CRED ERP 25

07/07/2025

The applicant (orphan designation holder) should submit via the IRIS Platform an application for maintenance of the orphan designation, which will be reviewed by the COMP. The submission should be made around day 121 of a marketing authorisation application or extension of an authorised indication to a new orphan condition. By derogation of the above, the submission should be made after validation for the following cases: REVIEW OF ORPHAN DESIGNATION AT TIME OF MARKETING AUTHORISATION

● accelerated marketing authorisation application, ● extension of an authorised indication within the same orphan condition

The sponsor's report includes data on:

● the current prevalence of the condition to be diagnosed, prevented or treated, or the potential return on investment; ● the current life-threatening or debilitating nature of the condition; ● the current existence of other methods for the diagnosis, prevention or treatment of the condition; ● if applicable, a justification of the medicine's significant benefit.[3]

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EXCLUSIVITY FOR ORPHAN PRODUCTS

● In the case of orphan medicines, additional exclusivity benefits apply, including 10 years of market protection per orphan-designated indication and 2 additional years market exclusivity for completing the studies in the PIP [4]

● To benefit from market exclusivity, a medicinal product must maintain its orphan

designation at the time of marketing authorisation. Orphan-designated indications and non orphan-designated indications cannot be included in the same marketing authorisation. ● The EC proposed revisions to the pharmaceutical legislation in a new Directive and Regulation, which included the proposition that market exclusivity will vary depending on the type of orphan medicinal product.[4]

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