Advanced therapy medicinal products are based on genes (gene therapy), cell (cell therapy) and tissues (tissue engineering). Advanced therapies offer the potential to provide therapies for the treatment of injuries, such as burns, and diseases, such as Alzheimer’s, cancer and others (see to EMA website for further information).
The EU regulatory framework was established with the entry into force of the Regulation on advanced therapies, Regulation (EC) 1394/2007.
The Regulation established a centralised marketing authorisation procedure and a new committee, the Committee for Advanced Therapies (CAT), within the EMA. The CAT is responsible for assessing the quality, safety and efficacy of advanced therapies and following scientific developments in the field.
In addition, the CAT also develops a range of regulatory guidelines:
The EBE has established an ad hoc Advanced Therapies Task Force. The most recent activities of the TF involve:
Article 28 of the Advanced Therapies Regulation (1394/2007) set out a procedure (the so-called hospital exemption) establishing a national procedure to regulate the manufacture of certain non-routine produced ATMPs outside the scope of the Regulation. Since the entry into force of the Regulation, there has not been a common interpretation of ‘non-routine’ by Member States leading to concern that this would establish a parallel circuit for national products not meeting the same criteria as centrally approved products.
The EBE TF participated in a joint Trade Association letter to the Commission highlighting the industry concerns on this issue.
In March 2013 the Commission launched a public consultation on the Regulation for Advanced Therapy Medicinal Products, inviting comments on a number of identified topics. EBE provided input.
For more information see the Commission summary of response on the public consultation can be viewed using the link.