长期临床试验的停止规则是基于两个后续的:拒绝虚假假设的否定
Mohamed Mubasher1, Howard E Rockette2
1Jaeb Center for Health Research.
Communications in statistics: theory and methods
|May 20, 2025
概括
这项研究引入了一种新的方法,用于停止使用时间到事件数据的长期临床试验. 它通过要求多次拒绝来确保试验有效性,为大型研究提供了现实的方法.
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
- 统计方法 统计方法
背景情况:
- 具有时间到事件结果的长期随机临床试验 (RCT) 需要强有力的停止规则.
- 现有的方法可能无法在中间分析期间充分控制I型错误率.
- 没有持续证据的早期终止可能会导致不可靠的结论.
研究的目的:
- 根据连续多次拒绝,制定和评估一个停止长期RCT的战略.
- 为了控制实验的误差率,同时保持统计的完整性.
- 为大型多中心试验提供更现实的和可靠的停止规则.
主要方法:
- 拟议的策略使用的标准要求在指定的alpha级别连续或非连续的多次拒绝.
- 临时测试的α水平是根据预先规定的总体测试大小来确定的.
- 使用多维集成和/或模拟方法计算了边界切断值.
主要成果:
- 该研究介绍了一种使用两个连续排斥的程序,用于中间测试的相应的alpha水平.
- 为了保持实验明智的错误率,所需的临时α水平的降低被发现是适度的.
- 测试的功率根据各种替代的积累和危险模式进行了评估.
结论:
- 开发的停止策略为终止长期临床试验提供了更现实的方法.
- 它确保只有在证明持续效果时才能停止试验,从而提高了可靠性.
- 这种方法对于大型的多中心试验尤其有利,在没有强有力的证据的情况下,早期终止是不可取的.
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