2022年,美国将在高收入的罕见病药物上花费大量资金
Helen Mooney, Aaron S Kesselheim, Benjamin N Rome1
1Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, 1620 Tremont St, Ste 3030, Boston, MA 02120.
The American journal of managed care
|November 25, 2025
概括
高收入的罕见病药物在2022年占美国处方药支出的很大一部分,尽管这些药物被少数患者使用. 这些发现凸显了需要在医疗保健政策中考虑罕见疾病药物成本的必要性. 关键词:罕见病药物,处方药支出,医疗保健政策.
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 罕见疾病
背景情况:
- 高收入的处方药对医疗保健支出产生重大影响.
- 罕见疾病的治疗方法,通常根据"孤儿药物法案"指定,存在独特的经济挑战.
- 了解这些专用药物的市场动态对于政策制定至关重要.
研究的目的:
- 估计2022年美国高收入罕见病药物的利用和相关医疗保健支出.
- 分析这些专用药物所占的处方药总支出的比例.
- 为了确定这些药物的不同保险类型 (商业,医疗保险,医疗补助) 覆盖的患者群体.
主要方法:
- 一项横截面研究分析了2022年美国收入最高的100种处方药.
- 根据"孤儿药物法案"专门批准用于罕见疾病的药物的识别.
- 使用制造商净销售额和医疗保险和医疗补助的公共数据估计药品支出;通过每位受益人医疗保险支出估计的患者人数.
主要成果:
- 在前100名药物中,有19种药物专门用于罕见疾病,年净销售额中位数为197亿美元.
- 这19种药物的总支出达到了451亿美元,占美国60.3亿美元处方药市场总额的7.5%.
- 这些药物平均为17,152名患者提供服务,支出分布在医疗保险 (23.6亿美元),商业保险 (18.0亿美元) 和医疗补助 (3.5亿美元) 上.
结论:
- 治疗罕见疾病的药物构成了高收入制药的显著部分,对医疗保健预算的影响与患者人数不成比例.
- 巨大的财务负担需要将它们纳入聚焦于处方药负担能力的政策讨论中.
- 政策干预应解决罕见病药物使用对患者和更广泛的医疗保健系统的经济影响.
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