Pre Investigational New Drug (IND) interactions

Published by IRDiRC

Early Access Support Milestone Meeting Pre Investigational New Drug Interaction Pre-NDA Meeting Regulatory Affairs Scientific Advice

A pre-IND meeting is an FDA milestone meeting also known as Type B meeting . In these meetings the drug sponsor seeks FDA feedback about the quality, non- clinical and clinical development plan. The Pre-IND meetings are not mandatory.

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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 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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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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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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Advanced Therapy Medicinal Products (ATMPs) Classification

Published by IRDiRC

Advanced Therapy ATMPs Classification Early Access Support European Union Regulatory Affairs

Companies can consult the European Medicines Agency (EMA) to determine whether a medicine they are developing is an advanced therapy medicinal product (ATMP). This is an opportunity for ATMP developers to receive confirmation that a medicine, which is based on genes, cells or tissues, meets the scientific criteria for defining an ATMP. The procedure can help developers to clarify the applicable regulatory framework since the beginning of the product development.

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EU Compassionate Use Programs

Published by IRDiRC

Clinical Development Compassionate Use Early Access Support EMA Compassionate Use European Union Orphan Drug Regulatory Affairs Research 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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Consultations on Pharmacogenomics / Biomarkers (As part of PMDA consultations)

Published by IRDiRC

Biomarker Early Access Support MHLW or PMDA Regulatory Pharmacogenomics PMDA Regulatory Affairs Scientific Advice

Consultations on Pharmacogenomics / Biomarkers is one of the PMDA consultations. The purpose of this consultation is to provide general scientific advice for utilization of pharmacogenomics or biomarkers that are not specified in a certain product.

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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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