Watch the video below to learn more about the importance of orphan medicines and how EMA supports their development in the EU.
EMA is responsible for reviewing applications from sponsors for orphan designation.
To qualify for orphan designation, a medicine must meet a number of criteria:
In February 2018, EMA published a question-and-answer document addressing common misunderstandings about the meaning of orphan designation and other aspects pertaining to orphan medicines:
For more information, see:
Finding effective treatment for patients with rare diseases can be very difficult.
Since the EU orphan regulations entered into force in 2000, it has played a central role in facilitating the development and authorisation of medicines for rare diseases.
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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:
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:
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:
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:
Since rare diseases are a global issue, EMA works closely with its international partners on the designation and assessment of orphan medicines, in particular:
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:
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.
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:
If you cannot find a solution, please create a ServiceDesk ticket.