Optimal sample sizes for phase II clinical trials and pilot studies
Optimal sample sizes for phase II clinical trials and pilot studies
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DOI：
10.1002/sim.4357
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发表时间：
2012-05-20
影响因子：
2
通讯作者：
Stallard, Nigel
中科院分区：
文献类型：
--
作者：
Stallard, Nigel
III期临床试验的样本量计算方法已得到充分确立并广泛使用。相比之下，对于早期临床试验或试点研究，尽管人们认为III期试验使用的方法不合适，但对于应该使用的方法几乎没有共识。本文从贝叶斯决策理论的角度探讨了这个问题。目的是获得适合于大型资助者（如公共部门机构或大型制药公司）资助的研究的样本量。所获得的样本量是最佳的，因为它们最大限度地减少了每种成功识别的有效疗法所需的平均患者数量，或等效地最大限度地增加了长期成功识别的有效疗法数量。表明II期临床试验入组的患者数量应约为III期研究计划入组患者数量的0.03倍。这与该领域其他研究人员提出的类似，尽管比许多II期试验实际使用的要小。版权所有（C）2011约翰威利父子有限公司
Methodology for sample size calculation for phase III clinical trials is well established and widely used. In contrast, for earlier phase clinical trials or pilot studies, although there is an acceptance that the methods used for phase III trials are not appropriate, there is little consensus over methods that should be used. This paper explores this problem from a Bayesian decision-theoretic perspective. The aim is to obtain sample sizes that would be appropriate for studies funded by a large funder such as a public sector body or major pharmaceutical company. The sample sizes obtained are optimal in that they minimise the average number of patients required per successfully identified effective therapy or equivalently maximise the number of effective therapies successfully identified over a long period. It is indicated that the number of patients included in a phase II clinical trial should be approximately 0.03 times that planned to be included in the phase III study. This is similar to that proposed by other researchers in this area, though rather smaller than actually used for many phase II trials. Copyright (C) 2011 John Wiley & Sons, Ltd.