Nat Rev Drug Discov. 2010 Mar;9(3):195-201. doi: 10.1038/nrd3052.
Advanced therapy medicinal products (ATMPs), which include gene therapy medicinal products, somatic cell therapy medicinal products and tissue-engineered products, are at the cutting edge of innovation and offer a major hope for various diseases for which there are limited or no therapeutic options. They have therefore been subject to considerable interest and debate. Following the European regulation on ATMPs, a consolidated regulatory framework for these innovative medicines has recently been established. Central to this framework is the Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMA), comprising a multidisciplinary scientific expert committee, representing all EU member states and European Free Trade Association countries, as well as patient and medical associations. In this article, the CAT discusses some of the typical issues raised by developers of ATMPs, and highlights the opportunities for such companies and research groups to approach the EMA and the CAT as a regulatory advisor during development.
先进治疗药物产品（ATMPs）包括基因治疗药物产品、体细胞治疗药物产品和组织工程产品，处于创新的前沿，为各种治疗选择有限或没有治疗选择的疾病带来了重大希望。因此，它们引起了相当大的关注和争议。根据欧洲关于 ATMPs 的法规，最近为这些创新药物建立了一个综合监管框架。该框架的核心是欧洲药品管理局（EMA）的先进治疗药物委员会（CAT），该委员会由一个多学科科学专家委员会组成，代表所有欧盟成员国和欧洲自由贸易联盟国家，以及患者和医疗协会。在本文中，CAT 讨论了 ATMPs 开发者提出的一些典型问题，并强调了此类公司和研究小组在开发过程中作为监管顾问与 EMA 和 CAT 联系的机会。
