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

Abstract

O rphan 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 patientstypically less than 1 in 2000 people. 1 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. 2 Disparities in the availability of OMPs in the EUA 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. 3 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. For instance, France reimburses 116 orphan drugs, England 68, Scotland 55, and Wales 47. A survey of access to OMPs in the United Kingdom, France, Germany, Italy, and Spain found that since the implementation of the OMP Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorization in the European Union. 4 These OMPs are most widely accessible in Germany and France, while in the other countries between 30 and 60% of OMPs are reimbursed. In addition, in some countries, such as Italy, differences in treatment availability and access to medicines, diagnosis, and care can occur regionally, depending on each regional health management system. The cost of OMPs is clearly a major factor explaining the disparities among different European countries. 5 The complex issue of drug pricing is dealt with in an accompanying European Hematology Association (EHA) position paper, 6 but here we will briefly consider the issues related to OMP prices. In addition, differences in normative approaches to rare diseases and OMPs between countries also play an important role.

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