6th Workshop, Barcelona: “Do Rare Disease Patients Have Real Access to Orphan Drugs in Europe?”
This workshop revealed the true extent of inequalities in access to orphan drugs that rare disease patients face in some EU countries. It helped raise awareness of the factors and stakeholders that influence the fate of orphan drugs after their centralised marketing authorisation at the EU level. Full transparency in this area and an open dialogue between all the actors are the only way to ensure that the orphan drug field becomes fully recognised as a priority in all of Europe and is sustainable and compatible with the different MS health systems in the long-term. Several avenues to improve orphan drug availability to patients were discussed.
Key feature: the EURORDIS 4th European survey on access to Orphan Drugs was presented for the first time.