对于平均等价性测试的有限样本校正
Younes Boulaguiem1, Julie Quartier2,3, Maria Lapteva2,3
1Geneva School of Economics and Management, University of Geneva, Switzerland.
Statistics in medicine
|December 20, 2023
概括
一种新的统计方法,即TOST,通过纠正两个单面测试 (TOST) 程序来改进平均 (生物) 相当性测试. 这增强了功率,特别是对于高度可变的药物,确保更可靠的等价性结论.
科学领域:
- 生物统计学 生物统计学
- 制药指标 (Pharmacometrics) 是一个指标.
- 统计推理 统计推理
背景情况:
- 平均 (生物) 相当性测试确定一个参数,如平均差异,是否在预定义的等效限内.
- 标准的两侧测试 (TOST) 程序是常用的,但可以保守,导致高可变性的功耗损失.
- 这种保守性可以阻止结论真正的等价性,特别是在药物应用中,药物反应是可变的.
研究的目的:
- 为TOST程序引入一个有限的样本校正,称为TOST.
- 为了保证纠正程序的受控测试大小 (I型错误率) 为.
- 提高同等性测试的效力,特别是在药物变异性较高的场景中.
主要方法:
- 开发了一个有限的样本和对TOST程序的显著性水平的变化性纠正.
- -TOST程序调整TOST以保持特定的I型错误率.
- 对TOST的操作特性和功率与现有方法进行了评估.
主要成果:
- -TOST程序比标准的TOST程序更为强大.
- 与竞争程序相比,拟议的方法表现出优越的操作特性.
- 一个关于生猪皮肤中埃科纳酸盐沉积的案例研究说明了TOST的实际好处和优势.
结论:
- -TOST为平均 (生物) 相当性测试提供了一个统计学上合理和更强大的替代方案.
- 这种更正的程序有效地解决了TOST在高可变性存在时的功率损失问题.
- -TOST在计算上简单,并为结论等价性提供了更高的可靠性.
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