Orphan Drug Designation

Orphan drugs are medicinal products intended for the diagnosis, prevention or treatment of rare diseases, which are diseases affecting less than 1 in 2,000 persons or a maximum of 250,000 citizens in the European Union.
(source: Eurodis)

The Orphan Designation is a legal procedure that allows for the designation of a medicinal substance with therapeutic potential for a rare disease, before its first administration in humans or during its clinical development.
(source: Orphanet)

Designation of orphan Drug is following Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal product.

Clinical Consulting deals with the submission to EMA and takes care of all the following procedures. We will help you to review the application regarding required criteria to obtain positive opinion of EMA and COMP:

  • if the drug is intended to the treatment, prevent or diagnose a disease that is life-threatening or chronically debilitating;

  • if the prevalence of the condition in the EU is not be more than 5 in 10,000 or it is unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;

  • if 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.