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In randomized dose-finding trials, although drug exposure data form a part of key information for dose selection, the evaluation of the dose-response (DR) relationship often mainly uses DR data. We examine the benefit of…

Methodology · Statistics 2025-08-07 Jixian Wang , Zhiwei Zhang , Ram Tiwari

In the drug discovery process, where experiments can be costly and time-consuming, computational models that predict drug-target interactions are valuable tools to accelerate the development of new therapeutic agents. Estimating the…

Machine Learning · Computer Science 2024-07-22 Hannah Rosa Friesacher , Ola Engkvist , Lewis Mervin , Yves Moreau , Adam Arany

The issue of determining not only an adequate dose but also a dosing frequency of a drug arises frequently in Phase II clinical trials. This results in the comparison of models which have some parameters in common. Planning such studies…

Methodology · Statistics 2017-11-16 Kirsten Schorning , Maria Konstantinou

In this paper, we use Time Scale Calculus (TSC) to formulate and solve pharmacokinetic models exploring multiple dose dynamics. TSC is a mathematical framework that allows the modeling of dynamical systems comprising continuous and discrete…

Dynamical Systems · Mathematics 2024-04-10 Santiago Torres Paz , Jose Ricardo Arteaga Bejarano

In medical treatment and elsewhere, it has become standard to base treatment intensity (dosage) on evidence in randomized trials. Yet it has been rare to study how outcomes vary with dosage. In trials to obtain drug approval, the norm has…

Econometrics · Economics 2023-05-30 Charles F. Manski

Dynamic treatment regimes in oncology and other disease areas often can be characterized by an alternating sequence of treatments or other actions and transition times between disease states. The sequence of transition states may vary…

Applications · Statistics 2015-11-17 Yanxun Xu , Peter Mueller , Abdus S. Wahed , Peter F. Thall

Multidisciplinary team (MDT) consultations are the gold standard for cancer care decision-making, yet current practice lacks structured mechanisms for quantifying consensus and ensuring decision traceability. We introduce a Multi-Agent…

Multiagent Systems · Computer Science 2025-12-17 Xudong Han , Xianglun Gao , Xiaoyi Qu , Zhenyu Yu

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…

Methodology · Statistics 2017-01-24 Assaf P. Oron , Peter D. Hoff

Identification of optimal dose combinations in early phase dose-finding trials is challenging, due to the trade-off between precisely estimating the many parameters required to flexibly model the possibly non-monotonic dose-response…

Methodology · Statistics 2024-02-13 James Willard , Shirin Golchi , Erica E. M. Moodie , Bruno Boulanger , Bradley P. Carlin

The FDA's Project Optimus initiative emphasizes patient-centered dose selection in oncology that balances efficacy and safety. We develop a framework for randomized dose optimization studies that uses clinically interpretable utility scores…

Applications · Statistics 2026-03-24 Xuemin Gu , Cong Xu , Lei Xu , Ying Yu

We consider the optimal design problem for identifying effective dose combinations within drug combination studies where the effect of the combination of two drugs is investigated. Drug combination studies are becoming increasingly…

Pharmacokinetics (PK) plays a critical role in drug development and regulatory decision-making for human and veterinary medicine, directly affecting public health through drug safety and efficacy assessments. However, PK data are often…

Dose-finding clinical trials in oncology aim to estimate the maximum tolerated dose (MTD), based on safety traditionally obtained from the clinician's perspective. While the collection of patient-reported outcomes (PROs) has been advocated…

Applications · Statistics 2023-04-03 Anaïs Andrillon , Lucie Biard , Shing M. Lee

Consider an experiment, where a new drug is tested for the first time on human subjects - healthy volunteers. Such experiments are often performed as dose-escalation studies: a set of increasing doses is pre-selected, individuals are…

Statistics Theory · Mathematics 2016-08-17 Samuel Rosa , Radoslav Harman

This work aims to study the generalizability of a pre-developed deep learning (DL) dose prediction model for volumetric modulated arc therapy (VMAT) for prostate cancer and to adapt the model to three different internal treatment planning…

We propose a data-dependent early completion of dose finding trials for drug-combination. The early completion is determined when a beta-binomial probability for dose retainment with the trial data and the number of remaining patients is…

Methodology · Statistics 2022-02-10 Masahiro Kojima

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…

Optimization and Control · Mathematics 2021-06-08 Corinna Maier , Jana de Wiljes , Niklas Hartung , Charlotte Kloft , Wilhelm Huisinga

Quality control in industrial processes is increasingly making use of prior scientific knowledge, often encoded in physical models that require numerical approximation. Statistical prediction, and subsequent optimization, is key to ensuring…

Other Statistics · Statistics 2018-10-23 Antony Overstall , David Woods , Kieran Martin

The primary goal of dose allocation in phase I trials is to minimize patient exposure to subtherapeutic or excessively toxic doses, while accurately recommending a phase II dose that is as close as possible to the maximum tolerated dose…

Methodology · Statistics 2025-03-21 Jiapeng Xu , Dehua Bi , Shenghua Kelly Fan , Bee Leng Lee , Ying Lu

We present a novel method to improve pharmacokinetics modeling, an essential step of drug development. Conventional models frequently fail to fully represent the intricacies of drug absorption and distribution, which limits their predictive…

Quantitative Methods · Quantitative Biology 2024-12-31 Nazanin Ahmadi , Shupeng Wang , George Karniadakis