对我国优先审评审批罕见病用药特征的思考

Thoughts on the characteristics of priority review and approval of rare disease drugs in China

摘要

目的: 分析2018—2024年我国通过优先审评审批程序上市的罕见病用药特征,为优化罕见病用药审评政策、完善用药保障提供参考。方法: 依托国家药品监督管理局及药智数据等数据源,对优先审评审批上市罕见病用药的主要特征进行描述性统计分析。结果: 本研究纳入2018—2024年共86个罕见病用药品种,审评审批平均时长463.55 d;其中,药品类型以化学药(58%)为主,进口药占比77.9%,国产药占比仅22.1%,44个品种纳入医保目录,儿童专属用药仅4个。结论: 我国已构建罕见病用药上市的多路径加速体系,显著提升了罕见病用药的可及性,但仍面临国产创新药供给不足、治疗领域覆盖不均、儿童用药短缺等问题。

Abstract

Objective: To analyze the characteristics of rare disease drugs approved through the priority review and approval process in China from 2018 to 2024, and to provide a reference for optimizing review policies for rare disease drugs and improving drug access. Methods: Based on data from the National Medical Products Administration and Yaozh Data (Drug Intelligence Data) and other sources, a descriptive statistical analysis was conducted on the main features of rare disease drugs approved through priority review and approval. Results: A total of 86 rare disease drugs were included in this study from 2018 to 2024, with an average review and approval duration of 463.55 days. Among them, chemical drugs accounted for 58%, imported drugs accounted for 77.9%, and domestic drugs only accounted for 22.1%. A total of 44 drugs were included in the national medical insurance catalog, and only 4 were pediatric-specific. Conclusion: China has established a multi-pathway accelerated approval system for rare disease drugs, significantly improving the accessibility of rare disease drugs. However, there are still issues such as insufficient supply of domestic innovative drugs, uneven coverage of therapeutic areas, and a shortage of pediatric drugs.

关键词

罕见病用药 / 优先审评审批 / 药品上市特征 / 孤儿药

Key words

rare disease drugs / priority review and approval / characteristics of drug marketing / orphan drugs

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基金资助

辽宁省2025年全省决策咨询和新型智库委托研究课题资助项目(202502)