Table 1 Orphan designation criteria at EMA and FDA

European Medicines Agency

Food and Drug Administration

(i) The medicine should be intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating disease, affecting a maximum of 500 in 1 million people in the EU;

(i) The medicine should be intended for a disease or condition that affects fewer than 200,000 people in the United States or, if the drug is a vaccine, diagnostic drug, or preventive drug, the persons to whom the drug will be administered in the United States are fewer than 200,000 per year;

(ii) It must be unlikely that the revenue after marketing authorisation will cover the investments in its development;

(ii) There is no reasonable expectation that costs of research and development of the drug for the indication can be recovered by sales of the drug in the United States;

(iii) No satisfactory treatment for the disease exists or the new medicinal product is of significant benefit to the patients;

(iii) An application for an orphan designation should contain the reasons why such therapy is needed, accompanied by a discussion of the scientific rationale for the use of the drug for the rare disease, including all data from nonclinical laboratory studies, clinical investigations, and other relevant data that are available to the sponsor, whether positive, negative, or inconclusive. Copies of pertinent unpublished and published papers are also required [4].

(iv) An application for an orphan designation should explain the medical rationale of the medicinal product by means of the mechanism of action as far as it’s known, and some preclinical or clinical date are ‘generally’ required, including all published references [3].