通过对同一个对照进行测试,使多种实验治疗的比较Poisson临床试验经济化
Joseph A Chiarappa1, Donald R Hoover2, Alison Pedley1
1Merck and Co. Inc., North Wales, PA, USA.
Statistical methods in medical research
|June 30, 2023
概括
设计C试验比较多种实验性治疗与单个对照组,通常需要的参与者比进行多个单独的试验 (设计A) 较少. 这种方法在临床研究中可以显著节省样本大小.
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
- 实验设计 实验设计
背景情况:
- 比较的Poisson试验 (设计A) 通常依赖于总事件的条件,用于使用二项式分布推断.
- 最近引入了设计C,用于将K个实验治疗与单个对照进行比较,使用负的多项式分布进行推断.
研究的目的:
- 为了比较设计C与K单独的设计A试验的样本大小效率.
- 在不受限制和限制的设置下评估设计.
主要方法:
- 模拟了1型错误,功率和发生率比率的各种组合.
- 根据不同的假设和假设,对C设计和A设计注册的预期受试者数量进行比较.
主要成果:
- 与设计A相比,设计C经常显示出显著的样本大小节省.
- 在未限制和限制的试验设置中都观察到节省.
结论:
- 进行单个设计C试验通常比多个设计A试验更有效.
- 设计C代表了与多个实验臂进行比较临床试验的潜在优势策略.
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