The European Alliance for Transformative Therapies was created in June 2020 as an informal interest group to foster effective dialogue around gene and cell therapies and provide evidence-based policy recommendations.
The European Alliance for Transformative Therapies was officially launched on 8 December 2020 during the online event “Preparing Europe for Novel Cell and Gene Therapies. The aim of this Alliance”, supported by EUCOPE (the European Confederation of Pharmaceutical Entrepreneurs) and BioMarin, is to put forward a constructive dialogue around gene and cell therapies, providing evidence-based policy recommendations.
EPTRI endorsed the Consensus Statement of the European Alliance for Transformative Therapies, developed to underline the importance, opportunities and challenges that can be derived from cell and gene therapies field. Genetic disorders and congenital abnormalities in the paediatric population are the cause of up to 30% of paediatric hospital admissions in industrialised countries and often the only way to realise a better life for these patients is represented by advanced therapies. Clenching the relevance of advanced therapies for genetic disorders and congenital abnormalities in the paediatric population, for EPTRI is important to guarantee innovative and advanced medicines and therapies for children suffering from these diseases.
This statement includes a Call to Action with the aim to underline how Eu instruments and initiatives related to the Pharmaceutical Strategy should promote these innovative and transformative therapies.