细胞治疗药品生产技术的发展与监管挑战  

Development of Manufacturing Technology of Cell Therapy Medicinal Products and Regulatory Challenges

作　　者：王雪 卢加琪 韦薇[1] 何伍[1] 王涛[1] WANG Xue;LU Jia-qi;WEI Wei;HE Wu;WANG Tao(Centre for Drug Evaluation,National Medical Products Administration)

机构地区：[1]国家药品监督管理局药品审评中心

出　　处：《中国食品药品监管》2024年第8期4-11,共8页China Food & Drug Administration Magazine

摘　　要：近年来,以嵌合抗原受体T细胞(CAR-T)为代表的细胞治疗药品在我国的申报数量大幅增加,多款产品成功获批上市,为血液系统恶性肿瘤等复发或难治性疾病提供了有效的治疗手段。现阶段细胞治疗药品生产成本高,价格昂贵,临床可及性受限,为更好地控制成本、提高药品安全性、解决供应链“卡脖子”的问题、让细胞治疗惠及更多患者群体,研发机构开始探索各种方法。然而,细胞治疗药品生产工艺复杂,工业界和监管方对工艺和质量的理解仍处于逐步加深过程中,且不断优化的生产工艺和创新技术的应用给研发和监管均带来了较大挑战。本文以CAR-T类细胞治疗药品为例,概述了创新生产技术在细胞治疗药品中的研究进展,并结合现阶段监管现状和审评积累,对细胞治疗药品研发和监管面临的挑战进行了分析,以期为该类产品的研发和监管政策制定提供参考。In recent years,the number of applications for cell therapy medicinal products,particularly those involving chimeric antigen receptor T cell(CAR-T)therapies,has surged significantly in China.Several products have been approved,providing effective treatment options for relapsed or refractory diseases such as hematological malignancies.Despite these advancements,the high production costs and prices limit the clinical accessibility of these therapies.To better control costs,enhance drug safety,solve supply chain challenges,and achieve broader patient access,research and development(R&D)institutions are exploring innovative methods.Nevertheless,the manufacturing processes of cell therapy products are intricate,and the understanding of these processes and their quality is still evolving among stakeholders and regulators.Continuous optimization of manufacturing processes and the application of innovative technologies pose significant challenges to both R&D and regulation.This paper discusses the progress of innovative manufacturing technologies in the context of CAR-T cell therapies and analyzes the challenges to R&D of cell therapy medicinal products based on current regulatory frameworks and evaluation experiences.These insights aim to provide a reference for the R&D and regulatory policy formulation of cell therapy medicinal products.

关 键 词：细胞治疗药品 创新生产技术 生产模式 自动化制备 药品监管 

分 类 号：R95[医药卫生—药学]