估计异质治疗效应与现实世界健康数据 - - 机器学习方法的范围审查
Michael Möller1, Eva-Maria Wild2, Winnie Tan1
1Hamburg Center for Health Economics, University of Hamburg, Esplanade 36, 20354 Hamburg, Germany.
概括
机器学习 (ML) 方法使用现实世界数据 (RWD) 估计异质治疗效应 (HTEs). 定制条件平均治疗效应 (CATE) 方法正在增长,但医疗经济学研究的方法质量和报告需要改进.
科学领域:
- 卫生经济学 卫生经济学
- 生物统计学 生物统计学
- 机器学习 机器学习
背景情况:
- 异质治疗效应 (HTEs) 描述了治疗反应的个体变化.
- 估计高高温体验可以优化患者护理和资源配置.
- 机器学习 (ML) 在随机对照试验不切实际的情况下提供工具来使用现实世界数据 (RWD) 估计HTEs.
研究的目的:
- 在RWD中对HTE估计的ML方法进行分类.
- 评估研究的方法质量,这些研究应用了HTE的ML.
- 为健康经济学研究提供指导.
主要方法:
- 根据PRISMA-ScR指南进行了范围审查.
- 搜索了主要的数据库 (PubMed,Scopus,科学网,EBSCO,MEDLINE) 来查找2014-2025年间的研究.
- 使用标准化检查清单评估方法质量.
主要成果:
- 74项研究符合纳入标准,分为仅预测 (n=8),结果建模 (n=9) 和定制条件平均治疗效果 (CATE) 估计 (n=58) 的研究.
- 大多数高耐久性瘤的ML创新源自卫生经济学之外.
- 方法质量不一致,医疗经济学中先进方法的采用有限.
结论:
- ML方法越来越多地用于RWD的HTE估计,以树为基础的模型和CATE方法获得引力.
- 改进的评估标准和透明的报告对于健康经济学的可靠应用至关重要.
- 需要进一步的研究来弥合ML进步和健康经济学实践之间的差距.
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