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PRMA Trends for Cell & Gene Therapies and ATMPs in the US and Europe

Executive Summary
The scientific poster showcases results from a review of pricing, reimbursement, and market access trends for all cell/gene therapies and ATMPs approved as of November 2021 and available in the US, EU4 (France, Germany, Italy, Spain) and the UK as of April 2022. The poster was presented by Indegene at ISPOR 2022.

Mycka J

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Dalal N

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Dellamano R

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Pollere D

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Ashton A

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Objective
To examine the pricing, reimbursement and market access (PRMA) landscape for cell and gene therapies and ATMPs approved in the US and Europe
Methods
Reviewed all cell/gene therapies and ATMPs approved as of November 2021 and available in the US, EU4 (France, Germany, Italy, Spain) and the UK as of April 2022
Also noted products that have been withdrawn
Analysed HTA, pricing, reimbursement status and time to market
Data gathered from EMA, national Health Technology Assessment (HTA) agencies and Pricing and Reimbursement (P&R) bodies
Sources for launch date and HTA information provided in Table 1 below
Table 1: Sources for HTA, pricing, reimbursement & time to market
Results
Availability and access for cell and gene therapies is not universal globally
A total of 17 cell/gene therapies approved globally and currently available in the US and EU (Figure 1)
Only 7 cell/gene therapies overlap in both jurisdictions (US/EU)
Overall, 12 cell and gene therapies are currently authorized by the FDA and marketed
Overall, 12 are currently authorized by the European Commission and available on the market
Data above does NOT include ATMPs that have been withdrawn from these markets
Although the value of these therapies is recognized in France and Italy, this is not the case in Germany
Only 2 therapies assigned hint of considerable/major added benefit in Germany with majority assigned a non-quantifiable benefit
60% of therapies evaluated were assigned ASMR II/III in at least one group in France and 70% assigned innovative status in Italy
Generally, net cost of therapy in Europe following negotiations was 10% to 40% below visible ex-factory price
Cell/gene therapies with the highest annual visible ex-factory price are:
US was Rethymic (Allogeneic processed thymus tissue–agdc) at $2.7M
Europe was Libmeldy (atidarsagene autotemcel) at $3.75M (£2,875,000)
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
Market access status of cell and gene therapies, ATMPs varies considerably across the six countries (Figure 2)
US: 100% of the cell and gene therapies are reimbursed under medical benefit
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%)
Germany: 67% of ATMPs are reimbursed following completion of the AMNOG procedure
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)
Italy: 67% of ATMPs are reimbursed, all with patient registries and several with Payment by Results (PbR) agreements
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
UK: 83% of ATMPs are currently recommended for reimbursement by NICE
Interestingly only one-third have been assessed via Highly Specialized (HST) Technologies pathway, which seems specifically designed for them
Data above does NOT include ATMPs that have been withdrawn from these markets
US: 2 therapies that are no longer marketed (Gintuit and Laviv)
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
Conclusions
Cell/gene therapy and ATMP markets are still developing as evidenced by the fragmented regulatory and market access landscape
Regulatory approval generally tends to be consistent across the US and EU, however this is not the case for cell/gene therapies and ATMPs
For manufacturers, this isn't a traditional market as evidenced by the heterogeneity in regulatory environment, HTA, acceptable prices and market access pathway(s)
Market access remains challenging particularly in Europe where 7 therapies have been withdrawn for clinical/commercial reasons
Within Europe, there is a huge variation in access with UK and Germany leading the way with largest number of reimbursed therapies and Spain lagging behind, although this has improved over the past year
Type of therapy also impacts access with CAR-Ts and gene therapies for rare pediatric conditions more likely to be reimbursed, at least in Europe, relative to 'tissue engineered therapies'
Visible ex-factory prices of recently introduced cell and gene therapies are still setting new highs; however, contracting (in the US) and confidential discounts / managed access agreements (in Europe) often reduce net prices
In general, gross to net differences in Europe are becoming increasingly important, with overall discounts for reimbursed cell and gene therapies ranging from 10% to 40%
Gross to net differences may grow in future, especially within more "competitive" categories (e.g. CAR-Ts,) and also as a consequence of gradual expansion into earlier lines of treatment / broader indications
Innovative approaches to market access will continue to evolve, as more cell/gene therapies and ATMPs are launched, bringing important health benefits, but also with substantial potential impact on pharmaceutical budgets

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