用二元终点进行双臂优势和非劣势试验的最佳样本大小分配
Marietta Kirchner1, Stefanie Schüpke2,3,4, Meinhard Kieser1
1Institute of Medical Biometry, University of Heidelberg, Heidelberg, Germany.
Pharmaceutical statistics
|March 12, 2024
概括
优化临床试验样本大小分配可以减少所需的参与者总数. 虽然相同的组大小是常见的,但在具有二进制终点的优越性和非劣势性试验中,不平等的分配可能更有效.
科学领域:
- 临床试验 临床试验
- 生物统计学 生物统计学
- 医学研究 医学研究
背景情况:
- 临床试验样本大小对于研究能力和资源管理至关重要.
- 组间样本大小的分配会影响整体试验大小.
- 由于道德和经济方面的考虑,必须避免不必要的大样本.
研究的目的:
- 用二进制终点确定双臂优势和非劣势试验的最佳样本大小分配比.
- 为了尽量减少所需的总样本大小,同时保持其他参数不变.
- 为了比较最佳分配与平等分配的效率.
主要方法:
- 在各种场景中进行了广泛的计算.
- 重点是使用二进制终点的双臂试验.
- 该研究分析了优越性和非劣势性试验设计.
主要成果:
- 在优势和非劣势试验中,最佳样本大小分配比率可能与平等分配有很大差异.
- 对二进制终点的平等分配的偏差被探索.
- 针对各种试验参数和场景进行了计算.
结论:
- 对于二元终点试验,最佳的样本大小分配可能并不总是相同的组大小.
- 虽然最佳分配可以减少样本大小,但与平等分配相比,节省的成本通常很小.
- 这些发现为有效的临床试验设计提供了指导.
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