Module 1 2021

30/06/2021

Orphan conditions bring challenges

Provide incentives stimulating research & development (push)

• Rare • Multi-systemic • Spectrum of severity • Under-diagnosed • Lacking validated endpoints

Modify market conditions (pull)

The Organisation for Professionals in Regulatory Affairs

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EU Regulatory context

• Regulation (EC) No 141/2000 • Criteria for orphan designation • Committee (COMP)

• Procedure • Incentives

• History

• 1996 – 1st draft regulation • 2000 – regulation into force

• 2001 – first MAA approval with orphan designation (OD) • 2020 – 3,678 submitted applications for designations ‒ 2,282 commission decision on OD ‒ 190 MAA approval with OD

The Organisation for Professionals in Regulatory Affairs

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