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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…

Applications · Statistics 2026-01-13 Kyong Ju Lee , Yuan Ji

Computer aided drug design is a promising approach to reduce the tremendous costs, i.e. time and resources, for developing new medicinal drugs. It finds application in aiding the traversal of the vast chemical space of potentially useful…

Neural and Evolutionary Computing · Computer Science 2024-05-02 Tomoya Hömberg , Sanaz Mostaghim , Satoru Hiwa , Tomoyuki Hiroyasu

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…

Applications · Statistics 2022-03-02 Huaqing Jin , Wenbin Du , Guosheng Yin

Debiased recommendation with a randomized dataset has shown very promising results in mitigating the system-induced biases. However, it still lacks more theoretical insights or an ideal optimization objective function compared with the…

Information Retrieval · Computer Science 2023-03-22 Dugang Liu , Pengxiang Cheng , Zinan Lin , Xiaolian Zhang , Zhenhua Dong , Rui Zhang , Xiuqiang He , Weike Pan , Zhong Ming

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

Moment-based distributionally robust optimization (DRO) provides an optimization framework to integrate statistical information with traditional optimization approaches. Under this framework, one assumes that the underlying joint…

Optimization and Control · Mathematics 2023-11-01 Shiyi Jiang , Jianqiang Cheng , Kai Pan , Zuo-Jun Max Shen

We study the design of experiments with multiple treatment levels, a setting common in clinical trials and online A/B/n testing. Unlike single-treatment studies, practical analyses of multi-treatment experiments typically first select a…

Methodology · Statistics 2025-10-07 Jiachen Xu , Jian Qian , Zijun Gao

The dose delivered to the planning target volume by proton beams is highly conformal, sparing organs at risk and normal tissues. New treatment planning systems adapted to spot scanning techniques have been recently proposed to…

Medical Physics · Physics 2022-05-18 François Smekens , Nicolas Freud , Bruno Sixou , Guillaume Beslon , Jean M Létang

Adaptive approaches, allowing for more flexible trial design, have been proposed for individually randomized trials to save time or reduce sample size. However, adaptive designs for cluster-randomized trials in which groups of participants…

Methodology · Statistics 2022-01-10 Junwei Shen , Shirin Golchi , Erica E. M. Moodie , David Benrimoh

This paper presents a quasi-sequential optimal design framework for toxicology experiments, specifically applied to sea urchin embryos. The authors propose a novel approach combining robust optimal design with adaptive, stage-based testing…

Methodology · Statistics 2025-03-04 Elvis Han Cui , Michael Collins , Jessica Munson , Weng Kee Wong

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…

Methodology · Statistics 2021-11-25 Yunshan Duan , Shijie Yuan , Yuan Ji , Peter Mueller

We consider design issues for toxicology studies when we have a continuous response and the true mean response is only known to be a member of a class of nested models. This class of non-linear models was proposed by toxicologists who were…

Statistics Theory · Mathematics 2010-11-29 Holger Dette , Andrey Pepelyshev , Piter Shpilev , Weng Kee Wong

The growing availability of observational databases like electronic health records (EHR) provides unprecedented opportunities for secondary use of such data in biomedical research. However, these data can be error-prone and need to be…

Methodology · Statistics 2024-05-28 Sarah C. Lotspeich , Gustavo G. C. Amorim , Pamela A. Shaw , Ran Tao , Bryan E. Shepherd

We present an optimized rerandomization design procedure for a non-sequential treatment-control experiment. Randomized experiments are the gold standard for finding causal effects in nature. But sometimes random assignments result in…

Methodology · Statistics 2021-01-26 Adam Kapelner , Abba M. Krieger , Michael Sklar , David Azriel

In most clinical trials, patients are randomized with equal probability among treatments to obtain an unbiased estimate of the treatment effect. Response-adaptive randomization (RAR) has been proposed for ethical reasons, where the…

Applications · Statistics 2019-09-16 Thevaa Chandereng , Rick Chappell

The goal of this paper is to make Optimal Experimental Design (OED) computationally feasible for problems involving significant computational expense. We focus exclusively on the Mean Objective Cost of Uncertainty (MOCU), which is a…

Optimization and Control · Mathematics 2020-12-09 Anthony M. DeGennaro , Francis J. Alexander

Group distributionally robust optimization (GDRO) aims to develop models that perform well across $m$ distributions simultaneously. Existing GDRO algorithms can only process a fixed number of samples per iteration, either 1 or $m$, and…

Machine Learning · Computer Science 2025-05-22 Haomin Bai , Dingzhi Yu , Shuai Li , Haipeng Luo , Lijun Zhang

Recent developments in artificial intelligence and automation support a new drug design paradigm: autonomous drug design. Under this paradigm, generative models can provide suggestions on thousands of molecules with specific properties, and…

Bayesian optimization (BO) is a popular approach for sample-efficient optimization of black-box objective functions. While BO has been successfully applied to a wide range of scientific applications, traditional approaches to…

Machine Learning · Computer Science 2023-05-04 Natalie Maus , Kaiwen Wu , David Eriksson , Jacob Gardner

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
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