An audit of sample sizes for pilot and feasibility trials being undertaken in the United Kingdom registered in the United Kingdom Clinical Research Network database.
An audit of sample sizes for pilot and feasibility trials being undertaken in the United Kingdom registered in the United Kingdom Clinical Research Network database.
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DOI：
10.1186/1471-2288-13-104
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发表时间：
2013-08-20
影响因子：
4
通讯作者：
Julious SA
中科院分区：
文献类型：
--
作者：
Billingham SA;Whitehead AL;Julious SA
关于试点或可行性试验所需的样本量，几乎没有出版的指导意见，尽管样本量的合理性是试验设计中的一个关键因素。样本量说明应说明达到试验目标所需的最低参与者人数。本文试图描述目前在英国进行的试点和可行性随机对照试验的目标样本量。数据从英国临床研究网络(UKCRN)数据库中收集，使用搜索词“试验”和“可行性”。通过这次搜索，对513项研究进行了资格评估，其中79项符合纳入标准。在UKCRN数据库的数据摘要不完整的情况下，数据也来自：国际标准化随机对照试验编号(ISRCTN)登记；Clinicaltrials.gov网站和资助者网站。在62项试验中，有必要通过电子邮件联系研究团队成员，以确保完整性。在所分析的79项试验中，50项(63.3%)被标记为试点试验，25项(31.6%)可行性试验，14项同时被描述为试点试验和可行性试验。大多数人有两个手臂(n = 68，86.1%)，最常见的两个终点是连续的(n = 45，57.0%)和二分的(n = 31，39.2%)。研究发现，与可行性试验(中位数 = 36，参与者范围 = 10至300)相比，试点试验每支手臂的样本量(中位数 = 30，参与者范围 = 8至114)要小。根据终点类型，在可行性试验和试点试验中，对于二分终点的试验，每臂样本大小的中位数为36( = 10至300人)，而对于连续终点的试验，每臂的中位数样本量为30( = 8至114人)。公共资助的试点试验似乎比行业资助的试点试验更大：中位数样本量分别为33(Range = 15至114参与者)和25(Range = 8至100参与者)。所有的研究都应该有样本量的合理性。然而，并不是所有的研究都需要计算样本量。对于试点和可行性试验，尽管样本量的合理性很重要，但正式的样本量计算可能并不合适。本文的结果描述了在UKCRN数据库上的可行性和试点随机对照试验中观察到的样本量。
There is little published guidance as to the sample size required for a pilot or feasibility trial despite the fact that a sample size justification is a key element in the design of a trial. A sample size justification should give the minimum number of participants needed in order to meet the objectives of the trial. This paper seeks to describe the target sample sizes set for pilot and feasibility randomised controlled trials, currently running within the United Kingdom. Data were gathered from the United Kingdom Clinical Research Network (UKCRN) database using the search terms ‘pilot’ and ‘feasibility’. From this search 513 studies were assessed for eligibility of which 79 met the inclusion criteria. Where the data summary on the UKCRN Database was incomplete, data were also gathered from: the International Standardised Randomised Controlled Trial Number (ISRCTN) register; the clinicaltrials.gov website and the website of the funders. For 62 of the trials, it was necessary to contact members of the research team by email to ensure completeness. Of the 79 trials analysed, 50 (63.3%) were labelled as pilot trials, 25 (31.6%) feasibility and 14 were described as both pilot and feasibility trials. The majority had two arms (n = 68, 86.1%) and the two most common endpoints were continuous (n = 45, 57.0%) and dichotomous (n = 31, 39.2%). Pilot trials were found to have a smaller sample size per arm (median = 30, range = 8 to 114 participants) than feasibility trials (median = 36, range = 10 to 300 participants). By type of endpoint, across feasibility and pilot trials, the median sample size per arm was 36 (range = 10 to 300 participants) for trials with a dichotomous endpoint and 30 (range = 8 to 114 participants) for trials with a continuous endpoint. Publicly funded pilot trials appear to be larger than industry funded pilot trials: median sample sizes of 33 (range = 15 to 114 participants) and 25 (range = 8 to 100 participants) respectively. All studies should have a sample size justification. Not all studies however need to have a sample size calculation. For pilot and feasibility trials, while a sample size justification is important, a formal sample size calculation may not be appropriate. The results in this paper describe the observed sample sizes in feasibility and pilot randomised controlled trials on the UKCRN Database.
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影响因子：
1.5
作者：
Julious, SA;Patterson, SD
通讯作者：
Patterson, SD
影响因子：
--
作者：
ALTMAN, DG
通讯作者：
ALTMAN, DG
影响因子：
2
作者：
Stallard, Nigel
通讯作者：
Stallard, Nigel
影响因子：
--
作者：
CAMPBELL, MJ;JULIOUS, SA;ALTMAN, DG
通讯作者：
ALTMAN, DG
影响因子：
2.5
作者：
PRESCOTT, PA;SOEKEN, KL
通讯作者：
SOEKEN, KL