Medicinal products for human use
Advanced therapy refers to new medical products that use gene therapy, cell therapy, and tissue engineering. They can be used to treat diseases or injuries, such as skin in burns victims, Alzheimer's, and cancer or muscular dystrophy, and have huge potential for the future of medicine.
EU Framework for Advanced Therapies
The EU's Regulation on advanced therapies, is designed to ensure the free movement of advanced therapy products within Europe, to facilitate access to the EU market, and to foster the competitiveness of European companies in the field, while guaranteeing the highest level of health protection for patients.
What are the main elements of the Regulation?
- A centralised marketing authorisation procedure.
- A multidisciplinary expert Committee (Committee for Advanced Therapies), within the European Medicines Agency (EMA), to assess advanced therapy products.
- Technical requirements adapted to the particular characteristics of these products.
- Special incentives for small and medium-sized enterprises.
The Regulation recognises that some advanced therapy products combine biological materials, such as tissues or cells, with chemical elements such as metal implants or polymer scaffolds. These combination products require adapted regulatory requirements.
GMP for ATMPs
The European Commission adopted Guidelines on Good Manufacturing Practice (GMP) specific for Advanced Therapy Medicinal Products (ATMP) in November 2017. The Guidelines provide a specific GMP framework that is adapted to the specific characteristics of ATMP.
GMO requirements for investigational products
Clinical trials with medicinal products that contain or consist of GMOs (Genetically modified organisms) are subject to both clinical trials and GMO legislations.
Dissemination of information about national regulatory requirements in respect of GMO aspects is expected to facilitate the development of gene therapy medicinal products in the EU.
- A repository of national regulatory requirements has been created to this effect
Action Plan on ATMPs
The Commission services and the European Medical Agency (EMA) have launched a joint action plan to foster the development of advanced therapy medicinal products (ATMPs), with the aim of streamlining procedures and better addressing the specific requirements of ATMP manufacturers.