parameters for each patient case. The optimization problems can be computationally expensive, requiring…

Mathematical Software · Computer Science 2023-05-30 Felix Liu , Måns I. Andersson , Albin Fredriksson , Stefano Markidis

With new advancements in technology, it is now possible to collect data for a variety of different metrics describing tumor growth, including tumor volume, composition, and vascularity, among others. For any proposed model of tumor growth…

Quantitative Methods · Quantitative Biology 2020-09-08 Heyrim Cho , Allison L. Lewis , Kathleen M. Storey

Clinical trials often collect data on multiple outcomes, such as overall survival (OS), progression-free survival (PFS), and response to treatment (RT). In most cases, however, study designs only use primary outcome data for interim and…

Applications · Statistics 2026-04-28 Massimiliano Russo , Steffen Ventz , Lorenzo Trippa

In oncology, phase II or multiple expansion cohort trials are crucial for clinical development plans. This is because they aid in identifying potent agents with sufficient activity to continue development and confirm the proof of concept.…

Methodology · Statistics 2024-05-24 Takuya Yoshimoto , Satoru Shinoda , Kouji Yamamoto , Kouji Tahata

A dynamic treatment regime is a sequence of medical decisions that adapts to the evolving clinical status of a patient over time. To facilitate personalized care, it is crucial to assess the probability of each available treatment option…

Methodology · Statistics 2024-11-05 Jiefeng Bi , Matteo Borrotti , Bernardo Nipoti

The move towards personalized treatment and digital twins for cancer therapy requires a complete understanding of the mathematical models upon which these optimized simulation-based strategies are formulated. This study investigates the…

Quantitative Methods · Quantitative Biology 2025-11-21 Changin Oh , Kathleen P. Wilkie

Causal effect estimation for dynamic treatment regimes (DTRs) contributes to sequential decision making. However, censoring and time-dependent confounding under DTRs are challenging as the amount of observational data declines over time due…

Machine Learning · Statistics 2021-09-27 Adi Lin , Jie Lu , Junyu Xuan , Fujin Zhu , Guangquan Zhang

In precision medicine, one of the most important problems is estimating the optimal individualized treatment rules (ITR), which typically involves recommending treatment decisions based on fully observed individual characteristics of…

Methodology · Statistics 2025-10-15 Yue Zhang , Shanshan Luo , Zhi Geng , Yangbo He

Opportunistic detection rules (ODRs) are variants of fixed-sample-size detection rules in which the statistician is allowed to make an early decision on the alternative hypothesis opportunistically based on the sequentially observed…

Information Theory · Computer Science 2016-02-15 Wenyi Zhang , George V. Moustakides , H. Vincent Poor

Traditionally, the major objective in phase I trials is to identify a working-dose for subsequent studies, whereas the major endpoint in phase II and III trials is treatment efficacy. The dose sought is typically referred to as the maximum…

Methodology · Statistics 2016-08-14 Mourad Tighiouart , André Rogatko

A dynamic treatment regime (DTR) is an approach to delivering precision medicine that uses patient characteristics to guide treatment decisions for optimal health outcomes. Numerous methods have been proposed for DTR estimation, including…

Methodology · Statistics 2025-02-03 Adel Ahmadi Nadi , Michael Wallace

An individualized treatment rule (ITR) is a decision rule that recommends treatments for patients based on their individual feature variables. In many practices, the ideal ITR for the primary outcome is also expected to cause minimal harm…

Methodology · Statistics 2024-05-24 Daiqi Gao , Yuanjia Wang , Donglin Zeng

The treatment allocation mechanism in a randomized clinical trial can be optimized by maximizing the nonparametric efficiency bound for a specific measure of treatment effect. Optimal treatment allocations which may or may not depend on…

Methodology · Statistics 2025-05-23 Wei Zhang , Zhiwei Zhang , Aiyi Liu

Traditionally, optimization of radiation therapy (RT) treatment plans has been done before the initiation of RT course, using population-wide estimates for patients' response to therapy. However, recent technological advancements have…

Medical Physics · Physics 2021-02-15 Stefan C. M. ten Eikelder , Ali Ajdari , Thomas Bortfeld , Dick den Hertog

Beam angle optimization (BAO) largely determines the performance of fixed-field intensity modulated radiation therapy (IMRT), and it is usually considered as non-convex optimization and an NP hard problem. In this work, BAO is reformulated…

Medical Physics · Physics 2021-05-12 Junbo Peng , Ailin Wu , Lei Zhu