An analysis of orphan medicine expenditure in Europe: is it sustainable?

Jorge Mestre-Ferrandiz, Christina Palaska, Tom Kelly, Adam Hutchings & Adam Parnaby; Orphanet J Rare Dis 14, 287 (2019) doi:10.1186/s13023-019-1246-7

Orphan medicinal product (OMP) prices are considered by some to be a challenge to the sustainability of healthcare expenditure. These concerns are compounded by the increasing number of OMPs receiving marketing authorisation (MA) annually. The aim of this study was to explore the sustainability of OMP expenditure within the context of total European pharmaceutical expenditure.
The EU regulation on OMPs has had success in fostering R&D addressing unmet needs for treatments for rare diseases, with approximately 150 OMPs being approved since its introduction. However, despite the success of the regulation in supporting the development of medicines for rare diseases, the findings from this analysis suggest that the resultant impact on OMP expenditure could be deemed sustainable when seen in the context of total pharmaceutical expenditure.


An analysis of orphan medicine expenditure in Europe: is it sustainable?