Results
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Availability and access for cell and gene therapies is not universal globally
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A total of 17 cell/gene therapies approved globally and currently available in the US and EU (Figure 1)
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Only 7 cell/gene therapies overlap in both jurisdictions (US/EU)
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Overall, 12 cell and gene therapies are currently authorized by the FDA and marketed
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Overall, 12 are currently authorized by the European Commission and available on the market
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Data above does NOT include ATMPs that have been withdrawn from these markets
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Although the value of these therapies is recognized in France and Italy, this is not the case in Germany
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Only 2 therapies assigned hint of considerable/major added benefit in Germany with majority assigned a non-quantifiable benefit
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60% of therapies evaluated were assigned ASMR II/III in at least one group in France and 70% assigned innovative status in Italy
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Generally, net cost of therapy in Europe following negotiations was 10% to 40% below visible ex-factory price
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Cell/gene therapies with the highest annual visible ex-factory price are:
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US was Rethymic (Allogeneic processed thymus tissue–agdc) at $2.7M
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Europe was Libmeldy (atidarsagene autotemcel) at $3.75M (£2,875,000)
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Neither product is approved by both FDA and EMA presently
Figure 1: Approval status of cell and gene therapies in the US and Europe as of November 2021
* 2 therapies are no longer marketed in the US: Gintuit, Laviv
**7 therapies withdrawn from EU market: Maci, Provenge, Chondrocelect, Glybera, Zalmoxis, Zynteglo, Skysona
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Market access status of cell and gene therapies, ATMPs varies considerably across the six countries (Figure 2)
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US: 100% of the cell and gene therapies are reimbursed under medical benefit
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France: While only 50% of therapies have completed negotiations and received positive reimbursement recommendations, patients do currently have access to an additional 33% undergoing negotiations via the early access pathway (overall 83%)
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Germany: 67% of ATMPs are reimbursed following completion of the AMNOG procedure
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An additional 25% of therapies are currently undergoing negotiations and patients have access given reimbursement is immediate post launch (overall 92%) (Spherox is the exception despite majority being assigned non-quantifiable additional rating)
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Italy: 67% of ATMPs are reimbursed, all with patient registries and several with Payment by Results (PbR) agreements
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Spain: Poorest access to ATMPs in EU4, with only 42% having completed negotiations with a positive recommendation based on protocol, PbR agreement, free drug for certain patients or spending ceilings
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UK: 83% of ATMPs are currently recommended for reimbursement by NICE
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Interestingly only one-third have been assessed via Highly Specialized (HST) Technologies pathway, which seems specifically designed for them
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Data above does NOT include ATMPs that have been withdrawn from these markets
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US: 2 therapies that are no longer marketed (Gintuit and Laviv)
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Europe: 7 therapies have been withdrawn post-approval for clinical/commercial reasons (Maci, Provenge, Chondrocelect, Glybera, Zalmoxis, Zynteglo, Skysona)
Figure 2: Reimbursement status of cell/gene therapies and ATMPs approved and currently available in the US, EU4 and the UK
*All ATMPs currently undergoing negotiations in France are included in the early access scheme
Figure 3: 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