Applying for orphan designation

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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.


 

Click to download the full image as PDF

Improved way to apply for and manage orphan designations

On 19 June 2018, EMA launched a new secure online portal for sponsors to submit applications for orphan designation and to manage post-designation activities

EMA expects the portal, named 'Iris', to reduce the time needed to prepare and submit applications. 

Users can check the status of their applications from any device and receive automatic notifications when the status changes. 

Applicants are still be able to use the existing submission process (described on this page) until 19 September 2018. However, the Agency strongly encourages companies to start using the new portal from today.

To help applicants with the transition, EMA has developed two guidance documents explaining how to use the new system and what has changed with its introduction:

EMA tested a pilot of the new system in March 2018 with 35 volunteers from 26 different organisations. Feedback from this test helped EMA to optimise the portal and showed high levels of satisfaction.

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

In future, EMA may extend the system to include other procedures, taking user feedback and experience into account.

General principles

Sponsors are no longer required to send a notification of intent to file an application for orphan designation to the EMA. Sponsors should instead follow one of the two options listed below:

  • Submit the application to EMA: presubmission meetings are not mandatory and sponsors are welcome to send an application for orphan drug designation without notice. However, EMA would appreciate that sponsors send the application preferably a few days before any of the published submission deadlines to allow more time for the validation process.
  • Request a presubmission meeting/teleconference: if sponsors feel they could benefit from a preliminary discussion before submitting an orphan drug application to EMA, they should request a presubmission meeting/teleconference at least two months prior to their planned submission date by sending an email to orphandrugs@ema.europa.eu. This should allow enough time for the organisation and any amendment of the application as recommended by 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 orphandrugs@ema.europa.eu via the secure system, Eudralink, clearly indicating in the subject message if the application is:

  • ‘Draft application for presubmission meeting on &ltdate&gt’
  • ‘Application for orphan drug designation submission deadline &ltdate&gt’

Sponsors should contact the online EMA Service Desk to open an account.

Presubmission meetings

EMA 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 EMA 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.

Application procedure

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

Refer to these documents for assistance completing these forms:

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 EMA secretariat.

Once the application form and sections A to E are complete, the sponsor should submit the complete application electronically to EMA.

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.

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:

  • United States before, EMA advises sponsors to apply for an orphan designation from the United States Food and Drug Administration (FDA). EMA and the FDA interact and collaborate regularly in the 'clusters' for 'Orphan medicinal products' and 'Rare diseases'.
  • Japanese authorities before, EMA 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:

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