摘要
近年来,国家出台了一系列政策鼓励罕见病药物的研发。本文分析罕见病酶替代疗法(enzyme replacement therapy,ERT)生物制品国内外注册情况及药学研发挑战,汇总了药学开发及技术要求相关政策支持信息,并结合药学审评经验及相关文献,对此类产品在药学研究中常见技术问题进行探讨。
In recent years,the state has introduced a series of policies to encourage the development of rare disease drugs.This article analyzes the registration situation of enzyme replacement therapy biologics for rare diseases both domestically and internationally,the research and development challenges,summarizes the policy support information at the pharmaceutical research and development level,and discusses common technical issues in the pharmaceutical development of such products based on pharmaceutical review experience and relevant literature.
作者
阚红金
程速远
KAN Hong-jin;CHENG Su-yuan(Center for Drug Evaluation,National Medical Products Administration,Beijing 100076,China)
出处
《中国新药杂志》
北大核心
2025年第24期2660-2667,共8页
Chinese Journal of New Drugs
基金
盖茨基金会国际专家赠款资助项目(二期):罕见病治疗用重组酶及蛋白药学研究与评价(1.4.10)。
关键词
罕见病
酶替代疗法
药学评价
rare diseases
enzyme replacement therapy
biological products
