The current revision of the orphan medicines regulation in the EU: what is at stake for gene and cell therapy?
Résumé
In the European Union, gene and cell therapy medicinal products are mainly regulated by the specific legal framework applicable to Advanced Therapy Medicinal Products (ATMPs): regulation (EC) n°1394/2007. Yet most ATMPs that have been authorised for marketing in the European Union are also classified as orphan medicinal products. The latter are regulated by a different but also specific legal instrument : Regulation (EC) n°141/2000, which aims to promote the development of innovative medicines for orphan diseases thanks to a set of incentives for investigators. However, after twenty years of implementation, results are still unsatisfactory for several reasons. One of these reasons is that the market of orphan medicinal products had “started to look more similar to ‘standard’ medicines” than innovative medicines like ATMPs. Moreover, it has also been observed that even when marketed, orphan medicinal products fail in being equally accessible to patients in the different Member States. The European Commission has started a long and thorough revision process of the pharmaceutical legislation in order to give a new impulse to the development of, and access to, truly innovative orphan medicinal products, which may have a positive impact for patients as well as for investigators working on gene and cell therapies. In this poster, we will discuss what aspects of the revision of the regulation on orphan medicinal products may have an impact on the development of gene and cell therapy in the European Union.
Domaines
Droit
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