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What is an orphan drug?

"Orphan drugs", or in the EU “orphan medicinal products”, are medicines intended to treat diseases so rare that sponsors (companies) are reluctant to develop them under usual marketing conditions. The process from the discovery of a new molecule to its marketing is long (10 years on average), expensive and, since much work is done in formerly uncharted territory, very risky. Of many molecules tested, none or only one may have a therapeutic effect. Under normal market conditions a company developing a drug intended to treat a rare disease may not recover the capital invested for its development. For this reason, industry and health authorities have jointly argued, first in the US and then at national and European levels (the European Commission) for incentives to stimulate the development of orphan drugs; these efforts led in 2000 to the European Parliament unanimously approving the EU Regulation on Orphan Medicinal Products (EC 141/2000).

The review of all marketing applications for orphan medicines is centralised through EMEA – the European Medicines Evaluation Agency) with the goal of making rapidly available, for rare diseases, medicines with a level of quality, efficacy and safety equivalent to that required for any other medicine.

Source: EPPOSI

 

Last updated: December 2010, by Dr Michael Angastiniotis, MD, DCH (Paediatrician)