Spring Intro 2023

30/03/2023

What ? • Special regulatory consideration/process to encourage development and authorisation of medicinal products for rare diseases Why ? • Encourages development and license applications for diseases that affect less than 5 in 10,000 • Access to Centralised Procedure and Protocol Assistance • Increased market exclusivity + additional 2 years on completion of agreed PIP When ? • At any stage of development, but before MAA • Reassessment at the time of the MAA How + Where ? • Formal defined submission route with EU regulators (COMP) Who ? • Internal – Regulatory, Project Team, Clinical Research, Epidemiology • External – EMA, COMP, CHMP Orphan Drugs

The Organisation for Professionals in Regulatory Affairs

63

Neil

Accelerating Access

MAA

Phase 1 Phase 2 Phase 3

Post Approval

The Organisation for Professionals in Regulatory Affairs The Organisation for

Professionals i Regulatory Affairs

64