Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls.
Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls.
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DOI：
10.1002/sim.6472
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发表时间：
2016-02-10
影响因子：
2
通讯作者：
Wassmer G
中科院分区：
文献类型：
--
作者：
Bauer P;Bretz F;Dragalin V;König F;Wassmer G
“适应性设计的多阶段测试”是Peter Bauer在1989年发表在德国医学与生物学杂志Biometrie und Informatik上的一篇文章的标题。该杂志已经不存在了，但在过去的25年里，该方法在科学界引起了广泛的兴趣。这种多阶段自适应设计的使用从一开始就引起了许多有争议的讨论，特别是在Bauer和Köhne 1994年在Biometrics上发表的文章之后：对这种设计的潜在应用的广泛热情面临着关于其统计效率的关键位置。尽管存在这一争议，或者可能是因为存在这一争议，多年来，这一方法及其应用领域稳步增长，世界各地学术界、工业界和机构的统计人员作出了重大贡献。与此同时，这种类型的自适应设计已成为美国和欧洲两个主要监管指导文件的主题，该领域仍在不断发展。在适应性设计的最重要应用中，包括样本量重新评估、治疗选择程序和群体富集设计的发展尤其值得注意。在这篇文章中，我们从不同的角度总结了过去25年的发展。我们提供了早期的历史概述，回顾了关键的方法概念，并总结了监管和行业对此类设计的看法。然后，我们用三个案例说明了适应性设计的应用，包括揭盲样本量重新评估，适应性治疗选择和适应性终点选择。我们还讨论了评估和执行这样的设计软件的可用性。最后，我们对一开始的期望是如何实现的进行了批判性的回顾，如果没有实现，我们将讨论为什么没有实现的潜在原因。© 2015作者。出版社：John Wiley & Sons Ltd
‘Multistage testing with adaptive designs’ was the title of an article by Peter Bauer that appeared 1989 in the German journal Biometrie und Informatik in Medizin und Biologie. The journal does not exist anymore but the methodology found widespread interest in the scientific community over the past 25 years. The use of such multistage adaptive designs raised many controversial discussions from the beginning on, especially after the publication by Bauer and Köhne 1994 in Biometrics: Broad enthusiasm about potential applications of such designs faced critical positions regarding their statistical efficiency. Despite, or possibly because of, this controversy, the methodology and its areas of applications grew steadily over the years, with significant contributions from statisticians working in academia, industry and agencies around the world. In the meantime, such type of adaptive designs have become the subject of two major regulatory guidance documents in the US and Europe and the field is still evolving. Developments are particularly noteworthy in the most important applications of adaptive designs, including sample size reassessment, treatment selection procedures, and population enrichment designs. In this article, we summarize the developments over the past 25 years from different perspectives. We provide a historical overview of the early days, review the key methodological concepts and summarize regulatory and industry perspectives on such designs. Then, we illustrate the application of adaptive designs with three case studies, including unblinded sample size reassessment, adaptive treatment selection, and adaptive endpoint selection. We also discuss the availability of software for evaluating and performing such designs. We conclude with a critical review of how expectations from the beginning were fulfilled, and – if not – discuss potential reasons why this did not happen. © 2015 The Authors. Statistics in Medicine Published by John Wiley & Sons Ltd.
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影响因子：
2
作者：
BIRKETT, MA;DAY, SJ
通讯作者：
DAY, SJ
影响因子：
1.7
作者：
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通讯作者：
BAUER, P
影响因子：
0.9
作者：
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通讯作者：
Posch, M
影响因子：
1.9
作者：
Brannath, Werner;Mehta, Cyrus R.;Posch, Martin
通讯作者：
Posch, Martin
影响因子：
2
作者：
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通讯作者：
Koenig, F