Submitting annual reports on medicine development
This page provides information on the report that sponsors must submit to the European Medicines Agency (EMA) every year after their medicine receives an orphan designation, until they submit an application for marketing authorisation.
Sponsors need to use EMA's IRIS system to submit all post-designation activities, including annual reports. For information and guidance on using IRIS, see the IRIS homepage.
Sponsors are not requested to submit any documents; it is sufficient to complete the requested fields in the IRIS system. Note that the fields on ‘compassionate use’, ‘marketing authorisation’ and ‘new orphan designation’ relate to global development (not only EU).
Optionally, additional documents can still be uploaded if appropriate, to provide information on the status of the development of the medicine, including:
- a review of ongoing clinical studies;
- a description of the investigation plan for the coming year;
- any anticipated or current problems in the process, difficulties in testing and potential changes that may have an impact on the medicine's orphan designation.
If a sponsor withdraws a marketing authorisation application, or the application receives a negative opinion from the Committee for Medicinal Products for Human Use (CHMP), the sponsor needs to resume submitting annual reports on medicine development for the designated orphan medicine.
Annual reports for orphan medicinal products designated by the EU should be submitted within 2 months following the anniversary of the grant of the designation or at World Rare Disease Day (last day of February).
If the sponsor wishes to inform EMA about Changing the name or address of a sponsor or Transferring an orphan designation, this should not be done in the annual report. For this purpose it is necessary to undergo the procedure outlined in section G of the Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designation form one sponsor to another (ENTR/6283/00).