从社会角度看血友病的经济负担: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.
PharmacoEconomics - open
|November 15, 2024
概括
血友病的经济负担很大,凝血因子替代疗法是最大的成本. 直接的非医疗和间接成本往往被低估,但对于获得治疗至关重要.
科学领域:
- 医学经济学 医学经济学
- 血液学 血液学 血液学
- 公共卫生 公共卫生
背景情况:
- 血友病是一种罕见的遗传出血疾病,具有显著的肌肉骨并发症和治疗成本.
- 准确的经济负担评估至关重要,但因难以量化非医疗,间接和无形成本而受到挑战.
研究的目的:
- 从社会角度对血友病的经济研究进行范围审查.
- 总结血友病护理现有经济分析的方法和发现.
主要方法:
- 在2023年7月之前,对8个数据库和灰色文献进行系统搜索.
- 包括从社会角度看先天性血友病的全文,英语经济分析.
- 排除基于模型的研究和从付款人的角度来看的研究;成本标准化为2022 I$和US$.
主要成果:
- 包括25项研究,涉及22个国家的7226名个人.
- 在所有研究中都报告了直接的医疗费用;血液静止治疗费用差异很大 (每年1,566 I $ 700,070 I $).
- 在某些情况下,凝血因子替代疗法 (CFRT) 占总成本的高达99.9%;非医疗和间接成本往往被低估.
结论:
- 在对血友病的社会经济分析定义成本类别方面存在显著的异质性.
- 虽然血液静止疗法是主要的成本驱动因素,但直接的非医疗和间接成本显著影响治疗机会.
- 标准化的成本框架对于未来的血友病经济研究至关重要,特别是新兴的新疗法.
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