Guidance on how to register to submit orphan designation applications via IRIS secure online portal

The European Medicines Agency (EMA) has launched a new secure online portal for orphan designation applications, which is named IRIS and has been operational since June 2018. IRIS provides a single space where applicants can submit and manage the information and documents related to their applications for orphan designation. Applicants will still be able to use the existing submission process until 19th September 2018, however the EMA encourages companies to start using the new portal now.  

Before you can use IRIS to request an orphan pre-submission meeting, or send an application for orphan drug designation, the following steps need to be completed:

  • Obtain an EMA user account.
  • Ensure that the sponsor is registered as an organisation in Organisation Management System (OMS)/Substances, Products, Organisations and Referentials (SPOR) (unless sponsor is an individual), and that the individual applying is affiliated to the sponsor.
  • Make sure the Active Substance(s) is registered as “current” in the European Union Telematics Controlled Terms (EUTCT) database.
  • If the product has never been given an orphan designation, a request for Research Product Identifier (RPI) should be submitted via the IRIS portal.

After the first two steps have been completed they are valid for each orphan procedure that follows, thus they only need to be completed once. Greater detail on how to carry out the above steps can be found in the ‘IRIS Quick Guide to Registration’. There is also more information regarding the application for orphan designation here.

The IRIS portal will work on mobile devices (tablets and phones) and allows editing of certain file types within a web browser. The IRIS portal can be accessed here.

It is no longer a requirement for sponsors to send a notification of intent to file an orphan drug application for designation to the EMA, however sponsors should follow one of the options below:

  1. Submit directly an application to the EMA, through the online web portal.

Pre-submission meetings are not mandatory and sponsors can send an application for orphan drug designation without notice. However, the EMA appreciates that sponsors send the application preferably 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.

  1. Request a pre-submission meeting/teleconference.

If a Sponsor feels they could benefit from a preliminary discussion before the submission of an orphan drug application to the EMA, they can request a pre-submission meeting/teleconference at least two months prior to their planned submission date via the IRIS portal.