EMA: Zeldzame ziekten, weesgeneesmiddelen – ‘Getting the facts straight’
In support of Rare Disease Day, the EMA has published a question-and-answer document addressing common misunderstandings about the meaning of orphan designation and other aspects pertaining to orphan medicines.
This document explains key concepts in the regulation of medicines for rare diseases such as orphan designation and significant benefit. The Q&A document is available by clicking here.
For more information about orphan designation please click here.