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.

Read the Fact Sheet "What is an Orphan Drug?"

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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
The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases