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In the first stage of a two-stage study, the researcher uses a statistical model to impute the unobserved exposures. In the second stage, imputed exposures serve as covariates in epidemiological models. Imputation error in the first stage…

Applications · Statistics 2021-07-19 Ron Sarafian , Itai Kloog , Jonathan D. Rosenblatt

Model-assisted designs have garnered significant attention in recent years due to their high accuracy in identifying the maximum tolerated dose (MTD) and their operational simplicity. To identify the MTD, they employ estimated dose limiting…

Applications · Statistics 2025-08-19 Rentaro Wakayama , Tomotaka Momozaki , Shuji Ando

We propose a rule-based statistical design for combination dose-finding trials with two agents. The Ci3+3 design is an extension of the i3+3 design with simple decision rules comparing the observed toxicity rates and equivalence intervals…

Applications · Statistics 2023-03-29 Shijie Yuan , Tianjian Zhou , Yawen Lin , Yuan Ji

Bayesian optimal experimental design (OED) seeks to conduct the most informative experiment under budget constraints to update the prior knowledge of a system to its posterior from the experimental data in a Bayesian framework. Such…

Machine Learning · Computer Science 2024-02-29 Rafael Orozco , Felix J. Herrmann , Peng Chen

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

In radiation therapy (RT) treatment planning, multi-criteria optimization (MCO) supports efficient plan selection but is usually solved for population-based dosimetric criteria and ignores patient-specific biological risk, potentially…

Medical Physics · Physics 2026-01-09 Mara Schubert , Katrin Teichert , Zhongxing Liao , Thomas Bortfeld , Ali Ajdari

The basis generation in reduced order modeling usually requires multiple high-fidelity large-scale simulations that could take a huge computational cost. In order to accelerate these numerical simulations, we introduce a FOM/ROM hybrid…

Numerical Analysis · Mathematics 2021-03-17 Lihong Feng , Guosheng Fu , Zhu Wang

We study the problem of learning individualized dose intervals using observational data. There are very few previous works for policy learning with continuous treatment, and all of them focused on recommending an optimal dose rather than an…

Methodology · Statistics 2022-02-25 Guanhua Chen , Xiaomao Li , Menggang Yu

Recommender systems (RSs) play a crucial role in shaping our digital interactions, influencing how we access and engage with information across various domains. Traditional research has predominantly centered on maximizing recommendation…

Machine Learning · Computer Science 2025-02-20 Hongxu Wang , Zhu Sun , Yingpeng Du , Lu Zhang , Tiantian He , Yew-Soon Ong

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

Model-assisted interval designs such as the Keyboard design are transparent and easy to implement in phase I oncology trials. However, interim decisions based solely on data from the current dose may overlook informative signals from…

Applications · Statistics 2026-05-26 Jiangyan Zhao , Xian Shi , Jin Xu

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

There has been an increasing interest in using interval-based Bayesian designs for dose finding, one of which is the modified toxicity probability interval (mTPI) method. We show that the decision rules in mTPI correspond to an optimal rule…

Methodology · Statistics 2016-09-29 Wentian Guo , Sue-Jane Wang , Shengjie Yang , Suiheng Lin , Yuan Ji

Simulation-based inference (SBI) methods tackle complex scientific models with challenging inverse problems. However, SBI models often face a significant hurdle due to their non-differentiable nature, which hampers the use of gradient-based…

Machine Learning · Computer Science 2023-06-29 Vincent D. Zaballa , Elliot E. Hui

The primary object of a phase I clinical trial is to determine the maximum tolerated dose (MTD). Typically, the MTD is identified using a dose-escalation study, where initial subjects are treated at the lowest dose level and subsequent…

Methodology · Statistics 2014-05-07 Joseph S. Koopmeiners , Andrew Wey

The landscape of dose-finding designs for phase I clinical trials is rapidly shifting in the recent years, noticeably marked by the emergence of interval-based designs. We categorize them as the iDesigns and the IB-Designs. The iDesigns are…

Methodology · Statistics 2017-06-15 Yuan Ji , Shengjie Yang

Biomarker-guided designs are increasingly used to evaluate personalized treatments based on patients' biomarker status in Phase II and III clinical trials. With adaptive enrichment, these designs can improve the efficiency of evaluating the…

Methodology · Statistics 2024-06-11 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

We consider a formal statistical design that allows simultaneous enrollment of a main cohort and a backfill cohort of patients in a dose-finding trial. The goal is to accumulate more information at various doses to facilitate dose…

Applications · Statistics 2024-04-03 Jiaxin Liu , Shijie Yuan , B. Nebiyou Bekele , Yuan Ji

We introduce a gradient-based approach for the problem of Bayesian optimal experimental design to learn causal models in a batch setting -- a critical component for causal discovery from finite data where interventions can be costly or…

Machine Learning · Computer Science 2023-06-05 Yashas Annadani , Panagiotis Tigas , Desi R. Ivanova , Andrew Jesson , Yarin Gal , Adam Foster , Stefan Bauer

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…