从社会角度对血友病的基于模型的经济分析:一个范围审查
Amr A El-Sayed1,2, Nancy S Bolous3
1Public Health Institute, Faculty of Health, Liverpool John Moores University, Liverpool, UK. dr.amr.elsayed.1983@gmail.com.
PharmacoEconomics - open
|November 21, 2025
概括
血友病 (一种罕见的出血障碍) 的经济评估往往缺乏标准化的报告. 本次审查强调了需要一个框架来提高社会成本分析的一致性和透明度.
科学领域:
- 卫生经济学 卫生经济学
- 药物经济学 药物经济学
- 罕见疾病 罕见疾病
背景情况:
- 血友病对人性和经济造成了长期的重大负担.
- 社会视角的经济评估对于全面的成本捕获至关重要.
- 由于数据的限制和稀有性,对血友病的建模至关重要.
研究的目的:
- 从社会角度确定和总结基于血友病的基于模型的经济研究.
- 在这些研究中评估模型特征和成本类别的报告质量.
主要方法:
- 在八个数据库,灰色文献和手动搜索中进行系统的文献搜索,直到2024年1月5日.
- 包括英语全文经济研究,使用社会视角的建模.
- 将报告成本调整为2022年国际美元 (I$) 和美国美元 (US$).
主要成果:
- 来自20个国家的26项研究符合标准,17项是完整的经济评估.
- 大多数研究集中在血友病A;报告模型特征和输入参数经常不完整.
- 预防,埃米齐祖马布和基因疗法通常表现出成本效益高于其他替代方案.
结论:
- 在血友病经济研究中,报告模型特征和输入参数存在重大差距.
- 遗漏和错误标记成本类别被注意到.
- 为了一致性和透明度,迫切需要对社会成本类别和可访问数据集的标准化框架.
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