What is an orphan drug?
"Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare.

A disease or disorder is defined as rare in Europe when it affects less than 1 in 2,000 citizens.
These drugs are called “orphan” because under normal market conditions the pharmaceutical industry has little interest in developing and marketing products intended for only a small number of patients.
For drug companies, the extremely high cost of bringing a medicinal product to market would not be recovered by the expected sales of the product.
As a result the potential market for new drug treatment is also small and the drug companies industry would actually incur a financial loss.
Therefore governments and rare disease patient organisations such as EURORDIS advocate for economic incentives to encourage drug companies to develop and market medicines for rare disease treatment.
Page created: 19/08/2009
Page last updated: 07/11/2014