Orphan Medicine

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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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Rare Pediatric Priority Review Voucher

Published by IRDiRC

Marketing AuthorisationOrphan MedicinePaediatric MedicineRare Paediatric Priority VoucherRegulatory AdviceRegulatory AffairsResearch and Drug Development

This program grants a voucher for priority review from the US Food and Drug Administration (FDA), which aim to render a decision within 6 months (in contrast to 10 for a standard review). The developed drug for which the voucher is awarded must be intended for a rare disease or condition and that primarily affect individuals from 0 to 18 years. The voucher can be utilized for any other drug development program.

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Mechanism of Coordinated Access to OMPs

Published by IRDiRC

EUnetHTAHTA BodiesMechanism of Coordinated Access to OMPsOrphan MedicineRegulatory AffairsReimbursementResearch and Drug Development

Early dialogue with payers /Medev – MoCA provides a mechanism for European countries to collaborate on coordinated access to orphan medicines in a voluntary, dialogue-based approach, intended to create a fluid set of interactions between key stakeholders, across all aspects of a product

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