Definition
Advanced therapy medicinal products comprise of gene therapy, somatic cell therapy and tissue-engineered products. The legal definitions and regulations have been laid down in European pharmaceutical legislation approved in 2001, 2004, 2007 and 2009 (http://ec.europa.eu/health/documents/eudralex/vol-1/index_en.htm).
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.
Advanced therapy medicinal products 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
All advanced therapies medicinal products 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. This 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 and published on the European Medicines Agency website: http://www.ema.europa.eu/htms/human/raguidelines/advanced_therapies.htm
Advanced therapy medicinal products that incorporate human tissues and cells must additionally comply with European Union directives approved in 2003 and 2006. In Estonia, the legislation covering these aspects is based on the abovementioned directives. 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.
In Estonia, the State Agency of Medicines is the supervisory authority for Estonian manufacturers and importers of centrally authorized advanced therapy medicinal products, as well as the competent authority for advanced therapy medicinal products which are prepared and used under the hospital exemption.
Results