Reimbursement Mechanisms Used by CAR-T Therapies in the EU5 (2018–2023)

OBJECTIVES: The clinical effectiveness of advanced therapy medicinal products (ATMP) is often uncertain due to the small disease populations and novel trial designs used in their development. Uncertainty in clinical effectiveness, along with their high price, introduces doubt in ATMPs’ cost-effectiveness and a reluctance to reimburse at the desired price or anticipated patient population. Market entry of ATMPs is likely to accelerate, increasing the attractiveness of risk-sharing reimbursement mechanisms. Our study assesses the reimbursement mechanisms of the six chimeric antigen receptor T-cell (CAR-T) therapies in the EU5, to understand reimbursement options used for ATMPs across these markets.

METHODS: Submission and reimbursement information was extracted from relevant health technology assessment (HTA) agencies, government websites, press releases and news articles over the period January 2018–December 2023. Where possible, we also investigated access agreement terms used, such as clinical performance criteria; payment structures used in performance-based reimbursement (PBR); and endpoints collected by national registries in managed entry agreements (MEA).

RESULTS: Thirty-two HTA submissions, of which 25 (78%) were reimbursed, were identified. The reimbursement mechanisms utilized were as follows: MEA, n=13; PBR, n=8; standard market entry, n=4. Reimbursement through MEAs was most common in France (n=6), followed by the UK (n=3) and Italy (n=2). Four (50%) of the PBRs were in Spain, and 2 each (25%) in Germany and Italy. All 4 instances of standard market entry were in Germany. Access agreement terms stipulated continued data collection via national registries (n=11) and payment structures based on clinical performance criteria (n=6).

CONCLUSIONS: Overall, although use of risk-sharing mechanisms was observed, there were more instances of standard market entry than anticipated. These results suggest that while ATMPs can leverage a range of reimbursement mechanisms, a standardized approach is lacking.