Special situations, market fragmentation I: orphan drugs for rare diseases

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In the European Union rare diseases have been defined as follows:

“Rare diseases, including those of genetic origin, are life-threatening or chronically debilitating diseases which are of such low prevalence that special combined efforts are needed to address them so as to prevent significant morbidity or perinatal or early mortality or a considerable reduction in an individual’s quality of life or socio-economic potential. As a guide, low prevalence is taken as prevalence of less than 5 per 10,000 persons in the European Union [1]”. While the prevalence number seems relatively small, currently it translates into approximately 250,000 persons in the EU with 27 Member States.

The views expressed in this chapter are the personal views of the authors and may not be used or quoted as being made on behalf of, or reflecting the position of, any national competent authority, the European Medicines Agency (EMA) or one of its committees or working parties.