The view of experts on initiatives to be undertaken to promote equity in the access to orphan drugs and specialised care for rare diseases in Spain: A Delphi consensus

Rare diseases are an important public health issue and a challenge for the medical community, not only due to the clinical and therapeutic complexity they represent, but also because of the lack of knowledge regarding their natural course, which jeopardises prompt diagnosis and the anticipation of serious risks with undesirable outcomes [1]. Although rare diseases affect a limited number of persons, defined in the European Union as 1 in 2000 individuals or less [2], according to the World Health Organisation, between 5000 and 8000 disorders are defined as “rare” [3].
Source: Health Policy - Category: Health Management Authors: Source Type: research