OBJECTIVES:

Examine the current pricing, reimbursement, and market access (PRMA) landscape for regenerative medicines, ATMPs, cell & gene therapies across three quite different jurisdictions US, Europe, and Japan.

METHODS:

Assess HTA, pricing and reimbursement for cell/gene therapies approved and available in the US, Europe, and Japan as of May 2022.

RESULTS:

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

• US: 23 FDA approved therapies (8 are cord blood products),13 of which are marketed and reimbursed
• Europe: 21 EC authorized therapies, 7 of which have been withdrawn for clinical/commercial reasons
• Japan: 14 regenerative medical products are PMDA approved and reimbursed, 4 have conditional/time limited authorizations.
• Only 5 cell/gene therapies (including 4 CAR-Ts and Zolgensma) overlap across these jurisdictions
• Cell/gene therapy with highest annual visible ex-factory vary: US was Rethymic (Allogeneic processed thymus tissue–agdc) at €2.59M; Europe was Libmeldy (atidarsagene autotemcel) at €3.33M; and Japan was Zolgensma (onasemnogene abeparvovec) €1.17 million
• European net therapy cost following negotiation is often 10% to 40% below visible ex-factory price
• Average time to reimbursed access post regulatory approval was 3 weeks in the US, ~15 weeks in Japan and ranged from 32 weeks in Germany to 88 weeks in France.

CONCLUSIONS:

Regenerative medicines markets are still developing as evidenced by the fragmented regulatory and market access landscape. Type of therapy impacts access, with CAR-Ts and gene therapies for rare paediatric conditions more likely to be approved and reimbursed across jurisdictions relative to “tissue engineered therapies.” The value of cell/gene therapies is being recognized (e.g., France has 6 of the 9 evaluated therapies with ASMR II/III and Italy has designated 7 of the 10 as “innovative status”). Visible ex-factory prices of recently introduced cell and gene therapies continue to present new and different challenges to bio/pharmaceutical budgets.