Regulatory Science

The information proposed in this section provides key elements on the regulation of medicines for Rare Diseases in human and provides support in the:
Health Technology Assessment,
Drug repurposing,
Preclinical suties,
Early access support,
Clinical Development,
Marketing Authorization,
Ethic and legal issues,
Regulatory Agencies.

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  2. Regulatory Science
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Regenerative Medicine Advanced Therapy (RMAT) Designation

Published by IRDiRC

Advanced TherapyEarly Access SupportRegenerative Medicine Advanced Therapy DesignationRegulatory Affairs

The RMAT Designation is analogous to the Breakthrough Designation designed for traditional drug candidates but applies to regenerative medicine treatments, and allows companies to interact with the FDA more frequently during the clinical development of the therapy. An RMAT-designated therapy is eligible for priority review and accelerated approval.

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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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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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JoInternationalEMA-FDA Scientific Advice

Published by IRDiRC

Early Access SupportJoInternationalEMA-FDA Scientific AdviceRegulatory SciencesScientific Advice

The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services have a program to provide parallel scientific advice (PSA) to sponsors. The goal of the PSA program is to provide a mechanism for EMA assessors and FDA reviewers to concurrently exchange with sponsors their views on scientific issues during the development phase of new medicinal products (i.e., new human drugs and Biologics).

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