ADVANCED THERAPY MEDICINAL PRODUCTS – AN OVERVIEW
ADVANCED THERAPY MEDICINAL PRODUCTS – AN OVERVIEW
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先进治疗药物产品 – 概述
DOI：
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发表时间：
2018
期刊：
影响因子：
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通讯作者：
M. Golemovic
中科院分区：
文献类型：
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作者：
M. Golemovic
先进的治疗药物产品（ATMP）代表了一组创新的药物，包括基因疗法，体细胞疗法和组织工程产品。这些疗法为许多疾病的新治疗方法提供了机会。然而，有许多问题与这种复杂疗法的开发有关，这些复杂疗法与新化学实体或生物制剂的传统开发有很大不同。监管框架、高开发成本、从研究到临床试验的转化以及最终进入临床实践仍然是使ATMP在欧盟（EU）的上市许可（MA）成为一项非常具有挑战性的任务的问题。在欧盟实施ATMP共同监管框架9年后，只有9家ATMP获得MA。其中三种已不在欧盟市场上销售。本概述旨在简要了解这一创新药物领域目前面临的挑战。
Advanced therapy medicinal products (ATMPs) represent an innovative group of medicines comprising gene therapies, somatic cell therapies and tissue engineered products. These therapies offer opportunities for new therapeutic approaches in numerous diseases. However, there are many issues related to the development of such complex therapies that greatly differ from traditional developments of new chemical entities or biologics. The regulatory framework, high cost of development, translation from research to clinical trials and finally into clinical practice are still issues that make marketing authorization (MA) of ATMPs in the European Union (EU) a very challenging task. Nine years after the implementation of a common regulatory framework for ATMPs in the EU, only 9 ATMPs have been granted MA. Three of them are not available on the EU market anymore. This overview aims to give a short insight into the challenges this innovative field of medicines is facing today.