Access to Affordable Orphan Medicines in Europe: An EHA Position Paper

Οrphan medicinal products (OMPs) are pharmaceuticals designed for the treatment of rare diseases, a family of more than 6000 medical conditions, each of which affects a small to ultra-small population of patients – typically less than 1 in 2000 people.

The June 2009 European Council Recommendation on action in the field of rare diseases emphasizes that “the principles and overarching values of universality, access to good quality care, equity and solidarity […] are of paramount importance for patients with rare diseases.” Although important advances have been made in the OMP field during the last decade, several deadlocks still impede optimal accessibility.

A survey conducted in 12 Eurasian countries on rare disease policies and orphan drug reimbursement systems showed that inequality in patient access to new OMPs still exists due to variations in national healthcare budgets, health insurance, and reimbursement systems Fragmentation of reimbursement policies translates into unequal access to treatment. The number of reimbursed OMPs ranged from >100 in Germany, France, Italy, and the Netherlands to zero in Armenia