一种基于标准的方法,用于对临床试验生存数据进行系统和透明的推断
Gabriel Tremblay1, Patrick Haines2, Andrew Briggs3
1Eisai Co. Ltd., Woodcliff Lake, NJ, USA.
Journal of health economics and outcomes research
|September 4, 2023
概括
推断临床试验生存数据对于健康技术评估评估至关重要. 使用参数模型的系统方法,如加速失效时间模型,可以确保治疗方法的准确和透明的生存估计.
科学领域:
- 卫生经济学 卫生经济学
- 生物统计学 生物统计学
- 在瘤学瘤学.
背景情况:
- 临床试验数据往往缺乏足够的时间持续时间,以达到时间到事件的终点.
- 卫生技术评估 (HTA) 机构需要总生存率 (OS) 和无进展生存率 (PFS) 的估计.
- 试验结果的推断对于健康经济和HTA评估是必要的,因为在试验期之外观察到的生存效应.
研究的目的:
- 探索用于推断临床试验数据的技术.
- 建立选择适当外推方法的标准.
- 开发一种系统的方法来证明生存估计的合理性,适用于转移性乳腺癌 (MBC).
主要方法:
- 设计了一个基于标准的准则指南,用于准确的生存率估计外推和证明.
- 应用了参数和零碎模型来进行生存推断.
- 利用统计和视觉测试来确定最合适的建模技术.
主要成果:
- 加速失效时间 (AFT) 参数模型与马分布和治疗共变量是MBC病例研究中OS的最佳结果.
- 在MBC病例研究中,Kaplan-Meier生存估计用于PFS.
- 该研究确定了不同生存终点的特定建模技术.
结论:
- 估计的生存时间必须被推算出来,以清楚地证明治疗效益.
- 一种系统的方法和正式的决策结构将偏见降到最低,并提高生存推断的透明度.
- 这些发现为HTA中可靠的生存估计提供了一个框架.
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