药品政策和罕见疾病的创新:一个叙事审查
Adrián Alonso Ruiz1, Kaitlin Large1, Suerie Moon1
1Global Health Centre, Graduate Institute of International and Development Studies, Geneva, 1211, Switzerland.
F1000Research
|May 23, 2024
概括
鼓励孤儿药物的公共政策增加了罕见病治疗,但也引起了人们对获取和负担能力的担忧. 建议进行改革,以应对这些挑战并改善全球影响.
科学领域:
- 卫生政策 卫生政策
- 制药经济学 制药经济学
- 罕见疾病研究研究 罕见疾病研究
背景情况:
- 罕见疾病,或孤儿疾病,缺乏商业利益,阻碍治疗开发.
- 公共政策旨在激励孤儿药物的开发.
- 美国的孤儿药物法案 (ODA) 作为全球政策的典范.
研究的目的:
- 综合有关鼓励孤儿药物开发的公共政策的文献.
- 评估这些政策在美国,欧盟和日本的影响.
- 确定挑战,并为孤儿药物激励措施提出改革建议.
主要方法:
- 专注于美国孤儿药物法案的文学综合.
- 术语"罕见疾病"的历史概述.
- 分析政策影响,包括积极和负面影响.
主要成果:
- 与增加孤儿药物开发相关的激励措施,并影响了制药研发.
- 政策显示对可负担性和获得孤儿药物的负面影响.
- 在罕见疾病领域的研究优先级中观察到差异.
结论:
- 孤儿药物模式推动了进展,但需要改革以确保公平的获取和负担能力.
- 日本和欧盟的国际政策有相似之处和挑战.
- 解决对低收入和中等收入国家 (LMICs) 的影响对于全球健康公平至关重要.
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