目的 介绍国内外罕见病酶替代疗法药物开发的最新研究进展及药学评价思考。方法 以国内外有代表性的文献为基础, 结合审评实践,简要就此类产品药学评价(分子设计、宿主选择、生产工艺与质量控制等)提出一些思考和建议。结果 由于罕见病酶替代疗法药物分子的“复杂性”与“异质性”,其质量研究是药学开发与评价的主要难点。 结论 近年来,为缓解中国罕见病药物缺乏现状,满足临床迫切需求,国家出台了一系列政策鼓励罕见病药物的研发。
Abstract
OBJECTIVE To summarize the latest research progress in the development of enzyme replacement therapy drugs for rare diseases at home and abroad and their pharmaceutical assessment. METHODS Based on the representative literature at home and abroad, this paper propose key points for consideration (such as molecular design, host selection, production process and quality control, etc.), as well as comments and suggestions for researcher of such products from review and evaluation perspective. RESULTS Due to the complexity and heterogeneity of the drug molecules of enzyme replacement therapy for rare diseases, the quality research is the main difficulty in pharmaceutical development and evaluation. CONCLUSION In recent years, in order to alleviate the lack of rare disease drugs in China and meet the urgent clinical needs, Chinese government has issued a series of policies to encourage the research and development of rare disease drugs.
关键词
酶替代疗法 /
罕见病 /
制备工艺 /
质量控制 /
药学评价
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Key words
enzyme replacement therapy /
rare disease /
manufacturing process /
quality control /
pharmaceutical evalution
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中图分类号：
R95
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参考文献
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