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Related papers: A Simulation Study Evaluating Phase I Clinical Tri…

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Hierarchical random effect models are used for different purposes in clinical research and other areas. In general, the main focus is on population parameters related to the expected treatment effects or group differences among all units of…

Applications · Statistics 2021-04-07 Maryna Prus , Norbert Benda , Rainer Schwabe

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

Often both Aggregate Data (AD) studies and Individual Patient Data (IPD) studies are available for specific treatments. Combining these two sources of data could improve the overall meta-analytic estimates of treatment effects. Moreover,…

Methodology · Statistics 2021-11-15 Neha Agarwala , Junyong Park , Anindya Roy

Optimizing doses for multiple indications is challenging. The pooled approach of finding a single optimal biological dose (OBD) for all indications ignores that dose-response or dose-toxicity curves may differ between indications, resulting…

Methodology · Statistics 2024-10-07 Shuqi Wang , Peter F. Thall , Kentaro Takeda , Ying Yuan

Project Optimus, an initiative by the FDA's Oncology Center of Excellence, seeks to reform the dose-optimization and dose-selection paradigm in oncology. We propose a dose-optimization design that considers plateau efficacy profiles,…

Applications · Statistics 2025-04-22 Rebecca B. Silva , Bin Cheng , Shing M. Lee

Designing and evaluating personalized and proactive assistant agents remains challenging due to the time, cost, and ethical concerns associated with human-in-the-loop experimentation. Existing Human-Computer Interaction (HCI) methods often…

Human-Computer Interaction · Computer Science 2025-11-25 Ziyi Xuan , Yiwen Wu , Xuhai Xu , Vinod Namboodiri , Mooi Choo Chuah , Yu Yang

We propose Locally Optimal Restricted Designs (LORDs) for phase I/II dose-finding studies that focus on both efficacy and toxicity outcomes. As an illustrative application, we find various LORDs for a 4-parameter continuation-ratio (CR)…

Methodology · Statistics 2025-09-09 Oleksandr Sverdlov , Yevgen Ryeznik , Weng Kee Wong

Many chemical and biological experiments involve multiple treatment factors and often it is convenient to fit a nonlinear model in these factors. This nonlinear model can be mechanistic, empirical or a hybrid of the two. Motivated by…

Computation · Statistics 2018-10-09 Yuanzhi Huang , Steven Gilmour , Kalliopi Mylona , Peter Goos

Educational research often studies subjects that are in naturally clustered groups of classrooms or schools. When designing a randomized experiment to evaluate an intervention directed at teachers, but with effects on teachers and their…

Applications · Statistics 2009-08-17 Brenda Jenney , Sharon Lohr

Differential Diagnosis (DDx) is a fundamental yet complex aspect of clinical decision-making, in which physicians iteratively refine a ranked list of possible diseases based on symptoms, antecedents, and medical knowledge. While recent…

Computation and Language · Computer Science 2025-06-16 Daniel Rose , Chia-Chien Hung , Marco Lepri , Israa Alqassem , Kiril Gashteovski , Carolin Lawrence

Phase I dose escalation trials in oncology generally aim to find the maximum tolerated dose (MTD). However, with the advent of molecular targeted therapies and antibody drug conjugates, dose limiting toxicities are less frequently observed,…

Methodology · Statistics 2025-08-19 Ayon Mukherjee , Jonathan L. Moscovici , Zheng Liu

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

We investigate the optimal design of experimental studies that have pre-treatment outcome data available. The average treatment effect is estimated as the difference between the weighted average outcomes of the treated and control units. A…

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

The long-term efficacy of targeted therapeutics for cancer treatment can be significantly limited by the type of therapy and development of drug resistance, inter alia. Experimental studies indicate that the factors enhancing acquisition of…

Tissues and Organs · Quantitative Biology 2021-02-16 Joseph Malinzi , Kevin Bosire Basita , Sara Padidar , Henry A. Adeola

Literature on the modeling and simulation of complex adaptive systems (cas) has primarily advanced vertically in different scientific domains with scientists developing a variety of domain-specific approaches and applications. However,…

Multiagent Systems · Computer Science 2017-08-09 Muaz A. Niazi

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…

Generally, combinatorial design concerns with the arrangement of a finite set of elements into patterns (subsets, words, arrays) according to specified rules. The usefulness of this design method is that the number of input combination can…

Networking and Internet Architecture · Computer Science 2018-04-24 Bestoun S. Ahmed , Amin S. Mohammad , Hemin T. Essa

The widespread application of Artificial Intelligence (AI) techniques has significantly influenced the development of new therapeutic agents. These computational methods can be used to design and predict the properties of generated…

Neural and Evolutionary Computing · Computer Science 2024-08-21 Arthur Cerveira , Frederico Kremer , Darling de Andrade Lourenço , Ulisses B Corrêa

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

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