Spring Intro 2023

30/03/2023

How? - Demonstrate ‘medical plausibility’

• Define condition • Is it an already recognised orphan condition? •

Proof of concept evidence • In vitro/in vivo preclinical data • Preliminary clinical data • Epidemiological data • Demonstration of significant benefit • no need to demonstrate significant benefit if no other therapy for that condition authorised • Challenging if there are existing therapies • Need to demonstrate improved efficacy, safety or other major contribution to patient care • Becoming more difficult as more medicines for orphan diseases become available, especially in particular ‘crowded’ therapy areas (certain oncology conditions)

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How? – Reassessment/Maintenance of Orphan designation

• Orphan designation is reassessed at the time of MAA submission by COMP

• Following positive CHMP opinion, COMP will make separate assessment whether significant benefit has been demonstrated and orphan status maintained

• Orphan Maintenance Assessment Report (OMAR) will be made available as part of the EPAR

The Organisation for Professionals in Regulatory Affairs

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