On June 19, 2018, the European Medicines Agency (EMA) launched a new secure online portal for Orphan Designation (OD) applications. The portal, named 'Iris', provides a single window where applicants can submit and manage the information and documents related to their applications for orphan designation12. This initiative is expected to reduce the time required to prepare and submit the applications. During the review process, applicants can check the status of their applications from any device and receive automatic notifications when the status of the application changes.

About Iris

IRIS is the online web portal through which applicants can apply to the EMA for orphan designation for a medicine. EMA plans to expand the scope of this portal to cover other regulatory and scientific procedures.

This new process, which will become mandatory after September 19, 2018, for procuring orphan designation, requires the following steps to be completed before any activity relating to an orphan designation procedure can be carried out using the new IRIS Portal13:

a) Both the Applicant and Sponsor of an orphan designation, or persons acting on their behalf, must have an active EMA user account and must be registered with IRIS user access roles of either 'Orphan Industry Manager' or 'Orphan Industry Contributor.

b) The 'Organization' for which the OD application is being submitted must be registered in the EMA's Organization Management System (OMS);

c) The 'Substance(s)' for which the application is being submitted must be registered and appear on the official EMA list of all substances, the European Union Telematics Controlled Terms (EUTCT) database;

d) Each new OD application must have a Research Product Identifier (RPI) - the process for requesting an RPI will be required before OD application.

About orphan drug designation

The European Medicines Agency (EMA) plays a central role in facilitating the development and authorization of medicines for rare diseases, which are termed 'orphan medicines' in the medical world. The medicine must fulfil following criteria for designation as an orphan medicine so that it can benefit from incentives such as protection from competition once on the marketyy It must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;

  • The prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
  • No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

Note- In order to help applicants with the transition, EMA has developed two guidance documents. These stepby- step guides provide detailed instructions on how to use the new system and explain what has changed with its introduction.

Footnotes

12 http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2018/06/news_detail_002976.jsp&mid=WC0b01ac058004d5c1

13 http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2018/06/WC500250762.pdf

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