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Related papers: ROSE: Randomized Optimal Selection Design for Dose…

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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…

Methodology · Statistics 2018-07-17 Pavel Mozgunov , Thomas Jaki

Significant evidence has become available that emphasizes the importance of personalization in medicine. In fact, it has become a common belief that personalized medicine is the future of medicine. The core of personalized medicine is the…

Methodology · Statistics 2020-04-30 Qiong Zhang , Amin Khademi , Yongjia Song

Efforts to develop biomarker-targeted anti-cancer therapies have progressed rapidly in recent years. Six antibodies acting on programmed death ligand 1 or programmed death 1 pathways were approved in 75 cancer indications between 2015 and…

Methodology · Statistics 2022-10-07 Emily C. Zabor , Alexander M. Kaizer , Nathan A. Pennell , Brian P. Hobbs

Sequential recommendation (SR) models are typically trained on user-item interactions which are affected by the system exposure bias, leading to the user preference learned from the biased SR model not being fully consistent with the true…

Information Retrieval · Computer Science 2023-12-13 Jiyuan Yang , Yue Ding , Yidan Wang , Pengjie Ren , Zhumin Chen , Fei Cai , Jun Ma , Rui Zhang , Zhaochun Ren , Xin Xin

We study the problem of finding the optimal dosage in early stage clinical trials through the multi-armed bandit lens. We advocate the use of the Thompson Sampling principle, a flexible algorithm that can accommodate different types of…

Machine Learning · Statistics 2020-04-09 Maryam Aziz , Emilie Kaufmann , Marie-Karelle Riviere

There is no doubt on the importance of randomisation in agricultural experiments by agronomists and biometricians. Even when agronomists extend the experimentation from small trials to large on-farm trials, randomised designs predominate…

Applications · Statistics 2022-06-22 Zhanglong Cao , Andrew Grose , Jordan Brown , Suman Rakshit

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

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

The question of selecting the "best" amongst different choices is a common problem in statistics. In drug development, our motivating setting, the question becomes, for example: what is the dose that gives me a pre-specified risk of…

Statistics Theory · Mathematics 2018-03-15 Pavel Mozgunov , Thomas Jaki

Background. The DURATIONS design has been recently proposed as a practical alternative to a standard two-arm non-inferiority design when the goal is to optimise some continuous aspect of treatment administration, e.g. duration or frequency,…

Applications · Statistics 2023-04-20 Matteo Quartagno , Ehsan Ghorani , Tim P Morris , Michael J Seckl , Mahesh KB Parmar

It is widely recognised that semiparametric efficient estimation can be hard to achieve in practice: estimators that are in theory efficient may require unattainable levels of accuracy for the estimation of complex nuisance functions. As a…

Statistics Theory · Mathematics 2024-12-18 Elliot H. Young , Rajen D. Shah

There is a movement in design of experiments away from the classic randomization put forward by Fisher, Cochran and others to one based on optimization. In fixed-sample trials comparing two groups, measurements of subjects are known in…

Methodology · Statistics 2018-10-22 Adam Kapelner , Abba M. Krieger , Uri Shalit , David Azriel

Treatment planning uncertainties are typically managed using margin-based or robust optimization. Margin-based methods expand the clinical target volume (CTV) to a planning target volume, generally unsuited for proton therapy. Robust…

Oncology dose-finding trials are shifting from identifying the maximum tolerated dose (MTD) to determining the optimal biological dose (OBD), driven by the need for efficient methods that consider both toxicity and efficacy. This is…

Methodology · Statistics 2025-04-01 Hao Sun , Jieqi Tu , Revathi Ananthakrishnan , Eunhee Kim

An accurately identified maximum tolerated dose (MTD) serves as the cornerstone of successful subsequent phases in oncology drug development. Bayesian logistic regression model (BLRM) is a popular and versatile model-based dose-finding…

Methodology · Statistics 2021-05-17 Hongtao Zhang , Alan Y Chiang , Jixian Wang

Multi-arm multi-stage (MAMS) trials have gained popularity, due to their improved efficiency in evaluating multiple treatments. A traditional MAMS trial often decreases the expected sample size of the trial compared to just running a…

In a recent paper Dette et al. (2014) introduced optimal design problems for dose fnding studies with an active control. These authors concentrated on regression models with normal distributed errors (with known variance) and the problem of…

Methodology · Statistics 2014-11-03 Holger Dette , Katrin Kettelhake , Frank Bretz

Dose optimization in oncology clinical trials has shifted from seeking the maximum tolerated dose to identifying the Optimal Biological Dose (OBD) that balances therapeutic benefits and risks across multiple clinical attributes. Existing…

Methodology · Statistics 2025-05-07 Fanni Zhang , Kristine Broglio , Michael Sweeting , Gina D'Angelo

Response adaptive randomization (RAR) is appealing from methodological, ethical, and pragmatic perspectives in the sense that subjects are more likely to be randomized to better performing treatment groups based on accumulating data.…

Methodology · Statistics 2022-08-03 Tianyu Zhan , Lu Cui , Ziqian Geng , Lanju Zhang , Yihua Gu , Ivan S. F. Chan

We consider distributed statistical optimization in one-shot setting, where there are $m$ machines each observing $n$ i.i.d. samples. Based on its observed samples, each machine then sends an $O(\log(mn))$-length message to a server, at…

Machine Learning · Computer Science 2019-11-12 Arsalan Sharifnassab , Saber Salehkaleybar , S. Jamaloddin Golestani