Th144 - The Hospital Exemption Is the Best European Pathway Towards the Final Authorization of an Academic Proposal in CAR-T Therapy

Program CAR-T & Advanced Therapies – Additional departments (mainly Pharmacology and research units) and near 300 involved people in the involved centers – Hospital Clínic de Barcelona; Julio Delgado – Hematology – Hospital Clínic de Barcelona; E. azucena Gonzalez-Navarro – Immunology – Hospital Clínic de Barcelona; Susana Rives – Hematology – Hospital Sant Joan de Deu de Barcelona; Alvaro Urbano – Hematology – Hospital Clínic de Barcelona

Abstract Text: By now, authorization for the use of cell and gene immunotherapies is just possible through pharma companies and conventional commercialization after fulll market authorization by general drug agencies (FDA in US and EMA in Europe). In this presentation we want to share our experience in a hospital of Barcelona, centred in the obtention of authorization based in the "hospital exemption" rule as an exemple of a middle step for arrive to authorize academic products. At least in Europe and for autologous procedures like the present for CAR-T immunotherapies, we want to present why this proposal can be considered the best solution for Public European Health Systems towards the final authorization of an academic proposal, providing global and future access to patients. By now Varnimcabtagene autoleucel (ARI-0001) and cesnicabtagene autoleucel (ARI-0002h) are the 2 first exemples that allowed us to treat more than 250 patients. ARI-0001 is also under evaluation of EMA thanks to prime designation of this product. Access, substainability and development of new options (mainly done by academic centers) should be the main aspects to consider and improve when we want consider new advanced cell-gene therapies.