OR15

Advanced therapy medicinal products, and CAR-T cells in particular, have revolutionised the treatment of haematological malignancies (acute lymphoblastic leukaemia, non-Hodgkin's B lymphoma and multiple myeloma). CAR-T cells are considered as ex-vivo gene therapy drugs. Since 2018, six CAR-T cells have been granted marketing authorisation in Europe.

The CAR-T cell manufacturing process requires scientific and technical skills to meet the acceptance criteria of pharmaceutical quality system. The most commonly used method for genetically modifying T lymphocytes is viral transduction, which requires prior access to a viral vector with Good Manufacturing Practice grade. Because of its high cost, this reagent is the most limiting factor for developing academic CAR-T cells. However, a CAR-T cell produced by a drug company is currently expensive (around € 350,000 per infusion) and the delivery to clinicians can take between three and five weeks.

The regulatory feasibility of production of CAR-T cells by the cell and gene therapy units of healthcare establishments was assessed and confirmed. The conditions for setting up such production by these establishments were then defined. Finally, the specific contribution of these structures for such preparations was discussed, showing in conclusion that such academic production would enable patients who are not eligible for authorised CAR-T cells and whose disease progression allows them to receive these treatments. Public-private partnerships and guidance from the regulatory authorities could represent the best strategy to support the growing demand for CAR-T cells while preserving the durabilitý of academic research in this area.