OBJECTIVES: To examine the current pricing, reimbursement and market access (PRMA) landscape for cell and gene therapies in the US and Europe

METHODS: Assessed HTA, pricing and reimbursement for cell/gene therapies on the market in the US and Europe as of November 2021.

RESULTS: Cell/gene therapies approved as of November 2021

• US: 14 authorized by the FDA, 12 of which are marketed and 9 are reimbursed
• Europe: 19 authorized by the EC, of which 6 therapies have been withdrawn for clinical/commercial reasons
• Only 7 cell/gene therapies overlap in both jurisdictions (US/EU)
• Cell/gene therapy with highest annual visible ex-factory in the US was Rethymic (Allogeneic processed thymus tissue–agdc) at $2.7M in and in Europe was Libmeldy (atidarsagene autotemcel) at $3.24M
• Generally, net cost of therapy in Europe following negotiations was 10% to 40% below visible ex-factory price
• Average time to reimbursed access post regulatory approval was 3 weeks in the US and ranged from 32 weeks in Germany to 88 weeks in France
• Market access status of available cell/gene therapies varied considerably in the US and Europe

CONCLUSIONS: Market access of cell/gene therapies remains challenging particularly in Europe. There is a huge variation in access with Germany leading the way with largest number of reimbursed therapies (77%) and Spain lagging behind (only 38%). Value of cell/gene therapies is being recognized in France as evidence by 6 of the 9 evaluated therapies being assigned ASMR II/III and 5 of the 8 assessed therapies in Italy being assigned innovative status. Early access opportunities are more favourable in France vs. Italy. Gross to net differences in Europe are becoming increasingly important as are different managed entry agreements. Payment models and their adaptation will continue to evolve, as more cell/gene therapies are launched, bringing important health benefits, but also substantially impacting pharmaceutical budgets.