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Related papers: DROID: Dose-ranging Approach to Optimizing Dose in…

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Dose-escalation trials in oncology drug development still today typically aim to identify 1-size-fits-all dose recommendations, as arbitrary quantiles of the toxicity thresholds evident in patient samples. In the late 1990s efforts to…

Category Theory · Mathematics 2025-07-03 David C. Norris

Broadening eligibility criteria in cancer trials has been advocated to represent the true patient population more accurately. While the advantages are clear in terms of generalizability and recruitment, novel dose-finding designs are needed…

Applications · Statistics 2023-01-12 Rebecca B. Silva , Bin Cheng , Richard D. Carvajal , Shing M. Lee

The primary objective of Phase I oncology trials is to assess the safety and tolerability of novel therapeutics. Conventional dose escalation methods identify the maximum tolerated dose (MTD) based on dose-limiting toxicity (DLT). However,…

Methodology · Statistics 2024-09-02 Yunlong Yang , Ying Yuan

While cancer has traditionally been considered a genetic disease, mounting evidence indicates an important role for non-genetic (epigenetic) mechanisms. Common anti-cancer drugs have recently been observed to induce the adoption of…

Populations and Evolution · Quantitative Biology 2025-07-04 Einar Bjarki Gunnarsson , Benedikt Vilji Magnússon , Jasmine Foo

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

An objective of phase I dose-finding trials is to find the maximum tolerated dose; the dose with a particular risk of toxicity. Frequently, this risk is assessed across the first cycle of therapy. However, in oncology, a course of treatment…

Applications · Statistics 2021-05-03 Helen Barnett , Oliver Boix , Dimintris Kontos , Thomas Jaki

Recent clinical trials have shown that the adaptive drug therapy can be more efficient than a standard MTD-based policy in treatment of cancer patients. The adaptive therapy paradigm is not based on a preset schedule; instead, the doses are…

Quantitative Methods · Quantitative Biology 2020-08-06 Mark Gluzman , Jacob G. Scott , Alexander Vladimirsky

In response to the U.S.\ Food and Drug Administration's (FDA) Project Optimus, a paradigm shift is underway in the design of early-phase oncology trials. To accelerate drug development, seamless Phase I/II designs have gained increasing…

Methodology · Statistics 2025-11-04 Kana Makino , Natsumi Makigusa , Masahiro Kojima

The primary objective of phase I cancer clinical trials is to evaluate the safety of a new experimental treatment and to find the maximum tolerated dose (MTD). We show that the MTD estimation problem can be regarded as a level set…

Machine Learning · Statistics 2025-04-15 Keiichiro Seno , Kota Matsui , Shogo Iwazaki , Yu Inatsu , Shion Takeno , Shigeyuki Matsui

We consider a real-world chemotherapy scheduling template design problem, where we cluster patient types into groups and find a representative time-slot duration for each group to accommodate all patient types assigned to that group, aiming…

Optimization and Control · Mathematics 2025-10-14 Qing Zhu , Xian Yu , Yu-Li Huang

Cancer is one of the most common diseases worldwide, posing a serious threat to human health and leading to the deaths of a large number of people. It was observed during the drug administration in chemotherapy that immune cells, cancer…

Quantitative Methods · Quantitative Biology 2020-08-20 SeyedMehdi Abtahi , Mojtaba Sharifi

Dynamic treatment regime (DTR) plays a critical role in precision medicine when assigning patient-specific treatments at multiple stages and optimizing a long term clinical outcome. However, most of existing work about DTRs have been…

Methodology · Statistics 2023-02-20 Chang Wang , Lu Wang

Chemotherapy is one of the primary modalities of cancer treatment. Chemotherapy drug administration is a complex problem that often requires expensive clinical trials to evaluate potential regimens. One way to alleviate this burden and…

Optimization and Control · Mathematics 2021-11-04 Temitayo Ajayi , Seyedmohammadhossein Hosseinian , Andrew J. Schaefer , Clifton D. Fuller

We conduct a theoretical study of various solution methods for the adaptive fractionation problem. The two messages of this paper are: (i) dynamic programming (DP) is a useful framework for adaptive radiation therapy, particularly adaptive…

Medical Physics · Physics 2012-02-16 Jagdish Ramakrishnan , David Craft , Thomas Bortfeld , John N. Tsitsiklis

The use of drug combinations in clinical trials is increasingly common during the last years since a more favorable therapeutic response may be obtained by combining drugs. In phase I clinical trials, most of the existing methodology…

Methodology · Statistics 2020-02-17 José L. Jiménez , Sungjin Kim , Mourad Tighiouart

Dose optimization in oncology clinical trials has shifted from seeking the maximum tolerated dose to identifying the Optimal Biological Dose (OBD) that balances therapeutic benefits and risks across multiple clinical attributes. Existing…

Methodology · Statistics 2025-05-07 Fanni Zhang , Kristine Broglio , Michael Sweeting , Gina D'Angelo

Drug discovery is a lengthy and resource-intensive process composed of multiple stages. Among these stages, lead optimization plays a critical role in transforming early hit compounds into viable drug candidates. This stage requires…

Machine Learning · Computer Science 2026-05-29 Lingxiao Li , Haobo Zhang , Ruohao Fan , Bin Chen , Jiayu Zhou

Phase I-II cancer clinical trial designs are intended to accelerate drug development. In cases where efficacy cannot be ascertained in a short period of time, it is common to divide the study in two stages: i) a first stage in which dose is…

Methodology · Statistics 2022-12-13 José L. Jiménez , Mourad Tighiouart

Reinforcement learning (RL) has demonstrated great success in the past several years. However, most of the scenarios focus on simulated environments. One of the main challenges of transferring the policy learned in a simulated environment…

Robotics · Computer Science 2021-02-24 Ya-Yen Tsai , Hui Xu , Zihan Ding , Chong Zhang , Edward Johns , Bidan Huang

We consider a formal statistical design that allows simultaneous enrollment of a main cohort and a backfill cohort of patients in a dose-finding trial. The goal is to accumulate more information at various doses to facilitate dose…

Applications · Statistics 2024-04-03 Jiaxin Liu , Shijie Yuan , B. Nebiyou Bekele , Yuan Ji