“Orphan drugs” are medical products intended for prevention, diagnosis or treatment of serious diseases or which constitute a risk to life and which are rare. They are designated as “orphan” because, under normal market conditions, the development and marketing of products directed at the small number of patients suffering from very rare diseases have little return for the pharmaceutical industry.
For a medicinal product to be considered orphan it must fulfil the following criteria specified in Regulation 847/2000:
General criterion - medicinal product intended for the diagnosis, prevention and treatment of a rare, life-threatening or chronically debilitating condition;
Epidemiologic criterion - the pathology which the medicinal product is intended for affects not more than 5 in 10,000 people;
Economic criterion - the medicinal product that does not generate the necessary return for its development and research;
Non-existent methods or clinical superiority of treatment - where there is no satisfactory method of diagnosis, prevention or treatment with the same effect authorised by the European Union; or alternatively meaning that, although such a method may exist, the medicinal product in question will be of significant therapeutic benefit.
Since 2000, the year in which the Regulation became effective, more than a hundred and fifty medicines have already been designated as Orphan Medicines. However, to date, few have obtained Marketing Authorisation, due to the complexity of assessment of the criteria of safety, efficacy and quality.* Author: Dr. Isabel Afonso (2003) * Source: INFARMED, Pharmacovigilance Bulletin