识别不那么繁重和更具成本效益的不完整的分阶形设计,以获得通过重复的横截面收集的连续结果
Ehsan Rezaei-Darzi1, Jessica Kasza1, Anisa R Assifi2
1School of Public Health and Preventive Medicine, Monash University, Melbourne, Australia.
Statistics in medicine
|April 25, 2025
概括
阶段试验可能很昂贵. 这项研究引入了一种成本效益指标,以寻找不那么繁,不完整的阶梯设计,以保持临床试验的高统计能力.
科学领域:
- 临床试验方法论 临床试验方法论
- 卫生经济学 卫生经济学
- 生物统计学 生物统计学
背景情况:
- 阶段形试验是有效的,但往往是昂贵和繁的.
- 减少负担的现有方法包括去除集群周期细胞,创建不完整的设计.
- 需要方法来优化不完整的设计,无论是成本还是统计能力.
研究的目的:
- 提出一种基于成本效益的新方法来探索不完整的阶梯设计.
- 为了确定平衡高统计能力与降低总试验成本的设计.
- 定义和最大化一个成本效益指标的阶段形试验设计.
主要方法:
- 定义成本效益为治疗效果估计器精度与试验总成本的比率.
- 每个集群,每个参与者 (干预/控制) 的内置成本和数据收集重新启动成本.
- 使用基于对成本效益的贡献的代细胞去除程序.
- 确定了最大限度地提高成本效益的最佳设计,并遵守最低功率限制.
- 考虑连续结果的线性混合模型,使用重复的横截面采样.
主要成果:
- 拟议的方法允许识别具有成本效益的不完整的阶梯形设计.
- 不完整的设计可以比完整的设计更轻松,更具成本效益.
- "楼梯"设计,集群只在处理开关周围贡献数据,成为特别具有成本效益的变体.
结论:
- 开发的方法使试验者能够选择分阶形设计,这些设计既不那么繁,又更具成本效益.
- 成本效率分析对于优化阶段形试验设计至关重要.
- 特定的不完整设计,如楼梯变体,在成本和效率方面提供了显著的优势.
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