评估Fill-it-up设计,以使用随机临床试验中的历史对照数据,与两个手臂平行组设计
Stephanie Wied1, Martin Posch2, Ralf-Dieter Hilgers3
1Institute of Medical Statistics, RWTH Aachen University, Aachen, Germany. swied@ukaachen.de.
BMC medical research methodology
|September 9, 2024
概括
填充设计是一种新的两阶段临床试验方法,将历史对照与随机对照试验 (RCT) 整合在一起. 这种方法可以提高数据利用率,同时管理偏差风险,为复杂的研究场景提供灵活的解决方案.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 流行病学 流行病学
背景情况:
- 越来越多的人对创新的临床研究设计的需求,以利用现有数据.
- 历史控制提供了有价值的信息,但引入了潜在的偏见.
- 需要将历史数据整合到新研究设计中的方法.
研究的目的:
- 介绍和评估Fill-it-up设计,这是一个两阶段的方法,结合了历史和随机控制.
- 评估该设计在I型错误率和统计功率方面的性能.
- 为在临床研究中利用历史数据提供框架.
主要方法:
- 填充式设计涉及初始等价性预测,以比较历史和随机对照.
- 如果确立了等价性,则将历史数据纳入;否则,将随机化扩展.
- 该研究调查了这种两阶段设计的样本大小要求和错误率.
主要成果:
- 填充式设计保持了5%的家庭智能错误率.
- 最大样本大小可以超过没有历史控制的单阶段设计.
- 对照组之间的异质性增加提高了最大样本大小.
结论:
- 填充设计是一种用于在临床试验中结合历史对照的频率主义方法.
- 由于可能更大的样本大小,需要强有力的先验信念.
- 在特殊情况下,作为混合设计的有价值的选择.
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