国内外细胞和基因治疗药品监管研究

doi:10.19803/j.1672-8629.20240160

摘要/Abstract

摘要： 目的为完善我国细胞和基因治疗药品监管,促进产业高质量发展提供建议。方法通过查阅法规和文献,对比分析美、欧、日国家或地区细胞和基因治疗产品监管基本情况,结合我国监管实际情况和产业发展需求,分析国外监管模式给予我国的启示。结果和结论 基于目前我国细胞和基因治疗药品审评审批的具体实际,借鉴美日欧监管模式和审评流程设计,从创新监管理念、整合审评资源、加强国际交流与合作以及加大扶持力度等方面着手推进我国监管体系的发展。

关键词: 细胞, 基因, 治疗, 美国食品药品监督管理局, 欧洲药品监督管理局, 日本药品和医疗器械管理局, 监管, 审评, 审批

Abstract: Objective To give tips about how to improve the regulation of cellular and gene therapies in China and promote high-quality development of the industry. Methods The ways in which cellular and gene therapy products were regulated in the US, Japan and Europe were compared by reviewing regulations and literature. The implications for China were analyzed in the light of actual regulation in China and the needs of industrial development. Results and Conclusion Based on the current practices related to the review and approval of cellular and gene therapies in China and by learning from the regulatory models and review process designs of the United States, Japan and Europe, China's regulatory system can be upgraded by means of innovative regulatory concepts, integration of review resources, more international exchanges and cooperation as well as intensified efforts.

Key words: cellular, gene, therapy, FDA, EMA, PMDA, regulatory, review, approval

中图分类号:

R951
R915

赵培培, 温宝书. 国内外细胞和基因治疗药品监管研究[J]. 中国药物警戒, 2024, 21(9): 1019-1024.

ZHAO Peipei, WEN Baoshu. Regulation of cellular and gene therapies at home and abroad[J]. Chinese Journal of Pharmacovigilance, 2024, 21(9): 1019-1024.

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链接本文: https://zgywjj.magtechjournal.com/CN/10.19803/j.1672-8629.20240160

https://zgywjj.magtechjournal.com/CN/Y2024/V21/I9/1019

参考文献

[1] LIANG X.Faith-based medicine“overcomes the enemy”with technology[J]. China Entrepreneur(中国企业家), 2022(7): 64-67.
[2] Frost China.White Paper on the Development of Cell and Gene Therapy Industry in China[Z/OL]. [2023-07-09].https://www.frostchina.com/content/insight/detail?id=62f207bf3a1cb46c9a9fd52d.
[3] Grandview Research Website. Cell Therapy Market Size, Share and Growth Report, 2030[EB/OL]. [2024-01-20]. https://www.grandviewresearch.com/industry-analysis/cell-therapy-market.
[4] Finance China. Gene therapy market explodes! Market size to exceed $30.54 billion by2025[EB/OL]. [2024-01-16]. https://finance.china.com.cn/industry/medicine/20210615/5593339.shtml.
[5] WU SX, YANG SJ, WU ZZ.Study on regulatory policies of cell therapy in the US, EU and Japan[J]. Chin Med Biotechno(中国医药生物技术), 2016, 11(6): 491-496.
[6] ZHAO CY,HUANG YH,GAO CY.Overview of expedited regulatory programs for regenerative medicine therapies in US FDA[J]. China Food & Drug Administration Magazine(中国食品药品监管), 2021(8): 24-33.
[7] EMA. Committee for Advanced Therapies(CAT) [Z/OL].[2023-07-16]. https://www.ema.europa.eu/en/committees/committee-advanced-therapies-cat.
[8] HUANG MQ, LIU YT, ZHOU QQ, et al.Legal risks and regulatory strategies in the field of immunotherapy[J]. Medicine and Philosophy(医学与哲学), 2023, 44(15): 51-55.
[9] WANG GJ, WANG Y, LI J, et al.Introduction to the regulatory system of cell therapy products abroad and its enlightenment for China[J]. China Food & Drug Administration Magazine(中国食品药品监管), 2023(9): 6-13.
[10] NAGAI S.Flexible and expedited regulatory review processes for innovative medicines and regenerative medical products in the US, the EU, and Japan[J]. International Journal of Molecular Sciences, 2019, 20(15): 3801.
[11] LI Y, ZHANG X.The experience and enlightenment of cell therapy regulation dual-track system in Japan[J]. China Biotechnology(中国生物工程杂志), 2020, 40(1/2): 174-179.
[12] Zhihu. What is the MHLW certification in Japan?[EB/OL]. (2021-05-18)[2024-04-07]. https://zhuanlan.zhihu.com/p/373236721.
[13] Zhihu. Introduction to the official website of PMDA Japan[EB/OL](2023-07-25)[2024-04-07]. https://zhuanlan.zhihu.com/p/645824122.
[14] LI LL. Regulatory model for regenerative medical products Japanese experience[N]. Pharmaceutical Economic News(医药经济报), 2023-07-03(2).
[15] PMDA. Pharmaceuticals and Medical Devices Agency[EB/OL].[2024-04-09]. https://www.pmda.go.jp/english/review-services/reviews/0001.html.
[16] LI X, SONG XT.Review of the legal system of regenerative medicine in Japan[J]. Social Sciences Abroad(国外社会科学), 2017, 4(3): 125-135.
[17] YU GJ, WU YF, WANG K, et al.Comparison of international regulation of cell and gene therapy products and implications for China[J]. China Food & Drug Administration Magazine(中国食品药品监管), 2019(8): 4-19.
[18] Frost China.Blue book on the status and trends of the gene drug industry[Z/OL]. [2024-01-16].https://www.frostchina.com/content/insight/detail?id=657feb0c58fb10b7d6fb122e.
[19] REN F, SHAO R.Research on incentive policies of innovative drugs in Japan and its effectiveness[J]. Economic Research Guide(经济研究导刊), 2017(4): 174-177.
[20] Zhihu. Harmony and difference, breaking through the Chinese dilemma! a brief analysis of orphan drug incentive policies in 4 countries[EB/OL]. (2023-01-13)[2024-02-08]. https://zhuanlan.zhihu.com/p/598705220.
[21] CHEN MF, XIE JP.Study on the Sakigake designation system and the enlightenment to China[J]. Chinese Journal of New Drugs(中国新药杂志), 2023, 32(13): 1281-1284.
[22] Sohu. Why the industry is about to explode from 20 years of China’s cellular policy (country level1993-2023)[EB/OL]. [2024-02-22]. https://www.sohu.com/a/www.sohu.com/a/745319744_550330.
[23] CHANG HY.Research on the current status and countermeasures of stem cell clinical research supervision in China[D]. Dalian: Dalian Medical University, 2020.
[24] ZHANG Y.Opportunities and challenges for the development and industrialization of new cell therapy drugs in China[J]. Progress in Pharmaceutical(药学进展), 2023, 47(1): 1-5.
[25] SHAO R, SUN HS, YAN JZ, et al.Study on the priority medicines in the European Union[J]. Chinese Journal of New Drugs(中国新药杂志), 2017, 26(6): 614-619.
[26] LI Y.International experience and enlightenment of cell gene therapy regulatory[D]. Nanjing: Southeast University, 2020.
[27] China Association for the Promotion of Pharmaceutical Innovation, China Association of Enterprises with Foreign Investment, Pharmaceutical Research and Development Industry Committee. Promoting simultaneous R&D, registration and review of innovative drugs to build China’s pharmaceutical innovation ecosystem - importance and general recommendations[J]. China Food & Drug Administration Magazine(中国食品药品监管), 2022(7): 4-13.