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Incorporating historical data or real-world evidence has a great potential to improve the efficiency of phase I clinical trials and to accelerate drug development. For model-based designs, such as the continuous reassessment method (CRM),…

统计方法学 · 统计学 2020-06-11 Yanhong Zhou , J. Jack Lee , Shunguang Wang , Stuart Bailey , Ying Yuan

Model-assisted interval designs such as the Keyboard design are transparent and easy to implement in phase I oncology trials. However, interim decisions based solely on data from the current dose may overlook informative signals from…

应用统计 · 统计学 2026-05-26 Jiangyan Zhao , Xian Shi , Jin Xu

Two useful strategies to speed up drug development are to increase the patient accrual rate and use novel adaptive designs. Unfortunately, these two strategies often conflict when the evaluation of the outcome cannot keep pace with the…

统计方法学 · 统计学 2018-07-24 Ruitao Lin , Ying Yuan

Purpose: The early identification of maximum tolerated dose (MTD) in phase I trial leads to faster progression to a phase II trial or an expansion cohort to confirm efficacy. Methods: We propose a novel adaptive design for identifying MTD…

统计方法学 · 统计学 2021-10-07 Masahiro Kojima

Background: Phase I trials desire to identify the maximum tolerated dose (MTD) early and proceed quickly to an expansion cohort or phase II trial for efficacy. We propose an early completion method based on multiple dosages to accelerate…

定量方法 · 定量生物学 2021-10-04 Masahiro Kojima

Adaptive subgroup enrichment design is an efficient design framework that allows accelerated development for investigational treatments while also having flexibility in population selection within the course of the trial. The adaptive…

统计方法学 · 统计学 2022-10-21 Liwen Wu , Qing Li , Mengya Liu , Jianchang Lin

In clinical trials, there is potential to improve precision and reduce the required sample size by appropriately adjusting for baseline variables in the statistical analysis. This is called covariate adjustment. Despite recommendations by…

统计方法学 · 统计学 2022-06-20 Kelly Van Lancker , Joshua Betz , Michael Rosenblum

We propose an adaptive design for early phase drug combination cancer trials with the goal of estimating the maximum tolerated dose (MTD). A nonparametric Bayesian model, using beta priors truncated to the set of partially ordered dose…

应用统计 · 统计学 2019-10-22 Zahra S. Razaee , Galen Wien-Cook , Mourad Tighiouart

In the development of new cancer treatment, an essential step is to determine the maximum tolerated dose (MTD) via phase I clinical trials. Generally speaking, phase I trial designs can be classified as either model-based or algorithm-based…

应用统计 · 统计学 2022-03-02 Huaqing Jin , Wenbin Du , Guosheng Yin

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

It is increasingly common for therapies in oncology to be given in combination. In some cases, patients can benefit from the interaction between two drugs, although often at the risk of higher toxicity. A large number of designs to conduct…

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

We propose a new integrated phase I/II trial design to identify the most efficacious dose combination that also satisfies certain safety requirements for drug-combination trials. We first take a Bayesian copula-type model for dose finding…

应用统计 · 统计学 2011-08-09 Ying Yuan , Guosheng Yin

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

Model-assisted designs have garnered significant attention in recent years due to their high accuracy in identifying the maximum tolerated dose (MTD) and their operational simplicity. To identify the MTD, they employ estimated dose limiting…

应用统计 · 统计学 2025-08-19 Rentaro Wakayama , Tomotaka Momozaki , Shuji Ando

Phase I oncology trials aim to identify a safe dose - often the maximum tolerated dose (MTD) - for subsequent studies. Conventional designs focus on population-level toxicity modeling, with recent attention on leveraging pharmacokinetic…

应用统计 · 统计学 2026-01-13 Kyong Ju Lee , Yuan Ji

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

Model-informed precision dosing (MIPD) is a quantitative dosing framework that combines prior knowledge on the drug-disease-patient system with patient data from therapeutic drug/ biomarker monitoring (TDM) to support individualized dosing…

最优化与控制 · 数学 2021-06-08 Corinna Maier , Jana de Wiljes , Niklas Hartung , Charlotte Kloft , Wilhelm Huisinga

Dose-finding trials are a key component of the drug development process and rely on a statistical design to help inform dosing decisions. Triallists wishing to choose a design require knowledge of operating characteristics of competing…

统计计算 · 统计学 2025-03-11 Michael Sweeting , Daniel Slade , Dan Jackson , Kristian Brock

Leveraging preclinical animal data for a phase I first-in-man trial is appealing yet challenging. A prior based on animal data may place large probability mass on values of the dose-toxicity model parameter(s), which appear infeasible in…

应用统计 · 统计学 2020-04-15 Haiyan Zheng , Lisa V. Hampson
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