| 张军,李笑蕾,任跃明.中国与欧盟、美国基因治疗产品安全性监管研究[J].中国药事,2021,35(4):366-379 |
| 中国与欧盟、美国基因治疗产品安全性监管研究 |
| On Safety Regulations of Gene Therapy Products in China, EU and USA |
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| DOI：10.16153/j.1002-7777.2021.04.002 |
| 中文关键词: 基因治疗产品 监管 安全性 基因编辑技术 |
| 英文关键词: gene therapy products supervision safety gene editing technology |
| 基金项目: |
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| 摘要点击次数: 943 |
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| 中文摘要: |
| 目的：为修订我国基因治疗产品安全性监管法规提供参考。方法：通过回顾基因治疗产品的研发史及伴随的安全性问题，分析欧盟及美国对基因治疗产品安全性监管法规，并与我国的相应法规进行比较，提出欧洲及美国的监管可借鉴的经验及我国在基因治疗产品安全性监管方面的差距。结果：2019年前，我国针对基因治疗产品的安全性监管法规，内容相对简单，规定较为笼统，针对具体问题缺乏详细说明和规定，法规约束性不强，审批上相对宽松。欧盟和美国在安全性监管体系方面，由于起步较早， 相对严谨和完善，提出了具体、详细的技术要求，如非临床试验原则、产品特性分析、动物种属选择、 药效学求证研究、药代动力学、安全药理学、毒理学和安全性评价等。针对上述问题，国家药品监督管理局在2021年2月发布了“基因治疗产品非临床研究与评价技术指导原则(征求意见稿)”，正在逐步完善该类产品的安全科学监管体系。结论：基因治疗产品是具有高度独特性、多样性及技术高度复杂性的全新产品，我国有待进一步完善适合全新产品监管的法律法规。在修订产品的安全性监管法规时，可以借鉴欧盟和美国的监管经验，使其符合国际趋势和要求，少走弯路。 |
| 英文摘要: |
| Objective: To provide references for the revision of regulations on the safety supervision of gene therapy products in China. Methods: By reviewing the history of the development of gene therapy products and the associated safety issues, the European and American regulations on the safety of gene therapy products were analyzed and compared with the corresponding regulations in China. The borrowed experience of European and American regulations and deficiencies in safety regulation of gene therapy products in China were proposed. Results: Before 2019, China's regulations on gene therapy products were relatively simple and general, lacking detailed descriptions and regulations for specific issues. The regulations were not very restrictive, and the approval is relatively lax. Due to the early start, European and American regulatory systems are relatively rigorous and perfect, putting forward specific and detailed technical requirements such as the principles of nonclinical trials, analysis of product characteristics, animal species selection, research on pharmacodynamics, pharmacokinetics, safety pharmacology and toxicological safety evaluation. In response to the above questions, the National Medical Products Administration released “Technical Guidelines for Non-clinical Research and Evaluation of Gene Therapy Products (draft)” in February 2021, which is gradually improving the safetyand the scientific regulatory system for such products. Conclusion: Gene therapy products are brand new products with high uniqueness, diversity and technical complexity, and China needs to further develop laws and regulations suitable for the supervision of brand-new products. When revising safety regulations for products, China may draw on the regulatory experience of Europe and the United States to make them in line with international trends and requirements to avoid detours. |
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