用于评估囊性纤维化治疗的成本效益分析方法:一个范围审查
Dominique Seo1, David C Young2, Eberechukwu Onukwugha3
1Department of Practice, Sciences, and Health Outcomes Research, University of Maryland School of Pharmacy, 220 Arch Street, 12th Floor, Room 01-515, Baltimore, MD, 21201, USA. dseo@umaryland.edu.
PharmacoEconomics
|April 29, 2025
概括
对于囊性纤维化 (CF) 治疗的成本效益分析 (CEA) 显示了可变性. 为了更好的CF护理决策,需要标准化方法,并包括更广泛的经济影响.
科学领域:
- 卫生经济学 卫生经济学
- 药物经济学 药物经济学
- 遗传疾病研究 遗传疾病研究
背景情况:
- 囊性纤维化 (CF) 是一种罕见的遗传性疾病,具有显著的终身医疗费用.
- 尽管治疗的进步提高了预期寿命,但CF管理的经济负担仍然很大.
- 成本效益分析 (CEA) 对于评估CF干预和查的经济价值至关重要.
研究的目的:
- 对现有的囊性纤维化 (CF) 治疗的成本效益分析 (CEA) 进行范围审查.
- 确定CF CEAs目前的证据库中的最佳实践和知识差距.
- 在CF研究中为开发更强大,更可比的CEA提供信息.
主要方法:
- 使用PubMed和Embase数据库进行了对文献的系统范围审查.
- 包含的研究是那些特色CEA专注于CF治疗的研究.
- 数据提取的重点是研究特征,模型输入和假设,随后是医疗保健和社会影响的定性综合.
主要成果:
- 包括11项研究,评估支持性疗法 (6项研究) 和CFTR调节剂 (5项研究).
- 在对比剂和药物成本方面观察到显著的异质性,阻碍了跨研究的比较.
- CEA捕捉了疾病进展,生活质量影响和直接的医疗费用,但在经济影响的范围上有所不同.
结论:
- 现有的CF治疗CEA表现出相当大的复杂性和异质性.
- 在CF CEA中,对标准化的方法极其需要.
- 包括更广泛的经济影响在内的全面评估对于CF治疗的知情决策至关重要.
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