Iglesias-López Carolina, Agustí Antonia, Obach Mercè, Vallano Antonio
Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, Barcelona, Spain.
Clinical Pharmacology Service, Vall d'Hebron University Hospital, Barcelona, Spain.
Front Pharmacol. 2019 Aug 30;10:921. doi: 10.3389/fphar.2019.00921. eCollection 2019.
Advanced therapy medicinal products (ATMPs) are a fast-growing field of innovative therapies. The European Union (EU) and the United States (US) are fostering their development. For both regions, ATMPs fall under the regulatory framework of biological products, which determines the legal basis for their development. Sub-classifications of advanced therapies are different between regions, while in EU, there are four major groups, i.e., gene therapy, somatic cell therapy, tissue-engineered therapies, and combined advanced therapies; in US, the sub-classification covers two major groups of products, i.e., gene therapy and cellular therapy. The inclusion criteria that define a gene therapy are equivalent in both regions, and the exclusion criteria are directly related to the indications of the product. In the EU, there is a clear differentiation between cell- and tissue-based products regarding their classification as advanced therapies or coverage by other legal frameworks, whereas in US, there is a broader classification about whether or not these products can be categorized as biologic products. Both in EU and in US, in order to classify a cell- or a tissue-based product as an advanced therapy, it must be ensured that the processing of the cells implies a manipulation that alters their biological characteristics, although the term of manipulation in US differentiates between structural and non-structural cells and tissues. The regulatory terminology used to define ATMPs and their sub-classification reveals some differences between EU and US.
先进治疗医药产品（ATMPs）是创新治疗领域中一个快速发展的领域。欧盟（EU）和美国（US）都在推动其发展。对于这两个地区而言，ATMPs属于生物制品监管框架范畴，该框架决定了其发展的法律依据。先进治疗的子分类在不同地区有所不同，在欧盟，有四大类，即基因治疗、体细胞治疗、组织工程治疗和联合先进治疗；在美国，子分类涵盖两大类产品，即基因治疗和细胞治疗。在这两个地区，定义基因治疗的纳入标准是等同的，而排除标准与产品的适应症直接相关。在欧盟，基于细胞和组织的产品在作为先进治疗的分类或其他法律框架的覆盖范围方面有明显区别，而在美国，关于这些产品是否可归类为生物制品有更广泛的分类。在欧盟和美国，为了将基于细胞或组织的产品归类为先进治疗，必须确保细胞的处理意味着一种改变其生物学特性的操作，尽管在美国，操作一词在结构和非结构细胞及组织之间有所区分。用于定义ATMPs及其子分类的监管术语揭示了欧盟和美国之间的一些差异。
