适应性平台试验:共同控制对第一类错误和功率的影响
Quynh Nguyen1,2, Katharina Hees1, Benjamin Hofner1,2
1Section Data Science and Methods, Paul-Ehrlich Institut, Langen, Germany.
Journal of biopharmaceutical statistics
|November 22, 2023
概括
使用共同控制的平台试验可以提高效率,但需要仔细考虑多重性调整. 具有共同控制的灵活平台试验提供了好处,尽管在某些情况下,单独的试验可能更好.
科学领域:
- 临床试验方法论 临床试验方法论
- 药物开发中的统计分析.
背景情况:
- 平台试验允许使用共同的控制来有效地研究多种干预措施.
- 讨论了平台试验中共同控制的多重性调整的必要性.
- 之前的研究集中在固定平台试验上;灵活的设计需要进一步调查.
研究的目的:
- 在灵活平台试验中调查共同控制对I型错误和功率的影响.
- 为了比较共同对比单个控制的效率增长.
- 评估多重性调整对固定和灵活平台试验中的功率的影响.
主要方法:
- 测试统计数据的推导相关性,以评估对照重叠的影响.
- 比较了平台试验的结果,将常见的控制对象与单独的控制对象进行比较.
- 评估统计能力和多重性调整的影响.
主要成果:
- 与多臂试验中的单独对照相比,共同对照减少了I型错误膨胀.
- 具有共同控制的平台试验通常在样本大小和功率方面具有优势,即使有多重性调整.
- 在特定情况下,共同的控制可能会否定效率的提高,这表明单独的试验可能更好.
结论:
- 具有共同控制的灵活平台试验可以有效,但需要仔细的统计规划.
- 多重性调整至关重要,但可能会影响功率.
- 在平台试验中使用共同控制的决定应基于对潜在的好处和缺点的个案评估.
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