Module 1 2021

30/06/2021

Orphan condition EC Guideline (ENTR/6283/00)

• Definition: Any deviation(s) from the normal structure or function of the body, as manifested by a characteristic set of signs and symptoms (typically a recognised distinct disease or a syndrome) • Development plausible based on pathogenesis and pharmacodynamics • Distinct pathophysiology, histology, clinical presentation • NOTE: Orphan Condition ≠ Orphan indication ≠ Therapeutic indication

Approved labelled indication

Orphan condition

Orphan indication

The Organisation for Professionals in Regulatory Affairs

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Source: O’Connor et al. - Defining orphan conditions in the context of the European orphan regulation: challenges and evolution. Nature reviews, 2018.

The Organisation for Professionals in Regulatory Affairs

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