OBJECTIVES

To compare the market access for ATMPs approved by the EMA since 2015 across the EU5 countries focusing on reimbursement trends.

METHODS

A targeted research on official EU5 reimbursement / HTA agencies websites were executed in June 2020.

RESULTS

11 innovative ATMPs were identified with an EMA market authorization (including Zalmoxis which was withdrawn in 2019). The reimbursement and pricing vary between countries as outlined below:

France: 7/11 ATMPs were assessed (vs 6/10 in 2019) and 6 (vs 5 in 2019) obtained a positive reimbursement decision: three (vs 0 last year) have a public price (Yescarta, Kymriah, Alofisel). Luxturna and Zinteglo are available through post-ATU process; Holoclar can be funded by hospitals.

Germany: 7/11 therapies (vs 5/10 in 2019) were assessed within the AMNOG process. Holoclar was taken as a procedure and it can be reimbursed through the DRG-System. Kymriah was the first ever therapy in Germany on which a pay-for-performance deal was agreed.

Italy: 5/11 drugs (vs 2/10 in 2019) are reimbursed to date (Strimvelis, Holoclar, Zalmoxis, Kymriah, Yescarta). 2/11 drugs are under negotiations with AIFA (Luxturna, Zynteglo). Alofisel received a negative reimbursement decision.

Spain: 3/11 therapies (vs 2/10 in 2019) are reimbursed (Kymriah, Yescarta and Alofisel; all of them with a payment by results agreement), Holoclar is commercialized but not reimbursed.

UK: 7/11 drugs (vs 6/10 in 2019) are approved by NICE: Spherox via STA with the two CAR-Ts included in the CDF. Three technologies (Holoclar, Imlygic and Luxturna) were recommended with a simple discount Patient Access Scheme. Two ATMPs, Zynteglo and Zolgensma are currently being evaluated by NICE.

CONCLUSIONS

Most ATMPs are granted patient access in EU5 even though HTA bodies have imposed monitoring requirements to ensure the value reflects the price.