Salmikangas P, Flory E, Reinhardt J, Hinz T, Maciulaitis R
National Agency for Medicines, Mannerheimintie 103b, FI-00301, Helsinki.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2010 Jan;53(1):24-9. doi: 10.1007/s00103-009-0991-5.
The new era of regenerative medicine has led to rapid development of new innovative therapies especially for diseases and tissue/organ defects for which traditional therapies and medicinal products have not provided satisfactory outcome. Although the clinical use and developments of cell-based medicinal products (CBMPs) could be witnessed already for a decade, robust scientific and regulatory provisions for these products have only recently been enacted. The new Regulation for Advanced Therapies (EC) 1394/2007 together with the revised Annex I, Part IV of Directive 2001/83/EC provides the new legal framework for CBMPs. The wide variety of cell-based products and the foreseen limitations (small sample sizes, short shelf life) vs. particular risks (microbiological purity, variability, immunogenicity, tumourigenicity) associated with CBMPs have called for a flexible, case-by-case regulatory approach for these products. Consequently, a risk-based approach has been developed to allow definition of the amount of scientific data needed for a Marketing Authorisation Application (MAA) of each CBMP. The article provides further insight into the initial risk evaluation, as well as to the quality, non-clinical, and clinical requirements of CBMPs. Special somatic cell therapies designed for active immunotherapy are also addressed.
再生医学的新时代推动了新型创新疗法的快速发展，尤其是针对传统疗法和医药产品未能提供满意疗效的疾病以及组织/器官缺陷。尽管基于细胞的医药产品（CBMPs）的临床应用和发展已有十年之久，但针对这些产品的强有力的科学和监管规定直到最近才得以颁布。新的《高级治疗法规》（EC）1394/2007以及对2001/83/EC指令附件I第四部分的修订为CBMPs提供了新的法律框架。CBMPs种类繁多，且存在预期的局限性（样本量小、保质期短）与特定风险（微生物纯度、变异性、免疫原性、致瘤性），这就要求对这些产品采取灵活的、逐案的监管方法。因此，已制定了基于风险的方法，以确定每种CBMP的上市许可申请（MAA）所需的科学数据量。本文进一步深入探讨了初始风险评估以及CBMPs的质量、非临床和临床要求。还讨论了为主动免疫治疗设计的特殊体细胞疗法。
