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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

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

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