Evaluation of the legislation on medicines for children and rare diseases
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