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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,…

Applications · Statistics 2024-06-19 Shijie Yuan , Zhanbo Huang , Jiaxin Liu , Yuan Ji

We developed a study design for rare disease clinical trials (RDTs) that efficiently evaluate treatments, promotes access to new treatments during treatment development, and optimizes healthcare resource utilization for future treatment…

Applications · Statistics 2016-07-04 Jian Yong , Sohaib H. Mohammad , Yan Yuan

This paper introduces a new Phase I design aimed at enhancing the performance of existing methods, including algorithm-based, model-based, and model-assisted designs. The design, developed by integrating the concept of Fisher information,…

Applications · Statistics 2024-12-11 Xiaojun Zhu

Despite all the advances achieved in the field of tumor-biology research, in most cases conventional therapies including chemotherapy are still the leading choices. The main disadvantage of these treatments, in addition to the low…

Tissues and Organs · Quantitative Biology 2024-01-25 Paola Sanchez-Moreno , Juan Luis Ortega-Vinuesa , Jose Manuel Peula-Garcia , Juan Antonio Marchal , Houria Boulaiz

Drug discovery is lengthy and expensive, with traditional computer-aided design facing limits. This paper examines integrating quantum computing across the drug development cycle to accelerate and enhance workflows and rigorous…

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

Adaptive designs for clinical trials permit alterations to a study in response to accumulating data in order to make trials more flexible, ethical and efficient. These benefits are achieved while preserving the integrity and validity of the…

Adaptive designs have been proposed for clinical trials in which the nuisance parameters or alternative of interest are unknown or likely to be misspecified before the trial. Whereas most previous works on adaptive designs and mid-course…

Methodology · Statistics 2011-05-18 Jay Bartroff , Tze Leung Lai

As a future trend of healthcare, personalized medicine tailors medical treatments to individual patients. It requires to identify a subset of patients with the best response to treatment. The subset can be defined by a biomarker (e.g.…

Methodology · Statistics 2021-08-16 Yitao Lu , Julie Zhou , Li Xing , Xuekui Zhang

Clinical trials are notorious for their high failure rates and steep costs, leading to wasted time and resources spend, prolonged development timelines, and delayed patient access to new therapies. A key contributor to these failures is…

Quantum Physics · Physics 2026-01-19 Laia Domingo , Christine Johnson

Optimal design of a Phase I cancer trial can be formulated as a stochastic optimization problem. By making use of recent advances in approximate dynamic programming to tackle the problem, we develop an approximation of the Bayesian optimal…

Methodology · Statistics 2010-12-01 Jay Bartroff , Tze Leung Lai

Dynamic treatment regimes are sequential decision rules that adapt treatment according to individual time-varying characteristics and outcomes to achieve optimal effects, with applications in precision medicine, personalized…

Methodology · Statistics 2025-10-24 Yuanshan Gao , Yang Bai , Yifan Cui

The appearance of a new dangerous and contagious disease requires the development of a drug therapy faster than what is foreseen by usual mechanisms. Many drug therapy developments consist in investigating through different clinical trials…

Quantitative Methods · Quantitative Biology 2020-03-31 Ezequiel Alvarez , Federico Lamagna , Manuel Szewc

Clinical trials with time-to-event endpoints, such as overall survival (OS) or progression-free survival (PFS), are fundamental for evaluating new treatments, particularly in immuno-oncology. However, modern therapies, such as…

Methodology · Statistics 2025-09-10 James Salsbury , Jeremy Oakley , Steven Julious , Lisa Hampson

Context: Seamless model-based development provides integrated chains of models, covering all software engineering phases. Non-functional requirements (NFRs), like reusability, further play a vital role in software and systems engineering,…

Software Engineering · Computer Science 2017-02-27 Jonas Eckhardt , Daniel Méndez Fernández , Andreas Vogelsang

Successful materials innovations can transform society. However, materials research often involves long timelines and low success probabilities, dissuading investors who have expectations of shorter times from bench to business. A…

Randomized controlled trials (RCTs) can be used to generate guarantees on treatment effects. However, RCTs often spend unnecessary resources exploring sub-optimal treatments, which can reduce the power of treatment guarantees. To address…

Computers and Society · Computer Science 2024-10-16 Santiago Cortes-Gomez , Naveen Raman , Aarti Singh , Bryan Wilder

Scientists are attempting to use models of ever increasing complexity, especially in medicine, where gene-based diseases such as cancer require better modeling of cell regulation. Complex models suffer from uncertainty and experiments are…

Molecular Networks · Quantitative Biology 2018-06-01 Mahdi Imani , Roozbeh Dehghannasiri , Ulisses M. Braga-Neto , Edward R. Dougherty

A clinical trial is an essential step in drug development, which is often costly and time-consuming. In silico trials are clinical trials conducted digitally through simulation and modeling as an alternative to traditional clinical trials.…

Quantitative Methods · Quantitative Biology 2022-09-20 Zifeng Wang , Chufan Gao , Lucas M. Glass , Jimeng Sun

In the search for effective treatments for COVID-19, initial emphasis has been on re-purposed treatments. To maximise the chances of finding successful treatments, novel treatments that have been developed for this disease in particular,…

Applications · Statistics 2020-10-14 Thomas Jaki , Helen Barnett , Andrew Titman , Pavel Mozgunov