The EU legal framework for medicines for human use is intended to ensure a high level of public health protection and to promote the functioning of the internal market, and includes measures that encourage innovation. Medicines need an authorisation before they can be marketed in the EU. This can be a national or an EU authorisation.
A rare or orphan disease has been defined in the EU as a disease that affects no more than five in 10,000 people. A study to support the evaluation shall also take into account the links between the areas of orphan and paediatric medicines, as a considerable number of paediatric diseases also qualify as a rare disease.
This consultation concerns both medicines for rare diseases and paediatric diseases that qualify as rare. Other medicines treating diseases that do not qualify as “rare” are out of the scope of this consultation.
