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In basket trials a treatment is investigated in several subgroups. They are primarily used in oncology in early clinical phases as single-arm trials with a binary endpoint. For their analysis primarily Bayesian methods have been suggested,…

统计方法学 · 统计学 2024-02-16 Lukas Baumann , Lukas Sauer , Meinhard Kieser

This work studies a dynamic mechanism design problem in which a principal delegates decision makings to a group of privately-informed agents without the monetary transfer or burning. We consider that the principal privately possesses…

计算机科学与博弈论 · 计算机科学 2022-01-19 Tao Zhang , Quanyan Zhu

Bayesian Optimal Experimental Design (BOED) provides a rigorous framework for decision-making tasks in which data acquisition is often the critical bottleneck, especially in resource-constrained settings. Traditionally, BOED typically…

机器学习 · 统计学 2026-04-24 Di Wu , Ling Liang , Haizhao Yang

Bayesian optimal experimental design (BOED) is a principled framework for making efficient use of limited experimental resources. Unfortunately, its applicability is hampered by the difficulty of obtaining accurate estimates of the expected…

Basket trials examine the efficacy of an intervention in multiple patient subgroups simultaneously. The division into subgroups, called baskets, is based on matching medical characteristics, which may result in small sample sizes within…

统计方法学 · 统计学 2024-09-17 Sabrina Schmitt , Lukas Baumann

The optimal design of experiments typically involves solving an NP-hard combinatorial optimization problem. In this paper, we aim to develop a globally convergent and practically efficient optimization algorithm. Specifically, we consider a…

计量经济学 · 经济学 2022-11-29 Yiping Lu , Jiajin Li , Lexing Ying , Jose Blanchet

Phase I clinical trials are designed to test the safety (non-toxicity) of drugs and find the maximum tolerated dose (MTD). This task becomes significantly more challenging when multiple-drug dose-combinations (DC) are involved, due to the…

机器学习 · 计算机科学 2021-01-27 Hyun-Suk Lee , Cong Shen , William Zame , Jang-Won Lee , Mihaela van der Schaar

There is growing interest in platform trials that allow for adding of new treatment arms as the trial progresses as well as being able to stop treatments part way through the trial for either lack of benefit/futility or for superiority. In…

统计方法学 · 统计学 2021-12-14 Peter Greenstreet , Thomas Jaki , Alun Bedding , Chris Harbron , Pavel Mozgunov

In this paper, a methodology is proposed that enables to analyze the sensitivity of the outcome of a therapy to unavoidable high dispersion of the patient specific parameters on one hand and to the choice of the parameters that define the…

系统与控制 · 电气工程与系统科学 2022-05-17 Mazen Alamir

In oncology, phase II studies are crucial for clinical development plans as such studies identify potent agents with sufficient activity to continue development in the subsequent phase III trials. Traditionally, phase II studies are…

统计方法学 · 统计学 2023-08-08 Takuya Yoshimoto , Satoru Shinoda , Kouji Yamamoto , Kouji Tahata

High dimensional case control studies are ubiquitous in the biological sciences, particularly genomics. To maximise power while constraining cost and to minimise type-1 error rates, researchers typically seek to replicate findings in a…

统计方法学 · 统计学 2017-07-11 James Liley

We define a Bayesian semi-parametric model to effectively conduct inference with unaligned longitudinal binary data. The proposed strategy is motivated by data from the Human Epilepsy Project (HEP), which collects seizure occurrence data…

统计方法学 · 统计学 2025-05-13 Beatrice Cantoni , Giovanni Poli , Elizabeth Juarez-Colunga , Peter Müller

Automatic Chemical Design is a framework for generating novel molecules with optimized properties. The original scheme, featuring Bayesian optimization over the latent space of a variational autoencoder, suffers from the pathology that it…

机器学习 · 统计学 2019-08-13 Ryan-Rhys Griffiths , José Miguel Hernández-Lobato

There is a growing interest in the implementation of platform trials, which provide the flexibility to incorporate new treatment arms during the trial and the ability to halt treatments early based on lack of benefit or observed…

统计方法学 · 统计学 2023-08-25 Peter Greenstreet , Thomas Jaki , Alun Bedding , Pavel Mozgunov

Bayesian experimental design involves the optimal allocation of resources in an experiment, with the aim of optimising cost and performance. For implicit models, where the likelihood is intractable but sampling from the model is possible,…

机器学习 · 统计学 2019-02-26 Steven Kleinegesse , Michael Gutmann

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…

统计理论 · 数学 2018-03-15 Pavel Mozgunov , Thomas Jaki

Regression Discontinuity Design (RDD) is a popular framework for estimating a causal effect in settings where treatment is assigned if an observed covariate exceeds a fixed threshold. We consider estimation and inference in the common…

统计理论 · 数学 2025-04-16 Kevin Tao , Y. Samuel Wang , David Ruppert

Breakthroughs in cancer biology have defined new research programs emphasizing the development of therapies that target specific pathways in tumor cells. Innovations in clinical trial design have followed with master protocols defined by…

统计方法学 · 统计学 2020-07-09 Alexander M. Kaizer , Joseph S. Koopmeiners , Nan Chen , Brian P. Hobbs

Group sequential designs drive innovation in clinical, industrial, and corporate settings. Early stopping for failure in sequential designs conserves experimental resources, whereas early stopping for success accelerates access to improved…

统计方法学 · 统计学 2025-11-27 Luke Hagar , Shirin Golchi , Marina B. Klein

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,…

应用统计 · 统计学 2024-12-11 Xiaojun Zhu