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Related papers: Joint TITE-CRM for Dual Agent Dose Finding Studies

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In radiation therapy (RT) treatment planning, multi-criteria optimization (MCO) supports efficient plan selection but is usually solved for population-based dosimetric criteria and ignores patient-specific biological risk, potentially…

Medical Physics · Physics 2026-01-09 Mara Schubert , Katrin Teichert , Zhongxing Liao , Thomas Bortfeld , Ali Ajdari

The commonplace description of phase 1 clinical trials in oncology as "primarily concerned with safety" is belied by their near universal adoption of dose-escalation practices which are inherently unsafe. In contrast with dose titration,…

Methodology · Statistics 2020-04-28 David C. Norris

Participation incentives is a well-known issue inhibiting randomized controlled trials (RCTs) in medicine, as well as a potential cause of user dissatisfaction for RCTs in online platforms. We frame this issue as a non-standard…

Computer Science and Game Theory · Computer Science 2026-01-13 Yingkai Li , Aleksandrs Slivkins

Objective: Proton spot-scanning arc therapy (ARC) is an emerging modality that can improve the high-dose conformity to targets compared with standard intensity-modulated proton therapy (IMPT). However, the efficient treatment delivery of…

Accurate models of clinical actions and their impacts on disease progression are critical for estimating personalized optimal dynamic treatment regimes (DTRs) in medical/health research, especially in managing chronic conditions.…

Methodology · Statistics 2021-02-19 William Hua , Hongyuan Mei , Sarah Zohar , Magali Giral , Yanxun Xu

Heterogeneous treatment effects (HTEs) are commonly identified during randomized controlled trials (RCTs). Identifying subgroups of patients with similar treatment effects is of high interest in clinical research to advance precision…

Machine Learning · Computer Science 2022-12-06 Peniel N. Argaw , Elizabeth Healey , Isaac S. Kohane

The vision of personalized medicine is to identify interventions that maintain or restore a person's health based on their individual biology. Medical digital twins, computational models that integrate a wide range of health-related data…

Quantitative Methods · Quantitative Biology 2025-10-21 Luis L. Fonseca , Lucas Böttcher , Borna Mehrad , Reinhard C. Laubenbacher

One primary goal of precision medicine is to estimate the individualized treatment rules (ITRs) that optimize patients' health outcomes based on individual characteristics. Health studies with multiple treatments are commonly seen in…

Methodology · Statistics 2025-05-07 Xuqiao Li , Qiuyan Zhou , Ying Wu , Ying Yan

The keyboard design is a novel phase I dose-finding method that is simple and has good operating characteristics. This paper studies theoretical properties of the keyboard design, including the optimality of its decision rules, coherence in…

Methodology · Statistics 2017-12-20 Haitao Pan , Ruitao Lin , Ying Yuan

When a novel treatment has successfully passed phase I, different options to design subsequent phase II trials are available. One approach is a single-arm trial, comparing the response rate in the intervention group against a fixed…

Methodology · Statistics 2020-08-03 Johannes Krisam , Dorothea Weber , Richard F. Schlenk , Meinhard Kieser

Randomized controlled trials (RCTs) are widely regarded as the gold standard for causal inference in biomedical research. For instance, when estimating the average treatment effect on the treated (ATT), a doubly robust estimation procedure…

Methodology · Statistics 2025-09-26 Chi-Shian Dai , Chao Ying , Yang Ning , Jiwei Zhao

Personalizing combination therapies in oncology requires navigating an immense space of possible drug and dose combinations, a task that remains largely infeasible through exhaustive experimentation. Recent developments in patient-derived…

Machine Learning · Computer Science 2025-09-10 Antoine de Mathelin , Christopher Tosh , Wesley Tansey

Type 2 diabetes mellitus (T2DM) is a chronic disease that often results in multiple complications. Risk prediction and profiling of T2DM complications is critical for healthcare professionals to design personalized treatment plans for…

Machine Learning · Computer Science 2019-04-03 Bin Liu , Ying Li , Soumya Ghosh , Zhaonan Sun , Kenney Ng , Jianying Hu

A treatment regime is a deterministic function that dictates personalized treatment based on patients' individual prognostic information. There is a fast-growing interest in finding optimal treatment regimes to maximize expected long-term…

Statistics Theory · Mathematics 2016-11-25 Runchao Jiang , Wenbin Lu , Rui Song , Marie Davidian

This paper focuses on developing Pareto-optimal estimation and policy learning to identify the most effective treatment that maximizes the total reward from both short-term and long-term effects, which might conflict with each other. For…

Machine Learning · Computer Science 2024-03-13 Yingrong Wang , Anpeng Wu , Haoxuan Li , Weiming Liu , Qiaowei Miao , Ruoxuan Xiong , Fei Wu , Kun Kuang

During drug development, evidence can emerge to suggest a treatment is more effective in a specific patient subgroup. Whilst early trials may be conducted in biomarker-mixed populations, later trials are more likely to enrol…

Methodology · Statistics 2023-06-07 Lorna Wheaton , Dan Jackson , Sylwia Bujkiewicz

In a Phase II dose-finding study with a placebo control, a new drug with several dose levels is compared with a placebo to test for the effectiveness of the new drug. The main focus of such studies often lies in the characterization of the…

Methodology · Statistics 2020-07-14 Saswati Saha , Werner Brannath

Adverse drug reactions (ADRs) induced from high-order drug-drug interactions (DDIs) due to polypharmacy represent a significant public health problem. In this paper, we formally formulate the to-avoid and safe (with respect to ADRs) drug…

Information Retrieval · Computer Science 2018-03-09 Wen-Hao Chiang , Li Shen , Lang Li , Xia Ning

To promote precision medicine, individualized treatment regimes (ITRs) are crucial for optimizing the expected clinical outcome based on patient-specific characteristics. However, existing ITR research has primarily focused on scenarios…

Methodology · Statistics 2024-02-20 Chang Wang , Lu Wang

Intensity Modulated Radiation Therapy is an effective cancer treatment. Models based on the Generalized Equivalent Uniform Dose (gEUD) provide radiation plans with excellent planning target volume coverage and low radiation for organs at…