CELL INTELLIGENCE · 肿瘤细胞治疗研究

Despite its great promise, implementation of CAR-T therapy-a personalized, logistically complex, and expensive treatment-remains challenging, hampering patient access across and within countries. Since 2018, six products have been centrally approved in Europe (i.e., the European Economic Area; EU-approved) for 15 hematologic malignancy indications. To better understand patient access to EU-approved commercial CAR-T therapy, we evaluated the current status in all 30 countries where EU-approval is valid plus the UK, addressing economic, clinical, and organizational aspects, and identifying challenges and strategies for improvement. A two-step approach was used, complementing data from marketing authorization holders (4/4 responded) with country-specific insights from clinical experts obtained via an online survey (30/31 responded). In August 2024, 26% of the 31 countries had no CAR-T products commercially available, 74% 1 product for non-Hodgkin lymphoma and leukemia, and 16% 1 product for multiple myeloma. One-time payment was the most used reimbursement method. Time to access varied significantly, with medians ranging from 0 (France/Germany) to 53 months (Slovakia). The median number of qualified CAR-T centers per 10 million population per country was 5.0 (IQR: 3.0-6.1). In most countries, patient eligibility assessment was decentralized. Costs and logistical complexity were main factors restricting access in countries with and without commercially available products. Proposed solutions included cost reductions, improving reimbursement processes, and increasing healthcare resources. This study shows that patient access to commercial CAR-T therapy in Europe remains limited. Its insights into this multi-faceted problem can guide policy-making, advocacy work, and research to make this transformative treatment accessible to more patients in need.