The number of orphan medicines in EU has increased significantly since the introduction of the orphan legislation, i.e., from 2000 to 2022. The selection of orphan medicines tends to focus on medicines used for treating cancer and inborn errors of metabolism or immune system disorders. The coverage of orphan medicines is still minor compared to number of orphan diseases and it seems that the orphan medicines market is focused on rare conditions with highest prevalence. The development of orphan medicines for paediatric use has not been proportionate to the prevalence of rare diseases in children. Orphan medicines with marketing authorisation often target diseases or disease groups that already have available treatments, while several rare diseases remain without available treatment.
Tuesday, June 3, 2025
EMA approved orphan medicines since the implementation of the orphan legislation
Hahl, E., Kurko, T., Koskinen, H. et al. EMA approved orphan medicines since the implementation of the orphan legislation. Orphanet J Rare Dis 20, 266 (2025).doi:10.1186/s13023-025-03756-7
