This page provides information for sponsors on how to apply to the European Medicines Agency for orphan designation for a medicine.
Click on the image below to view a guide (flowchart) for the orphan designation process for potential sponsors.
Sponsors are no longer required to send a notification of intent to file an orphan drug application for designation to the EMA. Sponsors should follow one of the two options listed below instead:
Submit the application to the EMA
Presubmission meetings are not mandatory and sponsors are most welcome to send an application for orphan drug designation without notice. However, we will appreciate that sponsors send the application preferably few days before any of the published submission deadlines available on the EMA website to allow more time for the validation process.
Request a presubmission meeting/teleconference
If sponsors feel they could benefit from a preliminary discussion before the submission of an orphan drug application to the EMA they should request a presubmission meeting/teleconference at least two months prior to their planned submission date by sending an email to email@example.com. This should allow enough time for the organisation and any amendment of the application as recommended by the EMA. Sponsors should send the draft application for the presubmission discussions one week in advance.
In any of the cases mentioned above sponsors should submit the application to firstname.lastname@example.org via the secure system, Eudralink, clearly indicating in the subject message if the application is:
- ‘Draft application for presubmission meeting on <date>’
- ‘Application for orphan drug designation submission deadline <date>’
Sponsors should contact the online EMA Service Desk to open an account.
The Agency strongly encourages sponsors to request a presubmission meeting with the Agency prior to filing an application.
Presubmission meetings usually take place via teleconference, unless the sponsor has a strong preference to come to the Agency in person.
Where possible, sponsors should request a pre-submission meeting at least two months prior to filing. Presubmission meetings for orphan designation are free of charge.
Presubmission meetings are useful since the evaluation process has a fixed duration of 90 days and cannot be lengthened to accommodate for the lack of data or other omissions in the application. Experience has shown that they have a positive impact on the success rate of the applications.
Sponsors should use the forms below to apply for orphan designation:
- Application form for orphan-medicinal-product designation
- Common European Medicines Agency / Food and Drug Administration (FDA) application form or application form for orphan medicinal product designation
- Template for sections A to E for the scientific part of the application for orphan designation
- Translations required with the submission of an application for orphan medicinal product designation
Refer to these documents for assistance completing these forms:
- Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another, 27 March 2014
- Points to consider on the calculation and reporting of the prevalence of a condition for orphan designation
- Recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation
- Data providers and sources to identify existing authorised medicinal products in the European Union and European Economic Area
In particular, when completing section A.3.2 'Plausibility of the orphan condition; rationale for use of the medicinal product', sponsors should clearly identify studies with the substance in a relevant model(s) of the condition and, if possible, preliminary clinical data in patients with the condition.
Each application is assigned two coordinators:
- one member of the Committee for Orphan Medicinal Products (COMP);
- one scientific administrator from the Agency secretariat.
Once the application form and sections A to E are complete, the sponsor should submit the complete application electronically to the Agency.
The application should include full copies of the bibliographical references. The application form and sections A to E should be supplied in Word format (compatible with version 97-2003). References and the signed application form should be supplied as PDF files. In the application, sponsors should clearly substantiate the claims and support the statements made, with references where possible.
Deadlines for submission of applications for orphan designation are available under submission deadlines.
Applications for orphan designation are free of charge.
The Agency will validate the application and will send the sponsor a validation issues letter explaining if the application is found to be invalid or incomplete. Once the validation is complete, the Agency will send a timetable for the evaluation procedure to the sponsor.
The Agency advises sponsors developing advanced therapies to apply separately to the Committee for Advanced Therapies (CAT) to have their medicine classified as an advanced-therapy medicine. For more information, see advanced-therapy medicinal product classification.
Parallel application with international regulators
The Agency encourages parallel applications for orphan designation with regulatory authorities outside the EU.
The Agency has special arrangements with regulators in the United States and Japan for this purpose:
- The Agency advises sponsors to use the common orphan application form with the United States Food and Drug Administration (FDA) to apply for orphan designation, particularly if an application has not been submitted in the United States before.
- If an application has not been submitted to the Japanese authorities before, the Agency also encourages the sponsor to seek orphan designation from the Ministry for Health, Labour and Welfare (MHLW) in Japan. Under the Japanese orphan designation system, the MHLW provides consultation on orphan designations before submission, whereas marketing-authorisation applications submitted following an orphan designation are assessed by the and the Pharmaceuticals and Medical Devices Agency (PMDA). The MHLW generally seeks scientific counsel from the PMDA on the orphan designation.
Evaluation of applications
After submission, the two coordinators prepare a summary report on the application, which is circulated to all COMP members and discussed at the COMP's next plenary meeting.
At this stage, the COMP will either adopt a positive opinion or raise a list of questions and invite the sponsor to an oral explanation at the next COMP plenary meeting.
The COMP should adopt an opinion by day 90 of the procedure. It forwards this to the European Commission for adoption of a decision.
If the COMP's opinion is negative, the sponsor can appeal.
European Commission decision
The European Commission will issue a decision on a COMP opinion within 30 days of receipt. Following a decision:
- the Agency publishes information on the orphan designation under rare disease (orphan) designations;
- the European Commission enters the orphan designation into the Community register of designated orphan medicinal products.
- Submission deadlines for orphan designations
- Questions and answers: orphan-designation application
- Guidance and forms
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