Valuing Chimeric Antigen Receptor T-Cell Therapy: Current Evidence, Uncertainties, and Payment Implications
Menées dans un contexte américain, ces différentes études abordent les aspects économiques et financiers liés au cancer et à ses traitements, qu'il s'agisse de l'impact de la maladie sur l'emploi des personnes atteintes, des stratégies pour réduire les coûts des médicaments, du coût des programmes de prévention ou de dépistage, des difficultés financières des patients ayant survécu à la maladie, etc.
Chimeric antigen receptor T-cell (CAR-T) therapy is a novel immunotherapy that uses cells from an individual’s immune system to fight cancer.1 A patient’s T cells are extracted from the blood and are engineered to express a chimeric antigen receptor. These engineered T cells are then infused back into the patient’s bloodstream, where the receptor binds to and kills cancerous cells. Tisagenlecleucel was the first CAR-T to receive approval in the United States, gaining approval in August 2017 to treat pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL).1 The approval of tisagenlecleucel was historic because it was the first gene therapy available in the United States and initiated “a new frontier in medial innovation,”1 giving hope to patients who had exhausted all other available medical treatments. The approval of tisagenlecleucel was also historic because it was priced at $475,000 for a single infusion, placing it among the most expensive cancer treatments to date and igniting conversations around the world related to value, affordability, and the allocation of finite health care resources.2 History continued to be made when the manufacturer of tisagenlecleucel announced a since-cancelled collaboration with the Centers for Medicare and Medicaid Services (CMS) for an outcomes-based payment agreement, where payment would only be required if a patient responded to tisagenlecleucel within 1 month of infusion.3 Tisagenlecleucel has initiated not only new frontiers in medical innovation, but also new frontiers in valuing high-priced medical treatments and innovative payment mechanisms.
Journal of Clinical Oncology , résumé, 2018