一个可负担的基因药物的路线图
Melinda Kliegman1, Manar Zaghlula2, Susan Abrahamson2
1University of California, Berkeley, Innovative Genomics Institute, Berkeley, CA, USA. melinda.kliegman@berkeley.edu.
Nature
|July 17, 2024
概括
第一个用于治疗状细胞疾病的CRISPR基因编辑疗法CASGEVY获得批准,但成本高昂. 有关可持续替代品的建议旨在降低价格10倍,改善基因治疗的获取.
科学领域:
- 生物技术
- 遗传学
- 医学经济学
背景情况:
- 美国食品和药物管理局已经批准了20种基因疗法,其中包括首个基于CRISPR的状细胞疾病基因编辑疗法CASGEVY.
- 这些先进的疗法在治疗遗传疾病和癌症方面具有显著的前景, 但成本极高, 每个患者超过400万美元.
研究的目的:
- 解决新批准的基因疗法的高成本和有限可用性.
- 提出从发现到市场的基因疗法开发和商业化可持续的替代模型.
主要方法:
- 召集一个多学科工作组制定建议.
- 提出一种新的定价结构和基因疗法开发的商业模式.
- 概述涉及学术许可,制造创新和监管支持的战略.
主要成果:
- 拟议的定价结构可能使每位患者的费用降低十倍.
- 概述了一种新的商业模式,分配责任并利用多种资金来源.
- 通过学术许可,制造和监管来降低成本的战略.
结论:
- 可持续的替代品对于提高转变性基因疗法的可负担性和获取性至关重要.
- 实施拟议的定价和商业模式,以及支持性法规和制造创新,可以扩大患者获得CASGEVY等治疗的机会.
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