样本大小重新估计在临床试验中的应用:系统性审查
Hirotaka Mano1, Yuji Tanaka1, Shunichiro Orihara1
1Biostatistics Group, Biometrics Department, Development Unit, R&D Division, Kyowa Kirin Co., Ltd., Otemachi Financial City Grand Cube, 1-9-2 Otemachi, Chiyoda-ku, Tokyo, Japan.
Contemporary clinical trials communications
|October 16, 2023
概括
样本大小重新估计 (SSR) 在研究中期重新计算试验样本大小. 这一系统性审查发现,SSR在早期临床试验中越来越多地使用,这表明其未来的广泛应用.
科学领域:
- 临床试验设计和方法论
- 生物统计学 生物统计学
- 药学研究 药学研究
背景情况:
- 样本大小重新估计 (SSR) 是在临床试验期间,当初始信息不足时调整样本大小的关键方法.
- 虽然SSR影响研究规模,持续时间,资源配置和总体成本,但其应用情况尚未得到充分记录.
- 进行了系统的文献审查,以阐明SSR在临床研究中的使用模式和背景.
研究的目的:
- 系统地审查和总结在临床试验中应用样本大小重新估计 (SSR) 的条件和情况.
- 了解SSR在不同临床试验阶段和治疗领域的当前应用环境.
主要方法:
- 使用PubMed作为主要数据库进行了系统的文献搜索,并补充了ClinicalTrials.gov.gov.
- 搜索策略的重点是确定采用样本大小重新估计 (SSR) 的临床试验.
- 提取和总结了确定试验的关键特征,包括研究阶段和开始年份.
主要成果:
- 总共有253篇出版物被选,导致识别了27项使用SSR的临床试验.
- 对研究阶段的分析显示,SSR在第一阶段 (7.4%),第二阶段 (18.5%),第三阶段 (40.7%) 和第四阶段 (7.4%) 试验中的应用.
- 在早期阶段 (I和II) 观察到SSR,这表明它在关键的早期决策过程中被使用.
结论:
- 样本大小重新估计 (SSR) 在早期临床试验 (I期和II期) 中使用,在先前数据有限时支持知情决策.
- 对于特定的治疗领域或使用的SSR类型,没有发现显著的趋势,这表明其广泛适用.
- 这些发现表明,SSR是一种可行且可能广泛适用于未来临床试验设计的方法.
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