Orphan designation: Overview
The European Medicines Agency (EMA) facilitates the development and authorisation of medicines for rare diseases, also called 'orphan medicines'.
HumanRare diseases
About 36 million people living in the European Union (EU) suffer from a rare disease.
A disease is considered rare if fewer than 5 in 10,000 people have it.
EMA plays a central role in facilitating the development and authorisation of medicines for rare diseases. The medical world refers to them as 'orphan medicines'.
For more information on the regulation of medicines for human use in the EU, see:
Role of EMA
EMA is responsible for reviewing applications from sponsors for orphan designation.
To qualify for orphan designation, a medicine must meet a number of criteria:
- it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
- the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
- no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.
Duration: 3:17 minutes.
A question-and-answer document addressing common misunderstandings about the meaning of orphan designation and other related aspects is available below:
Also on this topic
Facts and figures
1 out of 12
people in the EU has a rare disease
Over 3170
medicines with orphan designation since 2000
Over 275
orphan medicines authorised in the EU since 2000
Info sheet - Orphan medicines in the EU
Finding effective treatment for patients with rare diseases can be very difficult.
The EU orphan regulation has played a central role in facilitating the development and authorisation of medicines for rare diseases since it entered into force in 2000.
The EU offers incentives to encourage pharmaceutical companies to research and develop medicines for rare diseases that otherwise would not be developed.
To access these incentives, companies can apply for orphan designation for their medicine. They would need to meet certain criteria, as explained in the info sheet available in this section.
Read info sheet
Rare diseases at a glance
Most rare diseases (80%) have identified genetic origins, and affect between 3% and 4% of births. Other rare diseases are due to degenerative and proliferative causes.
Symptoms of some rare diseases may appear at birth or in childhood, including spinal muscular atrophy, lysosomal storage disorders, patent ductus arteriosus (PDA), familial adenomatous polyposis (FAP) and cystic fibrosis.
More than half of rare diseases appear during adulthood, such as renal-cell carcinoma, glioma and acute myeloid leukaemia.
Medical and scientific knowledge about rare diseases is lacking. The number of scientific publications about rare diseases continues to increase, with an average of 5 new diseases described every week in the medical literature. However, fewer than 1,000 diseases benefit from even minimal amounts of scientific knowledge. These tend to be the rare diseases that occur most frequently.
How apply for orphan designation
Applications for orphan designation are examined by the EMA's Committee for Orphan Medicinal Products (COMP), using the network of experts that the Committee has built up.
The evaluation process takes a maximum of 90 days from validation. EMA sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation.
For information on how to apply, see:
EMA makes available in IRIS a list with information on all orphan medicine designations and amendment opinions adopted by COMP. To consult it, see:
For the full list of orphan designations, see:
Orphan medicine incentives
Developing medicines intended for small numbers of patients has little commercial incentive under normal market conditions.
EU offers a range of incentives to encourage the development of designated orphan medicines.
Sponsors who obtain orphan designation benefit from:
- Protocol assistance, a type of scientific advice specific for designated orphan medicines
- Market exclusivity once the medicine is on the market
- Fee reductions, depending on the status of the sponsor and the type of service required
Applicants from academia are eligible to receive free protocol assistance for developing orphan medicines, as of 19 June 2020.
When planning the development of their medicinal product, sponsors should consult the relevant scientific guidelines.
Sponsors must submit an annual report to EMA summarising the status of development of the medicine. To find more on when and how a sponsor must submit these reports, see Submitting annual reports on medicine development
For more information, see:
Paediatric medicines
More than half of designated orphan medicines are intended for paediatric use.
Medicines authorised across the EU with the results of studies from a paediatric investigation plan included in the product information are eligible for an extension of their supplementary protection certificate.
For designated orphan medicines, the incentive is an additional two years of market exclusivity.
For more information, see:
Marketing authorisation applications for designated orphan medicines must be submitted to EMA for assessment through the centralised procedure.
They are assessed by EMA's Committee for Medicinal Products for Human Use (CHMP).
Designated orphan medicines are eligible for conditional marketing authorisation.
In some cases, designated orphan medicines may be allowed to be administered to patients under compassionate use, a treatment option that allows the use of an unauthorised medicine outside a clinical study.
At the time of marketing authorisation, sponsors also need to submit an application for maintenance of the orphan designation in order to be eligible for the ten-year market exclusivity incentive.
Sponsors may also need to submit an evaluation of orphan similarity.
EMA encourages companies developing orphan medicines to check if they can be classified as a micro, small or medium-sized enterprise (SME).
Such companies benefit from further incentives, including administrative and procedural assistance from EMA's SME office and fee reductions.
For more information, see:
Global dimension
Since rare diseases are a global issue, EMA works closely with its international partners on the designation and assessment of orphan medicines, in particular:
- the Japanese Ministry for Health, Labour and Welfare (MHLW) on issues related to orphan medicines;
- the United States Food and Drug Administration (FDA), sharing information on orphan medicines under their confidentiality arrangement.
EMA and FDA have also developed common procedures for applying for orphan designation and for submitting annual reports on the status of development of designated orphan medicines.
EMA works with organisations representing patients with rare diseases through the European Organisation for Rare Diseases (EURORDIS).
For more information, see:
Contact points
All submissions for orphan designation and related procedures must be submitted via the IRIS system.
Guidance on the use of the system and how to prepare submissions is available in the platform's homepage.
For communications related to an ongoing procedure in IRIS, reply to any email received from the system about that procedure. To ensure proper routing, please do not change the subject of the email.
For general questions about orphan designations and associated procedures, not related to an ongoing IRIS procedure, please submit your message via the form available on the following webpage:
Guidance is available in the IRIS homepage to help you with technical issues, such as:
- EMA accounts;
- roles and affiliation;
- SPOR and IRIS login issues;
- IRIS submission issues.
If you cannot find a solution, please create a ServiceDesk ticket.
Page update history
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27 February 2026
Updated section on 'Info sheet - Orphan medicines in the UE' with information included in a new version of the info sheet