Rare diseases are a major child health issue.
Although each rare disease is rare, as a group, they are common. Up to 10% of the population may have a rare disease. Rare diseases are often associated with pain, disability, uncertainty and early death. New information suggests that more than half of rare diseases start in childhood. This creates a large burden on children and their families very early in life.
To improve health and social outcomes for children and families living with rare disease, Governments must recognise that there is an unmet need. This need must be addressed with positive policy that allows the burden of rare disease to be recognised, and the best interests of these children to be met.