CRED 8 Presentations

01/07/2024

Orphan designation

At time of MAA

Before MAA submission

• Assessment by COMP; EC decision for designation (Orphan register) • To be granted at any stage before MAA submission • To be confirmed before the granting of the MAA

2 nd hurdle: maintenance of OD

1 st hurdle - OD

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The Organisation for Professionals in Regulatory Affairs

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Orphan designation criteria (cumulative)

•Rare condition affecting less than 5 per 10.000 persons in the EU, or •[exceptionally] it is unlikely that the medicine would generate sufficient returns to justify the investment

Prevalence

Seriousness of condition

•Life threatening and/or chronically debilitating condition

Comparison to available treatments

•No satisfactory methods exist, or •Significant benefit vs. existing methods

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The Organisation for Professionals in Regulatory Affairs

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