Design of randomized controlled confirmatory trials using historical control data to augment sample size for concurrent controls
Design of randomized controlled confirmatory trials using historical control data to augment sample size for concurrent controls
复制标题
使用历史对照数据设计随机对照验证性试验，以增加并发对照的样本量
DOI：
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发表时间：
2019
影响因子：
1.1
通讯作者：
U. Palm
中科院分区：
文献类型：
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作者：
Jiacheng Yuan;Jeen Liu;Ray Zhu;Ying Lu;U. Palm
摘要本文讨论了在临床研究中利用现有历史数据增加随机对照试验（CC）的方法。在他们的文章《与多个对照组匹配并调整组差异》中，Stuart和Rubin提出了一种匹配方法，其中主要/局部对照和次要/非局部对照都包含在倾向评分估计中。作者讨论了一种类似的方法，将CC作为主要研究对象，将历史对照作为次要研究对象，发现这种方法与不补充历史数据的传统随机设计相比，并没有节省随机试验的样本量。提出了一种新的匹配方法，节省样本量，其中倾向评分估计没有并行随机对照患者。提出了一个两阶段的设计，它允许一个检查的假设，新的匹配方法之前的承诺，在第二阶段使用的匹配方法。最后以临床试验数据为例说明了所提方法的可行性。仿真研究已被用来调查所提出的方法的操作特性。
ABSTRACT This paper deals with the methods to augment concurrent controls (CC) in a randomized controlled trial with available historical data in clinical studies. In their article, Matching with multiple control groups and adjusting for group differences, Stuart and Rubin proposed a matching method where the primary/local control and the secondary/non-local control are both included in the propensity score estimates. The authors discuss a similar approach taking the CC as the primary and the historical control as the secondary, and find that this approach does not save the sample size of the randomized trial compared to the traditional randomized design without supplementation of historical data. A new matching method that saves sample size is proposed, where propensity scores are estimated without the concurrent randomized control patients. A two-stage design is proposed, which allows one to examine the assumption of the new matching method before a commitment of using the matching method in the second stage. Previous clinical trials data is used as an example to illustrate the feasibility of the proposed methods. Simulation studies have been used to investigate operating characteristics of the proposed method.
影响因子：
5
作者：
Brookhart, M. Alan;Schneeweiss, Sebastian;Sturmer, Til
通讯作者：
Sturmer, Til
影响因子：
2
作者：
Normand, SLT
通讯作者：
Normand, SLT