CRED 8 Presentations

01/07/2024

Other Requirements: Orphan Maintenance* In parallel to MA submission • Only for Products with an orphan designation covering the indication in the MAA • A separate application should be submitted to the Committee for Orphan Medicinal Products (COMP); timelines depend on whether Accelerated Assessment has been granted • Submit orphan maintenance report with supporting annexes through IRIS platform • Justify the orphan criteria continue to be met and MAA indication falls within designation • Include: Prevalence, life-threatening and debilitating nature of condition, current methods for diagnosis and treatment, justification of medicine’s significant benefit • Possible to have a validation/post-submission COMP meeting to discuss application • COMP perform their own assessment, issuing final opinion after CHMP on maintenance • COMP likely to raise questions; it is possible to have a COMP Oral Explanation

*Orphan medicines are medicinal products for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating, with prevalence of less than 5 per 10000 EU inhabitants, which offer significant benefit in condition.

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Timelines: From Day 1 to Day 120* Activity Day Start of Procedure, application confirmed as valid Day 1

Day 80 Day 94

Co-Rapporteur and Rapporteur finalise preliminary Assessment Reports and send to Applicant

PRAC Rapporteur circulates the RMP assessment report (PRAC preliminary assessment report)

Day 100 - 112 Day 101 104 Day 120 Final LoQ

(Co-)Rapporteurs, other PRAC and CHMP Committee members and EMA send comments (including peer reviewers). T/C between all parties at Day 112. PRAC adopts PRAC RMP Assessment Overview and Advice for D120 LoQ (for accelerated assessments / ATMPs / PUMAs only) • Consolidated List of Questions (LoQ) adopted by CHMP and sent to Applicant- same structure as preliminary ARs • One list of questions : Rapp/Co-Rapp/PRAC/product information/QRD+mockups • At the latest by Day 120, adoption by CHMP of request for GMP/GLP/GCP inspection, if necessary (Inspection procedure starts). • Clock stop begins (1 -3 months or longer if requested)

*Refer to EMA submission timetables; steps & timelines depend on timetable being followed (e.g. ATMP/accelerated/standard)

The Organisation for Professionals in Regulatory Affairs

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