双适应性偏差硬币设计,以改善贝叶斯临床试验与时间到事件终点的贝叶斯临床试验
Wenhao Cao1, Hongjian Zhu2, Li Wang3
1Division of Biostatistics and Health Data Science, University of Minnesota, Minneapolis, Minnesota, USA.
Statistics in medicine
|February 22, 2024
概括
我们介绍了一种新的频率主义自适应随机化方法,与贝叶斯响应自适应随机化 (RAR) 分配比例相匹配. 这种设计提高了临床试验的效率和伦理,同时解决了对贝叶斯式RAR可变性的担忧.
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
- 适应性试验设计的设计
背景情况:
- 临床试验设计需要平衡科学严谨性与效率和患者安全.
- 贝叶斯响应自适应随机化 (RAR) 提供了好处,但对可变性和理论保证存在担忧.
- 现有的方法难以同时优化多个试验目标.
研究的目的:
- 提出一个频率的自适应性随机化设计,解决贝叶斯的RAR限制.
- 为了实现道德的分配比例和提高试验效率.
- 为临床试验提供强大的设计,具有时间到事件终点.
主要方法:
- 开发频率主义的双重适应性偏见硬币设计 (DBCD).
- 从贝叶斯框架中准分配比例.
- 理论性质的推导和全面的数值模拟.
主要成果:
- 拟议的DBCD实现了与贝叶斯式RAR可比的伦理分配比例.
- 设计证明了效率,而不会影响道德考虑.
- 理论和模拟结果支持拟议方法的实际实用性.
结论:
- 在临床试验中,Frequentist DBCD为贝叶斯式RAR提供了一个可行的替代方案.
- 这种设计为实际的适应性随机化提供了坚实的基础.
- 该方法有效地平衡了科学目标与伦理和效率方面的关注.
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