EMA Outlines Changes to Orphan Submissions via New Online Portal

After encouraging sponsors to begin using its new online portal for submitting and managing orphan designations last month, the European Medicines Agency (EMA) on Friday issued a guideline providing instructions on using the new portal.

In June, EMA launched the new secure portal, called Iris, giving sponsors three months to familiarize themselves with the system before it becomes mandatory on 19 September 2018. In the interim, sponsors may still use the existing process to file for orphan drug designation and EMA has updated its guideline for doing so, noting the 19 September deadline for switching to Iris.

According to EMA, the Iris portal will be accessible on mobile devices and will support some document editing within its web interface.

In the guideline, EMA lays out the procedure and technical requirements for submitting and managing orphan designation applications using the Iris portal.

To use the portal, sponsors must have an EMA user account; register for EMA's substances, products, organizations and referentials (SPOR) program; and ensure the active substance is registered in the European Union Telematics Controlled Terms (EUTCT) database.

For products that have never had an orphan designation, EMA says that sponsors should request a research product identifier (RPI) through Iris.

Going forward, EMA says that sponsors no longer need to send a notification that they intend to file for orphan drug designation ahead of their submission.

Instead, EMA says sponsor should submit their applications directly through the portal or request a pre-submission meeting at least two months ahead of their planned submission date.

While pre-submission meetings are not required, EMA says they are encouraged, free of charge, and are typically held via teleconference. For sponsors that request a pre-submission meeting, EMA says that sponsors should provide draft information through the IRIS portal at least one week before the meeting date.

Guideline