The use of a registry database in clinical trial design: Assessing the influence of entry criteria on statistical power and number of eligible patients
The use of a registry database in clinical trial design: Assessing the influence of entry criteria on statistical power and number of eligible patients
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DOI：
10.1016/j.ijmedinf.2006.05.036
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发表时间：
2007-02-01
影响因子：
4.9
通讯作者：
de Keizer, Nicolette F.
中科院分区：
文献类型：
--
作者：
Peelen, Linda;Peek, Niels;de Keizer, Nicolette F.
随机临床试验（RCT）是用于调查特定医疗干预效果的前瞻性实证研究。临床试验的设计是一个微妙的决策过程，因为在这个过程中做出的每一个决定都会影响临床试验的最终结果。尽管在试验设计中付出了努力，但许多试验未能显示干预的效果。在某些情况下，干预措施可能确实无效，然而，更多情况下，这是由于纳入患者的问题以及由此导致的缺乏显示效果的统计能力造成的。为了避免这个问题，在试验设计中，计算当前设计选择可以实现的统计功效，并与经济考虑进行平衡。然而，在入选标准的选择中，设计过程中的一个重要步骤，所选标准对统计功效和合格患者数量的影响未量化。由于这些标准影响研究人群的特征和符合试验条件的患者数量，从而影响发现干预效果的机会，我们认为在选择入选标准时也应明确估计符合条件的患者数量。使用登记数据库客观估计统计功效和合格患者数量。此外，我们描述了如何将这种方法纳入临床试验的入选标准的选择过程中。我们用一个严重脓毒症的例子来说明这种方法。(c)2006爱思唯尔爱尔兰有限公司保留所有权利。
Randomized clinical trials (RCTs) are prospective empirical studies used to investigate the effect of a particular medical intervention. The design of a clinical trial is a delicate decision process, as each of the decisions that are taken in this process influences the eventual result of the clinical trial. Despite the efforts that are put into trial design, many trials fail to show an effect of the intervention. In some of these situations the intervention may be truly ineffective, however, more often this is caused by problems with the inclusion of patients and a resulting lack of statistical power to show the effect. To avoid this problem, in the design of a trial, the statistical power that can be achieved with the current design choices is calculated and balanced with economic considerations. In the choice of the entry criteria however, an important step in the design process, the influence of the chosen criteria on statistical power and number of eligible patients is not quantified. As these criteria influence the characteristics of the study population and the number of patients that will be eligible for the trial, and thereby the chances of finding an effect of the intervention, we believe that also in the choice of entry criteria explicit estimates of the number of eligible patients should be made.This paper presents a method to arrive at precise, objective estimates of statistical power and the number of eligible patients, using a registry database. Furthermore, we describe how this method is incorporated in the process of choosing entry criteria for a clinical trial. We illustrate the method with an example in the area of severe sepsis. (c) 2006 Elsevier Ireland Ltd. All rights reserved.