Regenerative Medicine Advanced Therapy (RMAT) Designation

Published by IRDiRC

Advanced Therapy Early Access Support Regenerative Medicine Advanced Therapy Designation Regulatory 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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Neglected and Tropical Diseases Priority Review Voucher

Published by IRDiRC

Early Access Support Neglected and Tropical Disease Priority Review Voucher Regulatory Advice Regulatory Affairs Scientific Advice

This program grants a priority review voucher (PRV) from the US Food and Drug Administration (FDA) to sponsors of certain tropical disease product application

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Rare Pediatric Priority Review Voucher

Published by IRDiRC

Marketing Authorisation Orphan Medicine Paediatric Medicine Rare Paediatric Priority Voucher Regulatory Advice Regulatory Affairs Research 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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Rare Pediatric Disease Designation

Published by IRDiRC

Early Access Support Orphan Drug Designation Paediatric Medicine Rare Paediatric Disease Designation Regulatory Affairs

The FDA grants rare pediatric disease designation for diseases with serious or life- threatening conditions that affect people from birth to 18 years of age, that affect fewer than 200,000 people in the U.S.

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FDA Expedited Program for serious conditions – Special Protocol Assessment

Published by IRDiRC

Clinical Development Clinical Trial Design Regulatory Advice Regulatory Affairs Special Protocol Assessment

FDA-SPA is a process in which sponsors may ask to meet with FDA to reach agreement on the design and size of certain clinical trials, clinical studies, or animal studies, to determine if they adequately address scientific and regulatory requirements for a study that could support marketing approval.

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European Network of Paediatric Research at the European Medicines Agency

Published by IRDiRC

Early Access Support European Network of Paediatric Research Orphan Drug Paediatric Medicine Regulatory Affairs Research 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 Support JoInternationalEMA-FDA Scientific Advice Regulatory Sciences Scientific 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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EMA pre-submission meeting (Pre-MAA meetings)

Published by IRDiRC

EU Marketing Authorisation Road Map MAA Pre-Submission MAA Submission Marketing Authorisation Regulatory Regulatory Affairs

MAA Pre-submission meeting is not a mandatory meeting, however, it is strongly recommended as it is the final meeting before the registration (MAA submission) where the developer address product- specific legal, regulatory and scientific issues

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Magisterial hospital preparations – hospital exemptions

Published by IRDiRC

Advanced Therapy ATMPs Early Access Support Hospital Exemption Regulatory Affairs

Article 28 of the ATMP Regulation also empowers Member States to permit the manufacturing and use of certain non- routine produced advanced therapies that have not been authorised by the Commission under certain conditions (so-called “Hospital Exemption”) outside the scope of the Medicinal Product Directive 2001/23.

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Joint EMA-HTA Scientific Advice

Published by IRDiRC

EUnetHTA HTA Bodies Regulatory Affairs Scientific Advice

The European Medicines Agency (EMA) offers consultations in parallel with the European Network for Health Technology Assessment (EUnetHTA) as of July 2017. This aims to allow medicine developers to obtain feedback from regulators and health technology assessment (HTA) bodies on their evidence-generation plans to support decision-making on marketing authorisation and reimbursement of new medicines at the same time.

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The Innovation Management Toolbox was originally developed through funding from the EJP RD project, which received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 825575. The continued development and maintenance of the Toolbox are now supported by the ERDERA partnership, funded under the European Union’s Horizon Europe Research & Innovation programme, grant agreement No. 101156595.

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