Orphan Drugs

These are medicines which are developed specifically for rare diseases. Companies that are looking to gain "orphan medicinal product designation" for their products must submit their application to the Committee for Orphan Medicinal Products (COMP). They must demonstrate that they intend to develop their product(s) "for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 persons in the European Union"¹ (this equates to about 185,000 cases).

 

Under normal circumstances the Pharmaceutical Industry doesn't develop medicines for such small numbers of patients. Bearing this in mind, the EU provides a range of incentives to encourage the industry to develop Orphan Drugs and many companies are now taking advantage of these incentives.

Please refer to our regulatory services for further information or contact us with your specific regulatory compliance requirements.