Rare diseases are referred to as “orphan diseases”, and the products intended to treat them as “orphan medicinal products”. In addition to rare diseases, orphan diseases include those for which developing new therapeutic tools is not profitable. Following the international examples of the United States, Australia and Japan, Europe adopted its “Orphan Regulation” on December 16, 1999 (European Commission Regulations EC No. 141/2000 (1) and EC No. 847/2000 (2)).