CELL INTELLIGENCE · 肿瘤细胞治疗研究

BACKGROUND: Despite clinical efficacy and cost-effectiveness, time to reimbursement for chimeric antigen receptor (CAR) T-cell therapies varies greatly across Europe. We examined these differences, and quantified potential patient benefits with faster access. METHODS: A targeted literature search collated reimbursement statuses for all approved CAR T-cell therapy indications by 1 October 2024. Time to reimbursement decision (TTRD), defined as time between marketing authorization and published reimbursement decision, was assessed. By benchmarking against the TTRD of the three fastest countries, patient impact, in the form of potential gains in lives, life-years (LYs) and long-term survivors, for the first three approved indications for CAR T-cell therapies were estimated. Each outcome was multiplied by population size, incidence, CAR T-cell eligibility, and market share to obtain population-level impact. Scenario analyses were conducted by examining different TTRD assumptions. RESULTS: Across 12 identified indications for CAR T-cell therapy, France, Germany, and Switzerland had the fastest TTRD, compared to Denmark, the Netherlands, and Ireland which had the slowest. Among an estimated annual incident population of 6,594 across 3 L + diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), and pediatric acute lymphoblastic leukemia (ALL), 1,199 lives, 1,222 long-term survivorships, and 37,229 LYs, could potentially be gained by ensuring time to access across these countries was equivalent to the fastest three assessors in these 3L + DLBCL, MCL, and pediatric ALL. CONCLUSION: Access to CAR T-cell therapies varied widely across European countries. Faster access to these therapies can result in significant health gains. Ensuring prompt and equitable access to innovative therapies requires proactive alignments between manufacturers and HTA bodies, possibly through mechanisms such alternative pricing models and risk-sharing agreements. Chimeric antigen receptor (CAR) T-cell therapies are a one time, highly promising treatment for certain blood cancers, but it is costly and its long term effects are still being studied. These challenges have slowed patients receipt of these therapies. This study examines how long European countries take to approve payment for CAR T cell therapy and estimates how much patients could gain if these decisions were made more quickly. We found that not all countries agreed to pay for CAR T cell therapies. Out of the 12 approved uses for these treatments, Germany, Switzerland, and France covered all of them (100%), while Denmark, Ireland, and the Netherlands covered only three (25%). We also saw big differences in how long countries took to make these decisions: France, Germany, and Switzerland acted the fastest, while Denmark, the Netherlands, and Ireland took much longer. Across 12 European countries, speeding up decisions to pay for CAR T cell therapy in the first three approved diseases could save about 1,200 lives and allow around 1,220 more people to survive their cancer. Patients could also gain roughly 37,000 extra years of life overall. These findings suggest that people across Europe may not have the same chance to receive these innovative treatments. To help patients who urgently need CAR T cell therapy, European countries will need to adjust their policies and funding approaches so the treatment is easier to access, and is fair for all patients.