OBJECTIVES: Examine the pricing, reimbursement, and market access (PRMA) landscape for cell and gene therapies/ATMPs across the US and Europe (EU4/UK)

METHODS: Cell and gene therapies/ATMPs approved and available in the US and Europe as of December 2022 were identified from FDA and EMA websites. HTA, pricing and reimbursement data gathered from official national sources as of December 2023 (e.g. Medispan, HAS, GBA, AIFA, NICE).

RESULTS: Market access status of available cell/gene therapies varied considerably among these markets:

• US: 27 FDA-approved therapies (8 are cord blood products), 18 of which are marketed and reimbursed
• Europe: 17 EC-authorized therapies currently available
• US and Europe: 9 therapies are available across both jurisdictions

Cell and gene therapy with highest annual visible ex-factory vary: US was Hemgenix (etranacogene dezaparvovec-drlb) at $3.5M; Europe was Upstaza (eladocagene exuparvove) at $3.8M (Germany)

European net therapy cost following negotiation include ~10% to 45% reductions from the visible ex-factory price with some as high as ~60% (e.g. Roctavian- valoctocogene roxaparvovec, in Germany).

CONCLUSIONS: Cell and gene therapy space is growing with several products now on the market. Visible ex-factory prices continue to set new records raising concerns about affordability and sustainability, while the gulf between ex-factory and net prices seems to be increasing. Growing recognition and acceptance of outcomes-based payment models and real-world evidence generation to facilitate access is another trend in both the US and Europe.

Despite HTA bodies often criticizing the level of evidence provided, the value of cell and gene therapies is recognized (e.g., France has 6 of the 9 evaluated therapies with ASMR II/III and Italy designated 8 of the 12 as being “innovative”). Especially with the planned adoption of the EUnetHTA joint clinical assessments (JCAs) framework for ATMPs by 2024, developments in the evaluation at the European and national levels will need monitoring.