- About EURORDIS
- About Rare Diseases
- Rare Disease Policy
- Orphan Drugs & Treatments
- What is an orphan drug?
- Improve the safety of your medicines
- Compassionate Use
- 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
- Living with a Rare Disease
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Orphan drugs
Is there an upper limit on what should be spent on a single patient? – The case of orphan drugs. In the European Union, around 30 million people suffer from rare diseases. The EU defines a rare disease as a disease which affects less than one person in every 2,000 people. Because expected sales for drugs to treat rare diseases are small, there is little incentive for drug companies to develop new therapies to diagnose and treat such disorders.
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In 1999 the EU unanimously passed the Orphan Medicinal Products Regulation, to encourage industry to develop therapies for rare conditions by giving financial incentives. Orphan drugs are drugs developed for diagnosing and treating rare diseases. In the current system, drug candidates are designated as “orphan” and market authorised through a centralised European procedure. Availability and reimbursement remain a national responsibility. Although orphan drugs can improve health for millions of people, their high development costs, a small market and marketing exclusivity mean that tend to be very expensive for the patients and health care systems.
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The voice of rare disease patients in Europe
The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiative
Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative
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, Rare Diseases International is a EURORDIS initiative
Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative
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