Table 6 Outcome-based managed entry agreements for gene therapies in France, Germany, Italy and 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