The EU defines diseases with a prevalence of ≤5:10,000 individuals as rare diseases (RD). Approximately 7,000 of the circa 30,000 known diseases are therefore classified as RD. This large number means that roughly 30 million people in Europe and an approximate 4 million individuals in Germany are affected, of which many have no definitive diagnosis. More than 80% of RDs are genetic and therefore result in life-long illnesses. The often-late diagnosis and lack of a treatment contribute to severe courses of the diseases. Small patient numbers make the development of pharmaceutics unprofitable.