A rare 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.
With this consultation, the European Commission asks private citizens and healthcare professionals to share their experiences with and perspectives on access to orphan medicines in general, and on the role the EU Orphan Regulation plays in the development of
orphan medicines. More information you can find here.