Since 2007 and Directive n°1394/2007, ATMPs are considered as a specific class of medicinal products in Europe. Indeed, some products, initially considered by French texts as “cell therapy preparations” or “gene therapy preparations” have changed status to become “advanced therapy medicinal products”.
This change in status has had an impact on regulation at several levels, both for the products themselves and for the authorizations that establishments wishing to produce them must obtain. This is due in particular to the evolution of manufacturing standards and references, which are moving from good tissue/cell practices to good manufacturing practices (GMP) applicable to medicinal products.
Furthermore, their distribution in France in the context of clinical trials, given the pharmaceutical monopoly (L4211-1 Part IV Book 2 of the Public Health Code), is now exclusively carried out by the internal pharmacies of health centres (PUI, for pharmacie à usage intérieur).
As ATMPs are covered by European Regulation 1394/2007 and Directive 2001/83/EC (on medicinal products for human use), their marketing is managed at the European level. According to the French National Agency for Medicines and Health Products (ANSM) and the European Medicines Agency (EMA) guidelines, a medicinal product is considered to be an advanced therapy if it undergoes substantial modifications and/or if its essential function is not the same in the donor and the recipient.
A modification is considered substantial if it leads to “a change in the initial biological properties of the cells or tissues” according to Annex I of Directive 2001/83/EC.