Naslov
Defining orphan conditions in the context of the European orphan regulation: challenges and evolution

Autori
O’Connor, Daniel J. ; Sheean, Maria E. ; Hofer, Matthias P. ; Tsigkos, Stelios ; Mariz, Segundo ; Fregonese, Laura ; Larsson, Kristina ; Hivert, Virginie ; Westermark, Kerstin ; Naumann-Winter, Frauke ; Stoyanova-Beninska, Violeta ; Barišić, Ingeborg ; Capovilla, Giuseppe ; Magrelli, Armando ; Sepodes, Bruno

Izvornik
Nature Reviews Drug Discovery (1474-1776) 18 (2018), 7; 479-480

Vrsta, podvrsta i kategorija rada
Radovi u časopisima, pregledni rad, stručni

Ključne riječi
Orphan drugs ; orphan conditions ; European Comission Regulation

Sažetak
The European orphan regulation incentivizes the development of medicines for conditions for which the pharmaceutical industry would be unwilling to develop medicines under normal market conditions. Defining an orphan condition is the starting point of every orphan designation, and it is also the entity used to determine prevalence, which must not be more than 5 in 10, 000 for orphan designation to be permitted. In practice, most orphan conditions can be easily identified ; recognized distinct medical entities would generally be considered as valid conditions, defined in terms of their specific characteristics . However, in certain areas, there may be challenges in defining orphan conditions such that a condition is in keeping with the regulatory framework and consistent across all applications that are presented to the European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products (COMP).

Izvorni jezik
Engleski

POVEZANOST RADA