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

From 19 September 2018, 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

EMA first launched IRIS in June 2018 and optimised the system based on feedback from users during a three-month pilot phase.

IRIS reduces the time needed to prepare and submit applications. Users can check the status of applications from any device and receive automatic notifications when the status changes.

It is part of a longer-term EMA programme that aims to make the handling of product-related applications easier and utilises the domains of master data in pharmaceutical regulatory processes (SPOR).

EMA is extending the IRIS platform to cover other procedures, including parallel distribution.

Access to IRIS

EMA has published guidance documents on how to use IRIS for orphan designation and other procedures:

To register to use IRIS for orphan designation, you need to ensure that:

1. You have an active EMA account

Visit EMA's Account Management portal to check if you have an EMA account or obtain a new one.

Allow up to two working days if you need to reset the password for an account for which you have not set up your security questions.

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2. Your organisation is registered with EMA

Your EMA account needs to be associated with an industry organisation registered in EMA's Organisations Management Service (OMS). For more information, see SPOR user registration.

Allow from five to ten working days to register a new organisation or update organisation data in the OMS. 

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3. Your EMA account gives you access to IRIS

To access IRIS, you need to request an Orphan Industry 'Manager' or 'Contributor' role via EMA's Account Management portal. Your organisation's 'User Admin' needs to approve the request.

Your organisation must have at least one person assigned to the 'User Admin' role, requested via EMA's Account Management portal

Allow up to two working days for EMA to approve your organisation's first 'User Admin'.

To submit applications via IRIS you need to ensure that:

1. Your active substance is recognised

The active substance(s) contained in your application must be registered as 'authorised' and 'current' in the EU Telematics Controlled Terms (EUTCT) system.

Allow from four to eight days to register a new active substance

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2. You have a valid Research Product Identifier

If you do not have a valid Research Product Identifier (RPI, formerly called a 'Unique Product Identifier') for your active substance, you need to request this via IRIS. 

Allow up to two working days for EMA to approve a new RPI via IRIS. 

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 provide the draft application for the pre-submission discussions via the IRIS system one week in advance.

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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