Spring Intro 2023

30/03/2023

David

Drug Development for rare diseases – Orphan Drug Development

MAA

Phase 1 Phase 2 Phase 3

Post Approval

The Organisation for Professionals in Regulatory Affairs The Organisation for

Professionals i Regulatory Affairs

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What & Why ?

5000 - 8000 rare diseases in the EU affecting 27 – 36 million people • how to ensure these patients have the same quality of treatment as other patients in the EU? Regulation EC/141/2000 • by encouraging development and authorisation of medicinal products for rare diseases • with incentives for industry to meet development costs Has Regulation worked? • 2300 Orphan Designations granted, 192 approved orphan MAAs (EMA, 2020 figures)

The Organisation for Professionals in Regulatory Affairs

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