Why does there need to be a European Regulation about orphan drugs?

Some 20-30 million Europeans are affected by rare diseases. Before the creation of the EU Regulation, there were almost no treatments for these diseases. Since the Regulation, more than 650 applications1 for orphan designations were submitted relating to over 200 different rare conditions. There are now 58 medicinal products (as of July 2009) that have received a positive opinion by the European Medicines Agency (EMEA).

But challenges still remain. Patients still face delays in getting access to these approved treatments and there are still many diseases with no treatments.

1 Summaries of opinion on orphan designation. EMEA website: http://www.emea.europa.eu/htms/human/orphans/opinion.htm (as of 15 September 2009)