BRAINS FOR BRAIN Onlus Foundation (B4B)

New EMEA designation

understand-your-diagnosis_02The definition “Orphan drug” is commonly applied to a medicinal product intended to treat a disease so rare that sponsors are reluctant to develop them under usual marketing conditions.




Reluctancy is due to the fact that:

  • The process from the discovery of a new molecule to its marketing is long (10 years in average);
  • drugs for rare diseases are almost never cost effective (development cost several tens of millions of euros);
  • success is not guaranteed (among ten molecules tested, only one may have a therapeutic effect).

Although all these uncertainties about the benefits of therapy relative to the costs incurred in research and development to produce it, Pharmaceutical Industry continues to invest on orphan drugs R&D, not because of economic reasons but to respond to public health needs.

The European Medicines Agency EMEA is a decentralised agency of the European Union responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union. Please vist EMA website for more information.