评论:哪些原则应适用于国家罕见病战略?
1Associate Professor, School of Public Policy and Administration, Carleton University, Ottawa, ON.
Healthcare policy = Politiques de sante
|September 4, 2024
概括
联邦为罕见病药物提供资金应该支持发展,而不仅仅是购买. 基于结果的风险分担协议需要强大的机构能力来收集真实世界的证据.
科学领域:
- 卫生经济学 卫生经济学
- 制药政策 制药政策
- 罕见疾病研究研究.
背景情况:
- 莱克辛和塞尔斯 (2024) 提出了联邦政府为昂贵的罕见病药物提供资金的原则.
- 这些原则包括基于结果的风险分担协议 (OBRSA) 和证据生成.
研究的目的:
- 质疑为罕见病药物提供联邦资金的范围.
- 探索资金是否应该扩展到药物开发,而不仅仅是采购.
- 检查对OBRSAs的机构要求,以促进现实世界的证据收集.
主要方法:
- 重新加入现有原则的分析.
- 检查药品的融资机制.
- 评估机构的证据生成能力需求.
主要成果:
- 联邦对罕见病药物的资助可能会受到限制,如果仅限于购买新药.
- 资金的更广泛应用可能包括药物开发,促进创新.
- 成功实施OBRSAs需要大量的机构能力来收集真实世界的数据.
结论:
- 对罕见病药物的联邦资助政策应该考虑支持整个药物生命周期,包括开发.
- 基于结果的风险共享协议需要对必要的基础设施进行仔细规划,以便在现实世界中收集证据.
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