Challenges with advanced therapy medicinal products and how to meet them
Challenges with advanced therapy medicinal products and how to meet them
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DOI：
10.1038/nrd3052
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发表时间：
2010-03-01
影响因子：
120.1
通讯作者：
Celis, Patrick
中科院分区：
文献类型：
--
作者：
Schneider, Christian K.;Salmikangas, Paula;Celis, Patrick
先进治疗药品（ATMP），包括基因治疗药品，体细胞治疗药品和组织工程产品，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提供了主要希望。因此，它们引起了相当大的兴趣和辩论。继欧洲对ATMP的监管之后，最近为这些创新药物建立了一个统一的监管框架。该框架的核心是欧洲药品管理局（EMA）的先进疗法委员会（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学协会。在本文中，CAT讨论了ATMP开发人员提出的一些典型问题，并强调了这些公司和研究团体在开发过程中作为监管顾问接触EMA和CAT的机会。
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.