About rare disease
7,000+
known rare diseases
Different organizations recognize different numbers of rare diseases - the most cited number is around 7000, but recent studies report more than 10,000 rare diseases.
Definitions
vary by region
A condition is designated as rare if it affects fewer than...
-
200,000 people in the U.S.
-
5 in 10,000 people in the E.U.
-
1 in 2,000 people in the U.K.
-
50,000 people in Japan
400m people
affected globally
By some estimates, it's as many as 1 in 10 people.
1 in 2 patients is a child. And 30% of children with a rare disease don't reach the age of 5.
First orphan drug approval: 1983
In 1983, the FDA introduced the Orphan Drug Act, to stimulate development of treatments for rare diseases, and granted marketing approval for the first Orphan Drug: Panhematin for acute intermittent porphyria and other acute porphyria. Since then around 100 countries have adopted regulations for rare diseases.
Just 5% have
an FDA-approved treatment
Many rare diseases have no approved treatments available. Across the industry, we recognise an urgent and significant unmet need to accelerate the development of rare disease treatments.