Évaluation des CAR-Tcells par la Commission de la transparence de la Haute Autorité de santé (2018–23)

Abstract

CAR-T cells are genetically modified T lymphocytes expressing a chimeric antigen receptor (CAR), delivered via gene transfer vectors to the patient's T lymphocytes.Six products based on CAR-T cells in the field of hematological malignancies have been evaluated by the Transparency Commission (CT) of the French National Authority of Health (HAS) since 2018 corresponding to 11 common law opinions. All of these reimbursement requests received a favorable opinion with significant medical benefit (SMR). Between 2018 and 2021, despite the lack of comparative data, the CT has recognized progress by granting level III or IV added value (ASMR). As of 2021, the majority of CAR-T cells have got ASMR level V, reflecting high uncertainty about the relative effectiveness of these therapies. More recently, the CT was able to evaluate CAR-T cells presenting robust phase III data which lead to levels III to IV ASMR. Finally, since the implementation of the early access reform on July 1, 2021, HAS has authorized all the products evaluated.The evaluation of CAR-T cells continues in real-life, in particular through the French DESCAR-T registry. It remains essential to have randomized controlled trials that make it possible to overcome selection bias and assess the real added value of these therapies.