OBJECTIVES: Since 2018, six CAR-T cell therapies have obtained EU and UK marketing authorization. This research examines HTA body evaluations of the main clinical package and its impact on HTA ratings, where applicable, and reimbursement status on the six commercialized CAR-Ts in blood cancers in EU-4 and England.

METHODS: We conducted primary research with 12 payers / proxies with advanced therapy expertise from France, Germany, Spain, and Italy and England. This was complemented with findings from AccessCGT, Research Partnership’s cell and gene therapy analogue access and policy repository, based on publicly available HTA reports and other official Pricing & Reimbursement (P&R) documents.

RESULTS: As of June 2023, France had conducted HTA assessments for all six CAR-Ts; Germany had evaluated five; England, Italy and Spain had published evaluations for three. From evaluations conducted, negative reimbursements emerge only in France and Spain, largely single arm trial (SAT)-driven. French and Spanish payers surveyed strongly agreed on the difficulty of making P&R decisions based on SATs. SATs have also been raised as key HTA body criticisms in England and Italy; however, concerns have been mitigated with follow-up clinical trials, submission of real-world evidence (RWE) and inclusion in the mandatory AIFA registry. Although lack of comparative data prevented CAR-Ts from securing a quantifiable additional benefit in Germany, successful reimbursement outcomes have been attained. Surveyed payers in Germany confirmed previous calls from the G-BA that CGTs need controlled comparative studies to improve on the current non-quantifiable benefit as not every evidence gap can be bridged with indirect comparisons or RWE.

CONCLUSIONS: HTA bodies have made few adaptations to assessment frameworks to accommodate CAR-T specificities. As some CAR-Ts are now already reimbursed, payers expect HTA bodies to become more demanding about the level of evidence package to include robust comparative data to achieve favourable P&R outcomes.