证券化作为支付孤儿疾病细胞和基因疗法的手段:一项模拟研究
John M Lu1, Avi J Cherla1, Alexander W Carter1
1LSE Health, Department of Health Policy, London School of Economics, London, UK.
Gene therapy
|March 12, 2026
概括
通过预付付款为细胞和基因疗法提供资金是很困难的. 一项新提案使用基于30年业绩的年金来使支付与临床效益保持一致,提高了所有利益相关者的负担能力和价值.
科学领域:
- 生物医学科学 生物医学科学
- 卫生经济学 卫生经济学
- 卫生政策 卫生政策
背景情况:
- 细胞和基因疗法具有延长寿命的潜力,但由于长期有效性和成本不确定,因此面临支付挑战.
- 目前基于5年结果的分期付款计划不足以解决长期的成本效益问题.
研究的目的:
- 为细胞和基因疗法提出一个新的融资机制,以解决长期价值和可负担性.
- 为了使支付结构与长期临床效益的积累保持一致.
主要方法:
- 开发基于30年业绩的年金模式.
- 证券化技术对长期金融工具的应用.
- 使用基因治疗的案例研究分析 Zolgensma.
主要成果:
- 拟议的30年基于绩效的年金有效地减轻了对支付人的细胞和基因疗法的价值和可负担性方面的担忧.
- 证券化结合长期年金证明了高成本,长期治疗的融资可行性.
- 该机制平衡了开发者激励与付款人预算影响和患者访问.
结论:
- 由证券化支持的30年绩效年金,为资助细胞和基因疗法提供了可行的解决方案.
- 这种方法提高了财务可持续性和患者获得创新治疗的机会.
- 拟议的模型解决了先进疗法的现有支付结构中的关键缺口.
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