1. Orphan medicines

1.1. Criteria

To qualify for an orphan designation, a medicine must meet a number of criteria (as per the EMA definition):

  • It must be intended for the treatment, prevention or diagnosis (also referred to as “medical plausibility”) of a disease that is life-threatening or chronically debilitating.
  • The prevalence of the condition in the EU must not be more than 5 in 10.000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development.
  • No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

Applications for orphan designation are examined by the EMA's Committee for Orphan Medicinal Products (COMP), using a network of experts, and it also considers if medicines previously granted orphan designation can continue to be classified as orphan medicines at the time of marketing authorisation and beyond. For Marketing authorisations for designated orphan medicines the centralised procedure is obligatory and applications for marketing authorisation (MAA) go before the Committee for Medicinal Products for Human Use (CHMP). Moreover, MAHs can also apply for an orphan designation for new indications of an already authorised product.