OBJECTIVES: Identify pricing and decisions and reimbursement payment models for Gene Therapies across the EU-5

METHODS: A targeted literature review was used to identify current and past pricing decisions and reimbursement models for gene therapy in Germany, UK, France, Italy and Spain. Published information on official drug catalogues and tariffs for the selected countries was evaluated up to the 31^st May 2020. Analysis was performed to determine gene therapy analogue pricing between therapies for a model country.

RESULTS: Gene Therapy products are setting new standards in pricing strategies in Europe. Price constraints are unique for these innovative therapies, that require high upfront investments and often target limited patient populations. A targeted overview of pricing decisions for gene therapies across selected European countries provides insight into the pricing and reimbursement landscape and factors impacting willingness to pay and reimbursed price potential.

Gene therapies have a relatively uniform or flat-list price across European countries, with list price differences ranging between 0-15% across EU-5 countries for the same gene therapies.

Modelling of gene therapy analogue pricing for all therapies that achieved an EMA marketing authorisation shows no strong relationship between the gene therapy’s administration frequency (one-time treatment vs multiple administration), availability of other therapeutic options or indication’s prevalence, and the therapy’s price point.

Type of payment model varies between countries for the same gene therapy, including retroactive price rebates, staged payments linked to individual patient outcomes, deferred yearly instalment payments or reimbursement on the condition of collecting additional data.

CONCLUSIONS: As affordability and budget impact considerations arise for the funding of breakthrough novel therapies, performance-based agreements and other risk-sharing financial mechanisms are the preferred option for gene therapy reimbursement across Europe. Pricing and reimbursement decisions for gene therapies rely on a combination of different tools with an inconsistent approach across European countries.