Orphan Drugs

In response to an EU Consultation on the future of the single market in pharmaceuticals for human use, Eurordis has put forward recommendations specifically focused on orphan drugs. The Contribution document proposes measures to:

  • assess therapeutic added value and to establish reference pricing on a global level;
  • upgrade incentives on a national and EU level;
  • continue support for research and development of orphan drugs.


These measures aim at ensuring transparency and equality of access to orphan drugs across the EU and beyond.

The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases