临床试验的贝叶斯式设计使用尺度变换功率先前的尺度
Xinxin Chen1, Brady Nifong2, Ethan M Alt1
1Department of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
本研究引入了一种新的贝叶斯方法来确定样本大小 (SSD),使用具有不同结果的历史数据. 部分借款规模转换前功率 (straPP) 框架使得当历史和新研究结果分歧时,更准确的试验设计成为可能.
科学领域:
- 生物统计学 生物统计学
- 临床试验设计 临床试验设计
- 统计学方法论 统计学方法论
背景情况:
- 现有的贝叶斯样本大小确定方法 (SSD) 主要支持具有与新研究相同结果的历史数据.
- 当初始结果与新临床试验不同时,在整合历史数据的方法中存在差距.
- 这种限制阻碍了有效的试验设计和资源分配.
研究的目的:
- 为临床试验提出一种新的基于模拟的贝叶斯式SSD框架.
- 开发和验证部分借款规模转换前功率 (straPP) 方法.
- 为了使SSD中具有不同主要结果的历史数据能够有效地使用.
主要方法:
- 开发了一个基于模拟的贝叶斯式SSD框架.
- 引入了部分借款规模的变换功率先验 (straPP).
- 对传统的功率先值应用了一种新型的尺度转换,假设相同的标准化参数 (参数 * sqrt ((Fisher信息矩阵)).
主要成果:
- 在straPP方法允许结合历史数据,即使主要结果在研究之间有所不同.
- 模拟研究表明了straPP框架的实用性.
- 该方法使用先前完成的临床试验来说明该方法,以设计具有时间到事件终点的新试验.
结论:
- 建议使用部分借款straPP的贝叶斯式SSD框架有效地解决了历史研究和新研究之间的不同结果的挑战.
- 这种方法为优化临床试验设计和样本大小提供了有价值的工具.
- 它有助于在统计规划中更强大,更有效地利用事先信息.
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