孤儿医疗器械:解决欧盟和美国的监管和获取差距
Sanae Akodad1, Baptiste Haon2,3, Lise Rochaix2
1Institute for Interdisciplinary Innovation in Healthcare (I3h), Faculté de Médecine, Solvay Brussels School of Economics and Management, Université Libre de Bruxelles (ULB), Brussels, Belgium.
Frontiers in public health
|January 30, 2026
概括
孤儿医疗器械在欧盟和美国面临政策差距. 需要进行改革,以确保公平地获得这些对罕见疾病至关重要的技术.
科学领域:
- 医疗器械监管法规 医疗器械监管法规
- 罕见病政策 罕见病政策
- 卫生经济学 卫生经济学
背景情况:
- 孤儿医疗器械是一个政策盲点,特别是在欧盟.
- 自1990年以来,美国有人道主义使用设备 (HUD) 和人道主义设备豁免 (HDE) 框架.
- 欧盟在2024年引入了一个不具约束力的定义,强调了监管不对称性.
研究的目的:
- 比较欧盟和美国的孤儿医疗器械框架.
- 分析证据标准,获取途径,经济激励和市场后调整.
- 评估患者获得和护理连续性的现实世界后果.
主要方法:
- 基于文件的政策分析 (1990-2025年).
- 审查立法,监管指导和学术文献.
- 案例研究:柏林心脏EXCOR,Argus II,Medtronic的旋律门. 这是一个很好的例子.
主要成果:
- 欧盟法规缺乏孤儿设备的指定和协调的HTA;访问是分散的.
- 美国HUD/HDE途径提供灵活性,但面临有限的财务可持续性.
- 案例研究显示了不同的访问,过渡潜力和中小企业的脆弱性.
结论:
- 政策改革是必不可少的,反映了"孤儿药物法案".
- 拟议的支柱:法律指定,适应性证据,可持续激励,综合性报销和连续性计划.
- 提出了可行的改革,以弥合监管差距,防止市场放弃.
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