细胞和基因治疗产品在欧盟、美国、日本、韩国和中国的上市后监测框架：比较研究。
Post-marketing surveillance framework of cell and gene therapy products in the European Union, the United States, Japan, South Korea and China: a comparative study.
机构信息
Department of Clinical Pharmacy and Regulatory Science, School of Pharmacy, Fudan University, Shanghai, 201206, China.
Department of Rheumatology, Renji Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, 200001, China.
出版信息
BMC Med. 2024 Sep 27;22(1):421. doi: 10.1186/s12916-024-03637-z.
BACKGROUND
Cell and gene therapy products (CGTPs) often receive accelerated approvals, lacking comprehensive long-term safety and efficacy data, which can raise significant safety concerns. This research aims to study the post-marketing surveillance (PMS) of CGTPs in the European Union (EU), the United States (US), Japan, South Korea, and China, to offer insights for the development of a secure and standardized post-marketing regulatory framework for CGTPs.
METHODS
Related regulations and the implementation effect of PMS for approved CGTPs were studied searching PubMed, CNKI, and the official websites of the European Medicines Agency, the US Food and Drug Administration, Japan's Pharmaceuticals and Medical Device Agency, South Korea's Ministry of Food and Drug Safety, and the National Medical Products Administration of China.
RESULTS
Compared to those in China, the guidelines of PMS for CGTPs in the EU, the US, Japan, and South Korea was more comprehensive. Notably, the EU had dedicated regulations and supporting guidelines of PMS. Of the 26 CGTPs approved in the EU, 88% were under additional monitoring, 38% received conditional marketing authorization, and 12% were authorized under exceptional circumstances, with 77% designated as orphan drugs. The US had released 34 guidelines specifically for CGTPs which, forming the foundation of post-marketing risk management. Among the 27 CGTPs approved in the US, 22% were required to perform risk evaluation and mitigation strategies, 37% added black box warnings in the package inserts, 63% mandated to post-marketing requirements, and 15% subject to post-marketing commitments. In Japan, stringent supervision measures encompassing all-case surveillance (79%) and re-examination (53%) were applied to the 19 approved CGTPs, with 21% approved through conditional and time-limited approval. The PMS for CGTPs in South Korea, mainly included PSUR, re-examination, and re-evaluation. China had introduced several relevant regulations, which consisted of general statements and lacked detailed guidance.
CONCLUSIONS
This study demonstrates that the regulatory policies of PMS for CGTPs in the EU, the US, Japan, and South Korea were comprehensive. The implementation of PMS for CGTPs in the EU, the US, and Japan was well developed. This knowledge holds valuable insights for China's future learning and development in this field.
背景
细胞和基因治疗产品（CGTPs）通常获得加速批准，缺乏全面的长期安全性和疗效数据，这可能引发重大的安全性担忧。本研究旨在研究欧盟（EU）、美国（US）、日本、韩国和中国的 CGTP 上市后监测（PMS），为 CGTP 安全标准化的上市后监管框架的发展提供参考。
方法
通过检索 PubMed、CNKI 和欧洲药品管理局、美国食品和药物管理局、日本药品和医疗器械管理局、韩国食品药品安全部以及中国国家药品监督管理局的官方网站，研究了已批准 CGTPs 的相关法规和 PMS 的实施效果。
结果
与中国相比，欧盟、美国、日本和韩国的 CGTPs PMS 指南更加全面。值得注意的是，欧盟有专门的 PMS 法规和支持性指南。在欧盟批准的 26 种 CGTPs 中，有 88%处于额外监测中，38%获得有条件上市许可，12%获得特殊情况下的授权，77%被指定为孤儿药。美国发布了 34 项专门针对 CGTPs 的指南，这些指南构成了上市后风险管理的基础。在美国批准的 27 种 CGTPs 中，有 22%需要进行风险评估和缓解策略，37%在包装说明书中添加黑框警告，63%需要进行上市后要求，15%需要进行上市后承诺。在日本，对 19 种批准的 CGTPs 实施了严格的监管措施，包括全病例监测（79%）和再审查（53%），其中 21%通过有条件和限时批准获得批准。韩国的 CGTPs PMS 主要包括 PSUR、再审查和再评价。中国已经出台了几项相关法规，这些法规包括一般性说明，缺乏详细指导。
结论
本研究表明，欧盟、美国、日本和韩国的 CGTPs PMS 监管政策较为全面，欧盟、美国和日本的 CGTPs PMS 实施较为完善。这些知识为中国今后在该领域的学习和发展提供了有价值的参考。
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