CAR-T 细胞治疗：患者可及性与可负担性解决方案

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.

Chimeric Antigen Receptor (CAR)-T cell therapies, classified as Advanced Therapy Medicinal Products (ATMP) in the European Union, offer a potentially curative approach for treating hematologic malignancies. The development of these therapies, however, faces several health economic challenges, including the collection of clinical evidence, the selection of health economic data, and health technology assessment methodological issues.

The specific challenges in the selection of appropriate financing solutions to allow access to CAR-T cell therapies may be addressed through solutions such as managed entry agreements, supported by the collection of real-world data on the performance of CAR-T cell therapies in clinical practice. A holistic approach to the health economic assessment, involving collaboration among various stakeholders, is key to optimizing patient access to CAR-T cell therapies, while ensuring payer affordability.