同情用药制度对我国罕见病患者用药可及性研究

Study on the Drug Accessibility of Compassionate Use to Domestic Patients with Rare Diseases

目的:基于国内罕见病患者用药可及性现状,剖析国外同情用药的制度体系及实施成效,深入分析同情用药制度对国内罕见病患者的用药可及性问题,并提出贴合国内罕见病患者同情用药制度落实的细则。方法:查阅美国FDA发布的法规政策、技术指南、相关统计数据及文献资料,对美国同情用药的使用类别、申请及审批流程、费用支付方式等内容进行分析。结果与结论:美国已具备较为完善的同情用药制度体系,该制度在我国的建立或将成为国内罕见病患者用药可及的一条新路径,在一定程度上能够提高国内罕见病患者的用药可及性。

Objective:To analyze the system and implementation effectiveness of foreign compassionate use,and the accessibility of compassionate medication system for rare disease patients in China,based on the current situation of drug accessibility for rare disease patients in China,and to propose the detailed rules of compassionate use system for domestic rare diseases patients.Methods:The regulations,policies,technical guidelines,relevant statistics and literature published by the US FDA were searched,and the use categories,application and approval processes,fees and payment methods by consulting the regulations and policies,technical guidelines,relevant statistics and literature published by FDA were analyzed.Results and Conclusion:The United States has a relatively complete system of compassionate use.The establishment of this system in China may become a new path for domestic rare disease patients to obtain medication,and to a certain extent,it could improve the drug availability of domestic rare disease patients.