研究和开发支出与CFTR调节器批准后的收入相比
Jonathan Guo1, Grace Hennessy1, Benedict Young1
1School of Public Health, Faculty of Medicine, Imperial College London, United Kingdom.
概括
囊性纤维化跨膜导电调节器 (CFTR) 调节器的高价格阻碍了获取,特别是在低收入国家. 尽管研发成本很高,但收入很快弥补了这些费用,引发了人们对全球卫生公平的担忧.
科学领域:
- 制药经济学 制药经济学
- 药品定价分析 药品定价分析
- 卫生公平研究 卫生公平研究
背景情况:
- 囊性纤维化跨膜导电调节器 (CFTR) 调节器的高成本为患者创造了进入障碍,特别是在低收入国家.
- 研究和开发 (R&D) 支出经常被认为是药物定价的主要驱动因素.
- 围绕CFTR调节器的独特情况允许在批准前的研发投资和批准后的财务回报之间进行直接比较.
研究的目的:
- 分析Cystic Fibrosis跨膜导电性调节器 (CFTR) 调节器的研发支出和收入之间的关系.
- 评估CFTR调节器疗法的财务可持续性和可访问性.
- 为未来的药物开发战略和罕见病定价提供信息.
主要方法:
- 利用公司的证券交易委员会文件,从2001年到2024年为CFTR调节器提取了研发支出和收入数据.
- 将所有财务价值调整为2024年的通胀水平.
- 将资本成本纳入使用10.5%的折扣率,敏感性分析为7%和14%.
主要成果:
- 在elexacaftor/tezacaftor/ivacaftor获得营销批准之前,总的研发支出达到185亿美元,估计的资本成本为248亿美元.
- 从2012年到2024年,累计收入达到638亿美元,超过了2020年第二季度的研发支出.
- 每年Elexacaftor/tezacaftor/ivacaftor的营收超过102亿美元,使其成为收入最高的制药产品之一.
结论:
- 由于高收入,CFTR调节器的研发成本在其商业生命周期的早期得到了回收.
- 目前这些药物的定价策略危害了CF患者的全球健康公平,并使医疗保健系统的财政受到压力.
- 迫切需要开发可持续模型,以平衡公平获取与为罕见疾病治疗提供财政激励,包括新兴基因治疗.
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