CRED 8 Presentations

01/07/2024

Other specific EU regional requirements for orphan drugs

• Sponsor’s report on the maintenance of designation criteria at the time of MA or type II variation application for a designated orphan medicinal product (Maintenance of OD status report): • Template provided for this report on EMA website which contain relevant guidance documents: https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/orphan medicines/applying-marketing-authorisation-orphan-medicines • The maintenance report should be submitted via the IRIS system*, only after the sponsor receives the confirmation that the validation of the application for marketing authorisation has been completed. EMA may accept the submission of this report at around the time of responding to the day 120 questions (at the discretion of the EMA Product Lead) as not assessed by the Committee of Orphan Medicinal Products (COMP) until the end of the MAA review (post Day 210)

• * Please note that now IRIS is the only tool used for EMA scientific advice /Orphan Drugs/PIPs/ Inspections/ Marketing Status (Eudralink no longer used for these)

The Organisation for Professionals in Regulatory Affairs

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Other specific EU regional requirements for orphan drugs – Maintenance of OD status report

• In the status report, sponsor is requested to establish whether the applicant’s drug still meets the following requirements for orphan drug status: - Condition - Intention to diagnose, prevent or treat - Chronically debilitating and/or life-threatening nature - Number of people affected or at risk - Existing methods - Significant benefit • Will require updating with any major changes made during the review (e.g. addition of new data)

The Organisation for Professionals in Regulatory Affairs

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