1,000th designation of an orphan medicinal product in EU

According to a European Commission statement, over 30 million European citizens suffer from a rare disease. The small numbers of patients affected by a single disease and the fragmentation of knowledge about rare diseases translates into difficulties for people to get the right diagnosis, the right medical advise and the right medicine.

In many cases, the medicine they need has not yet been developed. Therefore, the Commission stated last week: “Today's 1,000th designation of an orphan medicinal product marks a tremendous success in improving this situation.”

Orphan medicines relate to diseases which affect not more than five persons in 10,000 in the European Union. As a consequence, the economic potential of such medicines is very limited which hampers the development of new medicines.