3. Special cases in marketing authorisations: Orphan Medicines

The aim of the orphan medicine legislation is to stimulate research and development of medicines for rare diseases (in short 'orphan medicines') to ensure access to treatment for patients. The European Union’s orphan legislation has been designed to help overcome the extra hurdles these medicines face to get on the market and to provide an incentive for companies to invest in the development of respective medicines despite the low number of patients and therefore the limited market.