OBJECTIVES

To explore reimbursement pathways and timelines for innovative gene and cell therapies across Europe

METHODS

A targeted literature review was executed to identify the reimbursement pathways and key challenges in gene therapy reimbursement across European countries. National reimbursement agencies databases were reviewed to compile reimbursement decisions and advertised timelines for gene therapies and Advanced Therapy Medicinal Products (ATMPs) up to 31^st of May 2020.

RESULTS

Historically 10 gene therapies have received EU marketing authorisation, with an increasing uptake in positive reimbursement decisions across European country.

The 4 gene therapies authorised by the EMA between 2012-2016, achieved positive reimbursement in between 1-3 European countries, compared to 7-32 countries for the 4 gene therapies authorised during 2017-2018 (currently undergoing further assessments).

A focused review of Gene therapy reimbursement decisions for Germany, UK, France, Italy and Spain show great differences in reimbursement timelines between countries and gene therapies within the same country. Time between marketing authorisation and published reimbursement decisions, ranges between 6 to 78 weeks within the same country.

There are multiple reimbursement pathways for innovative and advanced therapies such as Gene Therapies, including Early Access Programs (EAPs), traditional submission to HTA agencies or reimbursement bodies and specific access schemes or provisions.

CONCLUSIONS

Access to innovative and advanced therapies in Europe can be achieved through differing routes in each country, with an increasing number of countries implementing Early Access Schemes and setting up specific pathways and funding mechanisms for advanced therapies reimbursement. Great variability of schemes and factors affecting reimbursement decisions across countries lead to differences in patient access to gene therapies across Europe.