CAR-T cell therapies: patient access and affordability solutions
Gonçalves E
Chimeric Antigen Receptor (CAR)-T cell therapies, as potentially curative treatments, are a group of immunotherapy agents that are changing the paradigm for the treatment of hematologic malignancies. Ongoing research on CAR-T cell therapy is expected to expand the currently approved indications, which, given the high prices of these innovative therapeutic solutions, will increase the pressure on the sustainability of health systems, enhancing the need to establish adjusted financial solutions and promote the implementation of post-marketing monitoring procedures. This study examines the specific challenges in the development of robust clinical evidence to support the value measurement and cost-effectiveness assessment of CAR-T cell therapies and in the selection of adequate financing solutions. Managed Entry Agreements, which create mechanisms in which the risk associated with the uncertainty in long-term outcomes of these therapies is shared between the manufacturer and the payer, have emerged as preferred solutions in several European Union countries. The access barriers to CAR-T cell therapies are described, and recommendations on potential solutions to address affordability concerns using a framework of a life cycle approach to value assessment involving different stakeholders and adapted financing tools are proposed.
Future science OA, 2025-03-31