对于时间到事件结果的回归校准:减轻因现实世界终点测量错误而导致的偏差
Benjamin Ackerman1, Ryan W Gan1, Youyi Zhang1
1Johnson & Johnson, Raritan, NJ, USA.
Epidemiologic methods
|September 29, 2025
概括
生存回归校准 (SRC) 是一种通过纠正测量错误来减少现实数据 (RWD) 偏差的新方法. 这在结合临床试验和RWD时提高了治疗疗效证据的可靠性.
科学领域:
- 生物统计学 生物统计学
- 现实世界的证据.
- 药物开发 药物开发
背景情况:
- 现实世界数据 (RWD) 越来越多地与临床试验数据一起用于评估治疗疗效.
- 在试验和现实环境之间疾病评估的差异引入了测量错误和偏差.
- 这种偏见使患者结果的比较变得复杂,并阻碍了强有力的证据生成.
研究的目的:
- 开发一种新的统计方法,生存回归校准 (SRC),以减轻时间到事件RWD结果的测量误差偏差.
- 在结合临床试验数据与瘤学中的RWD时改进推断.
- 提高现实世界和试验终点的可比性.
主要方法:
- 通过解决时间到事件RWD结果的测量误差,SRC扩展了现有的回归校准.
- 它涉及将韦布尔回归模型与验证样本中的试验样 ("真实") 和现实世界样 ("错误测量") 结果措施相匹配.
- 然后,参数估计在完整的研究中根据韦布尔参数中估计的偏差进行校准.
主要成果:
- SRC有效地解释了现实世界无进展生存期 (PFS) 测量错误引起的偏差.
- 该方法表明,与标准回归校准技术相比,测量误差偏差的减少更大.
- 对于时间到事件结果的SRC的适用性提高了其性能.
结论:
- 在整合临床试验和RWD时,解决结果测量错误至关重要,以防止有偏见的结果.
- SRC 方法有效地减轻了偏差,提高了现实世界和试验终点的可比性.
- 这使得在药物开发中治疗疗效的证据更为有力.
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