Europe – Advanced therapy medicinal products regulations in the EU

This article explains some of the terminology relating to advanced therapy medicinal products (ATMPs), including gene and cell-based therapies, tissue-engineered products, and combined ATMPs. The author explains the key EU regulations and guidance documents for each therapy type, and discusses marketing authorization, accelerated regulatory pathways, and market access. He advises companies navigating this complex regulatory environment to engage with the regulatory agencies early and often during the approval process and to seek external regulatory support if in-house resources are limited.

Advanced therapy medicinal products include cell therapies, gene therapies, and tissue- engineered products. These highly complex treatments differ from traditional medicines, both in how they are made and administered, and in the type of benefits they may provide. These are products for which regulations were established fairly recently and that present challenges to the regulatory agencies on how to best to regulate them within the existing regulatory framework.