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- About Rare Diseases
- Rare Disease Policy
- Orphan Drugs & Treatments
- What is an orphan drug?
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- Role of EURORDIS
- Patient advocates involvement
- Access to orphan drugs
- Health technology assessment
- EURORDIS Round Table of Companies (ERTC)
- List of the latest marketing authorisations and orphan medicinal products designations
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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?"
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.
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The voice of rare disease patients in Europe
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
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.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
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 initiative
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
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.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
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 initiative