Module 1 2021

30/06/2021

Main characteristics orphan designation EU

• Medicinal products for human use • no veterinary use and no medical devices • Treatment, prevention or diagnosis of a rare disease • Procedure free of charge • Can be requested at any stage of development • Sponsor can be either company or individual established in the EEA • note: Brexit • Positive European Commission Decision gives access to incentives

The Organisation for Professionals in Regulatory Affairs

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Orphan Designation Criteria reg EC/141/2000

1. Intended for diagnosis, prevention or treatment 2. Life-threatening or chronic debilitating condition 3. Prevalence should be less than 5 in 10,000 OR insufficient return of investment 4. No other method authorised in EU for this condition OR significant benefit over existing satisfactory methods

The Organisation for Professionals in Regulatory Affairs

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