Director QM/RA & Medical Affairs bess AG Berlin, Germany
It has been estimated that there are more than 6000 rare diseases worldwide and despite that each rare disease affects a relatively small number of patients, collectively rare diseases represent a considerable health burden. Patients with rare diseases are entitled to expect the same quality of treatment as other patients with more common disease however, under normal market conditions, the costs of developing medicinal products for rare diseases are not expected to be recovered by the potential sales. Therefore, a regulatory framework exists in order to encourage the research and development of medicinal products for rare diseases, the so called ‘Orphan Drug or Orphan Medicinal Product, that have been released in both: the EU as well as in the USA. Although similarities exist, the criteria and processes for designation are not internationally harmonised and this abstract intends to summarize the Orphan Drug Designation in the European Union (EU) and the United States of America (USA).
Understand the differences between EU and USA regulatory requirements for OMP
Compare the requirements of the EMA and FDA for the OD designation
Conduct analysis and comparison between the Agencies