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This paper proposes a novel criterion for the allocation of patients in Phase~I dose-escalation clinical trials aiming to find the maximum tolerated dose (MTD). Conventionally, using a model-based approach the next patient is allocated to…

统计方法学 · 统计学 2018-07-17 Pavel Mozgunov , Thomas Jaki

In the era of targeted therapy, there has been increasing concern about the development of oncology drugs based on the "more is better" paradigm, developed decades ago for chemotherapy. Recently, the US Food and Drug Administration (FDA)…

统计方法学 · 统计学 2022-09-07 Beibei Guo , Ying Yuan

Phase I early-phase clinical studies aim at investigating the safety and the underlying dose-toxicity relationship of a drug or combination. While little may still be known about the compound's properties, it is crucial to consider…

统计方法学 · 统计学 2022-09-13 Christian Röver , Moreno Ursino , Tim Friede , Sarah Zohar

An accurately identified maximum tolerated dose (MTD) serves as the cornerstone of successful subsequent phases in oncology drug development. Bayesian logistic regression model (BLRM) is a popular and versatile model-based dose-finding…

统计方法学 · 统计学 2021-05-17 Hongtao Zhang , Alan Y Chiang , Jixian Wang

In Phase I/II dose-finding trials, the objective is to find the Optimal Biological Dose (OBD), a dose that is both safe and efficacious that maximises some optimality criterion based on safety and efficacy. This is further complicated when…

应用统计 · 统计学 2022-03-31 Helen Barnett , Oliver Boix , Dimitris Kontos , Thomas Jaki

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…

应用统计 · 统计学 2021-05-03 Helen Barnett , Oliver Boix , Dimintris Kontos , Thomas Jaki

The purpose of a phase I dose-finding clinical trial is to investigate the toxicity profiles of various doses for a new drug and identify the maximum tolerated dose. Over the past three decades, various dose-finding designs have been…

统计方法学 · 统计学 2021-11-25 Yunshan Duan , Shijie Yuan , Yuan Ji , Peter Mueller

Reinforcement learning (RL) based autonomous driving has emerged as a promising alternative to data-driven imitation learning approaches. However, crafting effective reward functions for RL poses challenges due to the complexity of defining…

机器人学 · 计算机科学 2024-03-29 Xin Ye , Feng Tao , Abhirup Mallik , Burhaneddin Yaman , Liu Ren

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…

机器学习 · 统计学 2025-04-15 Keiichiro Seno , Kota Matsui , Shogo Iwazaki , Yu Inatsu , Shion Takeno , Shigeyuki Matsui

The conventional more-is-better dose selection paradigm, which targets the maximum tolerated dose (MTD), is not suitable for the development of targeted therapies and immunotherapies as the efficacy of these novel therapies may not increase…

统计方法学 · 统计学 2023-08-31 Peng Yang , Daniel Li , Ruitao Lin , Bo Huang , Ying Yuan

The US Food and Drug Administration launched Project Optimus with the aim of shifting the paradigm of dose-finding and selection towards identifying the optimal biological dose that offers the best balance between benefit and risk, rather…

统计方法学 · 统计学 2023-09-13 Ying Yuan , Heng Zhou , Suyu Liu

The traditional more-is-better dose selection paradigm, developed based on cytotoxic chemotherapeutics, is often problematic When applied to the development of novel molecularly targeted agents (e.g., kinase inhibitors, monoclonal…

统计方法学 · 统计学 2022-11-04 Liyun Jiang , Ying Yuan

We consider a dose-optimization design for first-in-human oncology trial that aims to identify a suitable dose for late-phase drug development. The proposed approach, called the Pharmacometrics-Enabled DOse OPtimization (PEDOOP) design,…

应用统计 · 统计学 2024-06-19 Shijie Yuan , Zhanbo Huang , Jiaxin Liu , Yuan Ji

Dose-finding trials for oncology studies are traditionally designed to assess safety in the early stages of drug development. With the rise of molecularly targeted therapies and immuno-oncology compounds, biomarker-driven approaches have…

统计方法学 · 统计学 2025-09-15 Xijin Chen , Pavel Mozgunov , Richard D. Baird , Thomas Jaki

Phase I dose-finding trials are increasingly challenging as the relationship between efficacy and toxicity of new compounds (or combination of them) becomes more complex. Despite this, most commonly used methods in practice focus on…

机器学习 · 计算机科学 2020-06-16 Cong Shen , Zhiyang Wang , Sofia S. Villar , Mihaela van der Schaar

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,…

统计方法学 · 统计学 2024-09-02 Yunlong Yang , Ying Yuan

The US FDA's Project Optimus initiative that emphasizes dose optimization prior to marketing approval represents a pivotal shift in oncology drug development. It has a ripple effect for rethinking what changes may be made to conventional…

统计方法学 · 统计学 2024-06-04 Yuhan Li , Yiding Zhang , Gu Mi , Ji Lin

Recently, the strategy for dose optimization in oncology has shifted to conduct Phase 2 randomized controlled trials with multiple doses. Optimal biologic dose selection from Phase 1 trial data to determine candidate doses for Phase 2…

统计方法学 · 统计学 2023-02-14 Masahiro Kojima

Novel dose-finding designs, using estimation to assign the best estimated maximum- tolerated-dose (MTD) at each point in the experiment, most commonly via Bayesian techniques, have recently entered large-scale implementation in Phase I…

统计方法学 · 统计学 2017-01-24 Assaf P. Oron , Peter D. Hoff

Phase 1-2 designs provide a methodological advance over phase 1 designs for dose finding by using both clinical response and toxicity. A phase 1-2 trial still may fail to select a truly optimal dose. because early response is not a perfect…

应用统计 · 统计学 2024-04-03 Cheng-Han Yang , Peter F. Thall , Ruitao Lin
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