Applying for orphan designation

The European Medicines Agency (EMA) provides information and guidance to sponsors on applying for an orphan designation for a medicine.

Click on the image below to view a guide (flowchart) for the orphan designation process for potential sponsors.

Orphan designation process - Sponsor's guide

 

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Apply using the 'IRIS' platform

Sponsors need to use EMA's secure online IRIS platform to submit applications for orphan designation and to manage pre- and post-designation activities:

IRIS button

For information and guidance on using IRIS, see the IRIS homepage.

General principles

Sponsors should follow one of the two options below to submit an application for orphan designation:

  • Submit directly an application to EMA, through the IRIS system:

Pre-submission meetings are not mandatory and sponsors are welcome to send an application for orphan drug designation without notice. However, EMA would appreciate it if sponsors could send the application a few days before any of the published submission deadlines available on the EMA website to allow more time for the validation process and the possibility to intervene in case of technical problems.

  • Request a pre-submission meeting/teleconference:

EMA strongly encourages sponsors to request a pre-submission meeting with the Agency prior to filing an application. Pre-submission meetings usually take place via teleconference, unless the sponsor has a strong preference to come to EMA in person.

If a sponsor feels they could benefit from a preliminary discussion before the submission of an orphan drug application, they can request a pre-submission meeting/teleconference at least two months prior to their planned submission date via the IRIS portal. This should allow enough time for the organisation and any amendment of the application as recommended by EMA.

Sponsors should create an initial draft application for orphan designation before creating the application for the pre-submission discussion. These are two seperate submissions to be made in the IRIS system. The draft submission for orphan designation should be populated with the relevant data and documents, but not submitted, at least one week before the pre-submission meeting date.

Pre-submission 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.

Application procedure

Sponsors should use the forms below to apply for orphan designation:

Documents to refer to:

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:

Applications for orphan designation are free of charge.

EMA 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.

EMA 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

EMA encourages parallel applications for orphan designation with regulatory authorities outside the EU.

EMA has special arrangements with regulators in the United States and Japan for this purpose. If an application has not been submitted to the:

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.

The European Commission will issue a decision on a COMP opinion within 30 days of receipt. Following a decision:

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