适应性试验设计的信心区间I:方法论审查
David S Robertson1, Thomas Burnett2, Babak Choodari-Oskooei3
1MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
Statistics in medicine
|August 8, 2025
概括
在适应性临床试验中解释置信区间 (CI) 需要谨慎. 本综述探讨了在适应性设计 (ADs) 中构建准确CI的方法,以确保可靠的治疗效果估计.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 监管科学 监管科学
背景情况:
- 在适应性临床试验 (ADs) 中的置信区间 (CI) 往往表现为覆盖不足.
- 传统的CI方法无法考虑试验适应,导致对治疗效果的潜在误解.
- 监管指南强调需要在ADS中仔细解释CI.
研究的目的:
- 在ADs中构建CI的方法提供了全面的审查.
- 根据AD类型对可用的CI方法进行分类.
- 通过交通信号灯系统评估这些CI方法的理想特性.
主要方法:
- 对ADS的CI构造方法的系统文献综述.
- 根据AD类型对方法的分类.
- 评估CI属性,包括名义覆盖范围和与假设测试的一致性.
主要成果:
- 识别和描述了几类用于在AD中构建CI的技术.
- 一个系统的文献综述对现有方法进行了分类.
- 用一个评估框架 (交通信号灯系统) 来评估方法的性能.
结论:
- 准确的CI对于可靠地解释ADS治疗效应至关重要.
- 对于试验统计学家来说,了解不同CI方法的特性是必不可少的.
- 这一审查为适应性试验中的CI的实际实施和指导方针开发提供了基础.
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