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 the pharmaceutical industry has little interest under normal market conditions in developing and marketing products intended for only a small number of patients suffering from very rare conditions. For the drug companies, the cost of bringing a rare disease medicinal product to the market would not be recovered by the expected sales of the product. For this reason, governments and rare disease patient advocacy organisations such as Eurordis have emphasized the need for economic incentives to encourage drug companies to develop and market medicines for the many neglected and "orphaned" rare disease patients.
Author: Fabrizia Bignami
Editor: Julia Fitzgerald
Photo: © www.freeimages.co.uk
