OBJECTIVES: The aim is to evaluate the current status of market access of already registered gene therapy products (GTP) in the European Union (EU) including products in the process of evaluation and possible future products. Base on that their availability in the EU and especially possibilities for market access (MA) in Slovakia were explored.

METHODS: The status of already registered GTP was searched on the EMA website; their state of reimbursement and availability for patients was searched on the websites of national agencies in the EU. Possible future registered drugs, in Phase 2 or 3 of clinical research were scanned on standard databases Tripdatabase, EMBASE, MEDLINE, Cochrane Library, EMA, EU Clinical Trials Register and WoS based on information from conferences and ASGCT. Additionally, data from FDA were extracted to see differences between USA and EU in terms of MA status.

RESULTS: CONCLUSIONS: Market access for GTPs is very limited yet. Existed reimbursement is based on managed access schemes. Nevertheless, recently presented results from Phase II GTP used for the treatment of several diseases e.g. Wiskott-Aldrich syndrome, Spinal muscular atrophy, Haemophilia A make an urgency to be prepared for this advanced treatment of patients suffering by orphan diseases.