在世卫组织基本药物清单中,孤儿药物的摄入量
Enrico Costa1, Lorenzo Moja2, Veronika J Wirtz3
1WHO Collaborating Centre for Pharmaceutical Policy and Regulation, Department of Pharmacoepidemiology, Utrecht University, Utrecht, Kingdom of the Netherlands.
Bulletin of the World Health Organization
|January 2, 2024
概括
针对罕见疾病的孤儿药物越来越多地被列入基本药物清单,转向癌症等非传染性疾病. 然而,高昂的成本在贫穷国家带来了难以获得的挑战.
科学领域:
- 药物经济学 药物经济学
- 卫生政策 卫生政策
- 药物监管 药物监管 药物监管
背景情况:
- 美国食品和药物管理局 (FDA) 或欧洲药物管理局 (EMA) 批准的孤儿药物被列入世界卫生组织 (WHO) 基本药物模型清单 (EML) 和儿童EML (EMLc) 并未被系统评估.
- 旨在治疗罕见疾病的孤儿药物代表着制药市场的重要和不断增长的部门,引发了关于它们的可访问性和纳入全球卫生优先事项的问题.
研究的目的:
- 评估1977年至2021年间世卫组织EML和EMLc中孤儿药物的吸收情况.
- 分析孤儿药物纳入的趋势,包括监管批准的时间延迟以及纳入或排除的原因.
主要方法:
- 收集和分析了FDA或EMA批准的孤儿药物的数据.
- 该研究研究了世卫组织EML和EMLc上这些药物的存在,比较了2001年修订纳入标准之前和之后的趋势.
- 包括或拒绝孤儿药物的原因被系统地记录下来.
主要成果:
- 在EML中孤儿药物的比例从1977年的1.9%增加到2021年的14.6%.
- 从用于传染性疾病的孤儿药物到非传染性疾病,特别是癌症,观察到显著的转变.
- 从监管部门的批准到EML的中位时间是13.5年,临床证据有限,益处不确定是主要的拒绝原因.
结论:
- 世卫组织的EML已经扩大到包括用于罕见疾病的药物,尽管缺乏全球罕见疾病定义.
- 许多上市的孤儿药的高成本在资源较低的环境中造成了可访问性和报销的重大障碍.
- 持续监测至关重要,以确保为罕见和常见疾病提供基本药物的公平获取.
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