怀疑先验对具有二元结果的适应性临床试验的表现的影响
Anders Granholm1, Theis Lange2, Michael O Harhay3,4
1Department of Intensive Care 4131, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Pharmaceutical statistics
|March 29, 2024
概括
贝叶斯适应性试验中的怀疑先验与二进制结果可以增加样本大小,但改善决策. 中度怀疑在临床试验设计中提供了效率和可靠结果之间的平衡.
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
- 贝叶斯统计学贝叶斯统计学
背景情况:
- 适应性临床试验提供了灵活性,但其表现可能会受到先前的信念的影响.
- 对贝叶斯适应性试验设计与二元结果的怀疑先验的影响仍然不清楚.
研究的目的:
- 评估不同程度的怀疑先验如何影响贝叶斯适应性临床试验设计的表现.
- 评估不同的绩效指标,包括样本大小,事件率和决策概率.
主要方法:
- 模拟的多阶段,适应贝叶斯试验设计与二进制结果.
- 使用固定和校准的停止规则来保持1型错误率.
- 评估指标,如样本大小,事件计数,确定性概率和理想设计百分比 (IDP).
主要成果:
- 随着固定停止规则的增加,人们的怀疑态度导致了更大的样本规模和更多事件,但在优越性结果中,IDP也更高.
- 校准的停止规则减弱的样本大小随着怀疑而增加,在不确定的试验中增强了断定性和IDP.
- 具有温和适应和非信息化的先验的设计反映了具有中度怀疑先验的积极适应.
结论:
- 在具有二元结果的适应性试验设计中,在考虑多个性能指标时,使用有点怀疑的先验似乎是合理的.
- 怀疑主义影响试验的表现,校准的停止规则减轻了潜在的缺点.
- 结果表明,在适应性试验设计中,对预先选择的细微方法.
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