整合试验和非实验数据以检查治疗效果异质性的方法
Carly Lupton Brantner1, Ting-Hsuan Chang2, Trang Quynh Nguyen3
1Department of Biostatistics, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland 21205, USA.
概括
在研究中估计治疗效果异质性需要仔细考虑混和数据可用性. 本综述比较了结合数据以定制治疗的方法,突出了未来研究的领域.
科学领域:
- 生物统计学 生物统计学
- 流行病学 流行病学
- 医疗保健服务研究 医疗服务研究
背景情况:
- 个性化医学需要根据观察到的特征,根据个体量身定制的治疗效果进行估计.
- 混和充分的数据是估计治疗效果适度的关键挑战.
- 最近的进展侧重于使用多个随机对照试验和观察数据集来估计治疗效果异质性.
研究的目的:
- 通过综合多项研究的数据,审查和比较估计治疗效果异质性的方法.
- 为了确定不同数据设置的最佳方法,并突出未来的研究方向.
主要方法:
- 根据数据设置对方法的分类:聚合级数据,联合学习和个人参与者级数据.
- 条件平均治疗效果的定义和参数与非参数估计器的讨论.
- 单个研究和数据组合的关键假设列表.
主要成果:
- 通过结合数据集来估计治疗效果异质性的方法有多种.
- 介绍了现有方法的比较和对比.
- 确定了未来研究的开放领域.
结论:
- 通过案例研究和模拟来比较方法还有很大的工作要做.
- 需要进一步的研究,以将方法扩展到不同的环境中,并将其完善以应对现实世界的数据挑战.
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