Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Kanagawa, Japan.
Center for Regenerative Medicine, National Center for Child Health and Development, Tokyo, Japan.
Stem Cell Reports. 2023 Aug 8;18(8):1573-1591. doi: 10.1016/j.stemcr.2023.05.003.
Currently, many types of cell-based therapeutic products (CTPs) derived from pluripotent stem cells (PSCs) are being developed in a lot of countries, some of which are in clinical trial stages. CTPs are classified differently in different countries and regions. The evaluation of their efficacy, safety, and quality also differs from that for conventional small-molecule drugs and biopharmaceuticals, which reflects the complex properties of living cells and unmet medical needs. Since there are no international guidelines to evaluate CTPs, including PSC-derived products, it is necessary to be aware of differences in relevant laws and regulations in different countries and regions. International consortia are organized and actively working to standardize/harmonize the evaluation methods and regulations to facilitate the development and global distribution of PSC-derived CTPs. In this paper, we outline the regulations related to PSC-derived CTPs in the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use founding regions (US, EU/UK, Japan) and introduce representative consortia working on their standardization.
目前,许多国家正在开发基于多能干细胞(PSCs)的多种细胞治疗产品(CTPs),其中一些已进入临床试验阶段。不同国家和地区对 CTPs 的分类不同,对其疗效、安全性和质量的评估也与传统的小分子药物和生物制药不同,这反映了活细胞的复杂特性和未满足的医疗需求。由于目前尚无评估 CTPs(包括 PSC 衍生产品)的国际指南,因此有必要了解不同国家和地区相关法律法规的差异。国际联盟组织起来并积极努力规范/协调评估方法和法规,以促进 PSC 衍生 CTPs 的开发和全球分布。本文概述了人用药品技术要求国际协调理事会(美国、欧盟/英国、日本)成立地区与 PSC 衍生 CTPs 相关的法规,并介绍了正在标准化方面开展工作的代表性联盟。
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