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EuOrphan
EuOrphan is a database containing regulatory and scientific information on orphan medicines and other medicines available on the market for the diagnosis, prevention and treatment of rare diseases in both the European Union (EU) and the United States (USA) and allows search and access to updated information on their developmental status useful for clinicians, researchers and patients.
It was designed by Consorzio per Valutazioni Biologiche e Farmacologiche in the framework of a funded European IT-Technology project (eTen 510774 2003/C 118/19) and since 2008 it is voluntary managed and regularly updated by Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus.
The database, updated twice a year, represents one of few registries in Europe dedicated to providing the community with information on the available or under development treatments for rare diseases. The information included in EuOrphan allows the descriptive and statistical analyses by age, rare condition, therapeutic area and so on, estimating the coverage of therapeutic needs for rare diseases as well as comparing data between EU and the USA by consulting the FDA database.
Information on orphan medicines
Updates on the new orphan designations and approvals are sourced from the EMA database in Europe, while related Information on the rare conditions is sourced from Orphanet which provide a rare disease nomenclature, essential in improving the visibility of rare diseases in health and research information systems.
For all orphan medicines, information on the active substance designated as ‘orphan’, including the designation date the sponsor and the orphan indication, is collected. Moreover, information on orphan medicines approved for the diagnosis, prevention and treatment of rare diseases, including the trade name, approval date, the ATC (Anatomical Therapeutic Chemical Classification System) code, therapeutic indication and approved ages is gathered.
Active substances designated as ‘orphan’ in Europe
Rare diseases covered by orphan designations
Medicines approved for the EU market for rare diseases
Rare diseases covered by medicines approved for the EU market
Medicines approved for the EU market for the paediatric population
Rare diseases' features
Rare diseases of paediatric interest: 426
Genetic rare diseases: 260
Last update on 14th December 2020
Publications
The analyses conducted with the information included in the EuOrphan database result in several publications that offer an insight on relevant aspects related to the research and development of orphan medicines.
Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs
Giannuzzi V, Landi A, Bosone E, Giannuzzi F, Nicotri s, Torrent-Farnell J, Bonifazi F, Felisi M, Bonifazi D, Ceci A.
Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs.
BMJ Open. 2017 Sep 11;7(9):e017358. doi: 10.1136/bmjopen-2017-017358
Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen
Giannuzzi V, Conte R, Landi A, Ottomano SA, Bonifazi D, Baiardi P, Bonifazi F, Ceci A.
Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen.
Orphanet J Rare Dis.2017 Apr 3;12(1):64
