EMA approved orphan medicines since the implementation of the orphan legislation
The number of orphan medicines available within EU increased significantly after the orphan legislation came into force. The development of orphan medicines seemed to often focus on diseases or disease groups that already have available treatment options, while several rare diseases remain without available treatment. Even though rare diseases are more common in children, orphan medicines have not been developed for paediatric use in the same proportion.
Authors
- Hanna Koskinen (ORCID: https://orcid.org/0000-0002-8001-3076)
- Kati Sarnola (ORCID: https://orcid.org/0000-0003-1300-7482)
- Marja Airaksinen (ORCID: https://orcid.org/0000-0002-6077-5671)
- Terhi Kurko (ORCID: https://orcid.org/0009-0004-3672-6628)
- Eveliina Hahl
Institutions
- University of Helsinki (FI)
- Social Insurance Institution (FI)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-06-02
- DOI
- https://doi.org/10.1186/s13023-025-03756-7
- Citations
- 3
- Primary Topic
- Pharmaceutical studies and practices
- Type
- article
- Field-Weighted Citation Impact
- 10.48
Funders
- Helsingin Yliopisto