Advanced therapy medicinal products (ATMPs) are based on genes (gene therapy), cells (cell therapy) and tissues (tissue engineering). Advanced therapies offer the potential to provide treatments for injuries, such as burns, and diseases, such as Alzheimer’s, cancer and others (see the EMA website for further information). They have a great potential to offer true cures.
The EU regulatory framework for such products was established with the Regulation (EC) 1394/2007, which entered into force on 30 December 2008.
The regulation established the centralised marketing authorisation procedure as the applicable route to authorisation for advanced therapies. Furthermore, it established a new committee within the European Medicines Agency (EMA), the Committee for Advanced Therapies (CAT). The CAT is responsible for assessing the quality, safety and efficacy of advanced therapies and for following scientific developments in the field. In addition, the CAT also develops a range of regulatory guidelines for advanced therapies.
In 2006, EBE established a Working Group on Advanced Therapy Medicinal Products, which since mid 2015 is a joint group with EFPIA.
The activities of the Working Group include:
Regulatory conference on advanced therapies
Since 2011, EBE annually organises a conference to discuss the regulatory challenges of advanced therapies, with the support of the EMA. This event has become a forum to discuss the evolution of the regulation of advanced therapies in Europe and beyond. Read here about the most recent discussions in December 2016, where the focus was on gene therapy products.
Good manufacturing practices for ATMPs
Article 5 of Regulation (EC) No 1394/2007 mandates the Commission to draw up guidelines in line with the principles of good manufacturing practice and specific to advanced therapy medicinal products. EBE participated actively in the drafting process by submitting comments during the two rounds of Consultation in 2016 and earlier in 2015.
Article 28 of Regulation (EC) No 1394/2007 sets out a national approval procedure to regulate the manufacture of certain ATMPs meeting certain conditions. This process has been set up in order to comply with an individual medical prescription for a custom-made product for an individual patient prepared on a non-routine basis and used in a hospital in the same Member State under the exclusive professional responsibility of a medical practitioner. The provision is referred to as “hospital exemption”. Since entry into force of the regulation, no common interpretation of ‘non-routine basis’ has developed in Member States. The concern is that this provision creates parallel circuit for national products, potentially not meeting the same criteria as centrally approved products and potentially impeding patient access in other Member States. There is currently no transparency on how all EU Member States implement the hospital exemption provision. EBE, in 2013, published a position paper on this particular point, and participated in a joint letter by several Trade Association addressed to the Commission highlighting the industry concerns on this. In 2017, the joint EBE-EFPIA Working Group issued an updated position on Hospital Exemptions, which is available here.
Commission consultation on advanced therapies
EBE provided input to the 2013 Commission public consultation on Regulation for Advanced Therapy Medicinal Products. For more information see the Commission summary of response on the public consultation can be viewed using the link.