학술논문
Defining orphan conditions in the context of the European orphan regulation: challenges and evolution
Document Type
Report
Author
Source
Nature Reviews Drug Discovery. July 2019, Vol. 18 Issue 7, 479
Subject
Language
English
ISSN
1474-1776
Abstract
Author(s): Daniel J. O'Connor [sup.1] , Maria E. Sheean [sup.2] [sup.3] , Matthias P. Hofer [sup.2] , Stelios Tsigkos [sup.2] , Segundo Mariz [sup.2] , Laura Fregonese [sup.2] , Kristina [...]
The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and consequently the eligibility for incentives. Here, based on the experiences of the Committee for Orphan Medicinal Products, we discuss how to define orphan conditions in the context of the European regulatory framework.
The definition and acceptability of an orphan condition is pivotal for the assessment of European orphan medicinal product designation applications, and consequently the eligibility for incentives. Here, based on the experiences of the Committee for Orphan Medicinal Products, we discuss how to define orphan conditions in the context of the European regulatory framework.