Gene therapy medicinal products contain genes, administration of which into the human body, serve the therapeutic, prophylactic or diagnostic effect via expression of inserted recombinant nucleic acid.
Cell-based medicinal products cover a wide range of cell therapies, including somatic cell and tissue-engineered products that have been manufactured from a patient’s own cells (autologous), donor cells (allogeneic) or animal cells (xenogeneic). Cells in these biological medicinal products have usually undergone substantial manipulation and could be used to perform different essential function. Tissue-engineered products are developed with the aim to repair, regenerate or replace various tissue defects.
ATMPs may incorporate as an integral part of the product, one or more medical devices, in which case they are referred to as “combined advanced therapy medicinal products”.
Regulation of advanced therapies medicinal products
Directives and Regulations relevant to the licensing of ATMPs are:
- Directive 2001/83/EC
- Directive 2009/120/EC
- Regulation (EC) No 726/2004
- Regulation (EC) No 1394/2007 (‘the ATMP Regulation’)
ATMPs are evaluated via a centralised procedure as defined by European Union legislation (Article 8 of Regulation (EC) No 1394/2007). This process ensures that these medicinal products benefit from a single evaluation and authorisation procedure applicable across the European Union.
The Committee for Advanced Therapies at the European Medicines Agency prepares a draft opinion on the quality, safety and efficacy of each advanced therapy medicinal product subject to marketing authorisation. The application for a marketing authorisation for a combined ATMP which contains a medical device should include evidence that the device meets the essential requirements set out in the medical devices legislation. The CAT’s opinion is then sent to the Committee for Medicinal Products for Human Use, the committee responsible for human medicines at the European Medicines Agency. Based on the Committee for Advanced Therapies opinion, the Committee for Medicinal Products for Human Use adopts a recommendation for presentation to the European Commission for decision binding in all Member States. Such evaluation is carried out in line with existing European Union legislation. Further information about the CAT, EMA’s role in the regulation of ATMPs and guidelines specific to ATMPs is available on EMA’s website.
If tissues and cells are being used as starting materials in a medicinal product, the donation, procurement and testing of the cells are covered by the Tissues and Cells Directive (2004/23/EC). In Estonia, the legislation covering these aspects is based on the abovementioned directive. In line with European Union legislation collection and testing must be done in accordance with an appropriate quality control system for which standards and specifications are set out within the legislation. Tissue establishments supplying such materials are required to have a system in place to trace all substances coming into contact with the cells and tissues while maintaining donor and patient confidentiality.
If blood and blood components are being used as starting materials in a medicinal product, the donation and testing are covered by the Blood Directive 2002/98/EC and by the requirements set in national legislation.
In Estonia, the State Agency of Medicines (SAM) is the supervisory authority:
- For Estonian manufacturers and importers of centrally authorized ATMPs, as well as
- For ATMPs which are prepared and used under the hospital exemption (HE). The HE applies for ATMPs which are prepared on a non-routine basis and are used in a hospital with a prescription for a specific patient.
- Moreover, the SAM is the competent authority for clinical trial authorisation for ATMPs, in the same way as for all other medicines.
If you need advice about getting a manufacturer license from SAM email [email protected]
Last updated: 19.11.2021