. 2023 May 13;23:484. doi: 10.1186/s12913-023-09494-5

Table 6.

Outcome-based managed entry agreements for gene therapies in France, Germany, Italy and Spain

Gene & Cell therapies	France	Germany	Italy	Spain
Strimvelis	-	-	Performance-linked-reimbursement (through Drug Registry)^a	-
Kymriah	The Transparency Committee recommended to collect further data through the Lymphoma Academic Research Organization Registry: effectiveness (survival, remission status, disease progression) and adverse events, to be recorded at 28 days, 100 days, 6 months and every subsequent 6 months after injection^b	Rebates to the health insurers for patients dying after treatment (time horizon for measuring survival, and the magnitude of the rebate not available in public dominion)^b	Outcome-based staged payments. Installments: infusion, 6 months, 12 months Outcome indicator: unknown (confidentiality agreement)^a	Outcome-based staged payments. Installments: time of treatment (52%), 18 months (48%) Outcome indicator: complete response to the treatment^b
Yescarta			Outcome-based staged payments. Installments: 180, 270, 365 days after infusion Outcome indicator: unknown (confidentiality agreement)^a	Outcome-based staged payments. Installments: time of treatment (52%), 18 months (48%) Outcome indicator: survival^b
Luxturna	-	-	-	Price agreement with additional data collection^a
Zolgensma	-	Mandatory collection of long-term RWD until 2027^a	Outcome-based staged payments. Installments: 0, 1, 2, 3, 4 years Outcome indicator: unknown (confidentiality agreement)^a	Price agreement with additional data collection^a

RWD real-world data

Sources:

^a ATMP Forum [55]: Quarto Report italiano sulle Advanced Therapy Medicinal Product (https://www.atmpforum.com/report/)

^b Jørgensen et al. [13]; Jørgensen et al.[12]