运输随机试验结果以估计目标人群中的反事实生存功能
Zhiqiang Cao1, Youngjoo Cho2, Fan Li3,4
1Department of Mathematics, College of Big Data and Internet, Shenzhen Technology University, Shenzhen, People's Republic of China.
Pharmaceutical statistics
|January 17, 2024
概括
本研究介绍了将临床试验结果传输到目标人群的方法,即使具有不同的特征. 它解决了使用复杂的调查数据在新人群中估计治疗效果的挑战.
科学领域:
- 生物统计学 生物统计学
- 流行病学 流行病学
- 临床试验 临床试验
背景情况:
- 随机试验可能与目标人群有不同的效果修饰剂分布,导致偏差的治疗效果估计.
- 试验结果的可转移性对于概括性至关重要,特别是对于有审查的时间到事件结果.
- 现有的可运输性方法仅限于对目标人群的反事实生存功能的估计.
研究的目的:
- 开发和评估运输临床试验结果的方法,以估计目标人群中的反事实生存功能.
- 在存在不同效果修饰剂分布的情况下,估计治疗效应的目标平均存活率.
- 将复杂的调查数据权重纳入可运输性估计器中.
主要方法:
- 使用逆概率权重和双倍强大的估计器来估计生存功能.
- 为拟议的方法开发了近似差异估计器.
- 在目标人群中包含复杂调查数据的纳入调查权重.
主要成果:
- 模拟研究表明,在各种模型规范下,拟议的估计器的有限样本性能 (偏差,效率,覆盖率).
- 这些方法用于评估"控制糖尿病和血压中的心血管风险行动" (ACCORD-BP) 试验结果的可转移性.
- 评估了效果修饰剂的差异分布对治疗效果估计的影响.
结论:
- 拟议的反向概率权重和双重可靠的估计器提供了有效的方法,用于将时间到事件的试验数据传输到目标人群.
- 这些方法有效地适应不同效果变量分布,并结合复杂的调查数据.
- 在评估心血管风险降低试验结果的概括性方面证明了实际应用.
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