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相关论文: A web application for the design of multi-arm clin…

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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

The development of applications for obtaining interpretable results in a simple and summarized manner in multi-state models is a research field with great potential, namely in terms of using open source tools that can be easily implemented…

统计计算 · 统计学 2022-02-21 Gustavo Soutinho , Luís Meira-Machado

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

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

统计方法学 · 统计学 2021-08-31 Elja Arjas , Dario Gasbarra

A multi-arm multi-stage trial is a multi-arm trial which includes interim analyses - analysing the data at certain specified points, generally discontinuing treatments which are concluded to not work and proceeding with the remainder. It is…

统计方法学 · 统计学 2022-11-14 Martin Law

Multi-arm multi-stage trial designs can bring notable gains in efficiency to the drug development process. However, for normally distributed endpoints, the determination of a design typically depends on the assumption that the patient…

统计方法学 · 统计学 2017-10-11 Michael Grayling , James Wason , Adrian Mander

The precise calculation of sample sizes is a crucial aspect in the design of clinical trials particularly for pharmaceutical statisticians. While various R statistical software packages have been developed by researchers to estimate…

The interAdapt R package is designed to be used by statisticians and clinical investigators to plan randomized trials. It can be used to determine if certain adaptive designs offer tangible benefits compared to standard designs, in the…

应用统计 · 统计学 2014-06-19 Aaron Fisher , Harris Jaffee , Michael Rosenblum

The micro-randomized trial (MRT) is a new experimental design which allows for the investigation of the proximal effects of a "just-in-time" treatment, often provided via a mobile device as part of a mobile health intervention. As with a…

统计方法学 · 统计学 2020-08-07 Nicholas J. Seewald , Ji Sun , Peng Liao

Numerous publications have now addressed the principles of designing, analyzing, and reporting the results of, stepped-wedge cluster randomized trials. In contrast, there is little research available pertaining to the design and analysis of…

统计方法学 · 统计学 2018-06-29 Michael Grayling , Adrian Mander , James Wason

The package mvlearnR and accompanying Shiny App is intended for integrating data from multiple sources or views or modalities (e.g. genomics, proteomics, clinical and demographic data). Most existing software packages for multiview learning…

基因组学 · 定量生物学 2023-11-29 Elise F. Palzer , Sandra E. Safo

Background: trials to identify the minimal effective treatment duration are needed in different therapeutic areas, including bacterial infections, TB and Hepatitis--C. However, standard non-inferiority designs have several limitations,…

Simulations are valuable tools for empirically evaluating the properties of statistical methods and are primarily employed in methodological research to draw general conclusions about methods. In addition, they can often be useful to…

其他统计学 · 统计学 2025-10-08 Anne-Laure Boulesteix , Patrick Callahan , Luzia Hanssum , Vincent Gaertner , Eva Hoster

Multi-arm trials are gaining interest in practice given the statistical and logistical advantages they can offer. The standard approach uses a fixed allocation ratio, but there is a call for making it adaptive and skewing the allocation of…

统计方法学 · 统计学 2026-01-16 Gianmarco Caruso , Pavel Mozgunov

Simulation offers a simple and flexible way to estimate the power of a clinical trial when analytic formulae are not available. The computational burden of using simulation has, however, restricted its application to only the simplest of…

统计方法学 · 统计学 2020-12-04 Duncan T. Wilson , Rebecca E. A. Walwyn , Richard Hooper , Julia Brown , Amanda J. Farrin

We consider applying multi-armed bandits to model-assisted designs for dose-finding clinical trials. Multi-armed bandits are very simple and powerful methods to determine actions to maximize a reward in a limited number of trials. Among the…

统计方法学 · 统计学 2022-01-17 Masahiro Kojima

It is difficult or infeasible to directly measure how much of a drug actually enters the human brain and a brain tumor, how long it remains there, and to estimate drug-specific or patient-specific parameters, as well as how changes in these…

动力系统 · 数学 2026-03-13 Charuka D. Wickramasinghe , Nelum S. S. M. Hapuhinna

Randomized experiments play a major role in data-driven decision making across many different fields and disciplines. In medicine, for example, randomized controlled trials (RCTs) are the backbone of clinical trial methodology for testing…

应用统计 · 统计学 2016-08-30 Andrew W. Correia

Combination drug therapies hold significant promise for enhancing treatment efficacy, particularly in fields such as oncology, immunotherapy, and infectious diseases. However, designing clinical trials for these regimens poses unique…

统计方法学 · 统计学 2025-06-04 Nan Miles Xi , Man Mandy Jin , Lin Wang , Xin Huang

External controls (ECs) from historical trials or real-world data have gained increasing attention as a way to augment hybrid and single-arm trials, especially when balanced randomization is infeasible. While most existing work has focused…

统计方法学 · 统计学 2025-12-15 Yujing Gao , Xiang Zhang , Shu Yang
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