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.