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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

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

Basket trials test a single therapeutic treatment on several patient populations under one master protocol. A desirable adaptive design feature in these studies may be the incorporation of new baskets to an ongoing study. Limited basket…

统计方法学 · 统计学 2024-07-09 Libby Daniells , Pavel Mozgunov , Helen Barnett , Alun Bedding , Thomas Jaki

An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…

Basket designs are prospective clinical trials that are devised with the hypothesis that the presence of selected molecular features determine a patient's subsequent response to a particular "targeted" treatment strategy. Basket trials are…

统计计算 · 统计学 2019-08-05 Michael J. Kane , Nan Chen , Alexander M. Kaizer , Xun Jiang , H. Amy Xia , Brian P. Hobbs

Basket trials have emerged as a new class of efficient approaches in oncology to evaluate a new treatment in several patient subgroups simultaneously. In this paper, we extend the key ideas to disease areas outside of oncology, developing a…

统计方法学 · 统计学 2020-06-01 Haiyan Zheng , James M. S. Wason

In recent years, basket trials, which allow the evaluation of an experimental therapy across multiple tumor types within a single protocol, have gained prominence in early-phase oncology development. Unlike traditional trials, which…

应用统计 · 统计学 2025-07-18 Haiming Zhou , Rex Shen , Sutan Wu , Philip He

Therapeutic advancements in oncology have shifted towards targeted therapy based on genomic aberrations. This necessitates innovative statistical approaches in clinical trials, particularly in master protocol studies. Basket trials, a type…

应用统计 · 统计学 2025-02-12 Antonios Daletzakis , Rutger van den Bor , Vincent van der Noort , Kit CB Roes

Precision medicine has led to a paradigm shift allowing the development of targeted drugs that are agnostic to the tumor location. In this context, basket trials aim to identify which tumor types - or baskets - would benefit from the…

统计方法学 · 统计学 2026-01-05 Marcio A. Diniz , Hulya Kocyigit , Erin Moshier , Madhu Mazumdar , Deukwoo Kwon

In experiments to estimate parameters of a parametric model, Bayesian experiment design allows measurement settings to be chosen based on utility, which is the predicted improvement of parameter distributions due to modeled measurement…

统计方法学 · 统计学 2023-01-26 Robert D. McMichael , Sean M. Blakley

Basket trials are increasingly used for the simultaneous evaluation of a new treatment in various patient subgroups under one overarching protocol. We propose a Bayesian approach to sample size determination in basket trials that permit…

统计方法学 · 统计学 2022-09-02 Haiyan Zheng , Michael J. Grayling , Pavel Mozgunov , Thomas Jaki , James M. S. Wason

The conventional approach to Bayesian decision-theoretic experiment design involves searching over possible experiments to select a design that maximizes the expected value of a specified utility function. The expectation is over the joint…

统计方法学 · 统计学 2023-04-18 Tommie A. Catanach , Niladri Das

Motivated by recent developments in designing algorithms based on individual item scores for solving utility maximization problems, we study the framework of using test scores, defined as a statistic of observed individual item performance…

数据结构与算法 · 计算机科学 2022-02-28 Dabeen Lee , Milan Vojnovic , Se-Young Yun

Bayesian clinical trials can benefit of available historical information through the elicitation of informative prior distributions. Concerns are however often raised about the potential for prior-data conflict and the impact of Bayes test…

统计方法学 · 统计学 2022-12-01 Silvia Calderazzo , Annette Kopp-Schneider

We present a statistical perspective on boosting. Special emphasis is given to estimating potentially complex parametric or nonparametric models, including generalized linear and additive models as well as regression models for survival…

统计方法学 · 统计学 2008-12-18 Peter Bühlmann , Torsten Hothorn

Heterogeneity in efficacy is sometimes observed across baskets in basket trials. In this study, we propose a model-free clustering framework that groups baskets based on transition probabilities derived from the trajectories of treatment…

统计方法学 · 统计学 2026-01-05 Masahiro Kojima , Keisuke Hanada , Atsuya Sato

Validation of building energy simulation programs is of major interest to both users and modellers. To achieve such a task, it is essential to apply a methodology based on a priori test and empirical validation. A priori test consists in…

计算工程、金融与科学 · 计算机科学 2012-12-26 Ted Soubdhan , Thierry Alex Mara , Harry Boyer , Anis Younès

Basket trials are a new type of clinical trial in which a treatment is investigated in several subgroups. For the analysis of these trials, information is shared between the subgroups based on the observed data to increase the power. Many…

统计计算 · 统计学 2024-03-27 Lukas Baumann

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

Due to the high computational demands executing a rigorous comparison between hyperparameter optimization (HPO) methods is often cumbersome. The goal of this paper is to facilitate a better empirical evaluation of HPO methods by providing…

机器学习 · 计算机科学 2019-05-14 Aaron Klein , Frank Hutter
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