Orphan Drug

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All SubjectsOrphan Drug

Single Patient Expanded Access

Published by IRDiRC

Early Access SupportOrphan DrugOrphan MedicineRegulatory AffairsResearch and Drug DevelopmentSingle Patient Expanded Accesss

This FDA program allows early access to drugs and medical devices before marketing approval, under regulatory oversight with collection of Safety data, to individual patients with serious or immediately life-threatening diseases or conditions who lack therapeutic alternatives. This includes emergency and non-emergency expanded access.

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European Network of Paediatric Research at the European Medicines Agency

Published by IRDiRC

Early Access SupportEuropean Network of Paediatric ResearchOrphan DrugPaediatric MedicineRegulatory AffairsResearch and Drug Development

The European Network of Paediatric Research at the European Medicines Agency (Enpr- EMA) is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children. Enpr-EMA enables networking and collaboration with members from within and outside the European Union (EU), including academia and the pharmaceutical industry

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EU Compassionate Use Programs

Published by IRDiRC

Clinical DevelopmentCompassionate UseEarly Access SupportEMA Compassionate UseEuropean UnionOrphan DrugRegulatory AffairsResearch and Drug Development

Compassionate use is a treatment option that allows the use of an unauthorised medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials.

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