Adaptive Pathways

Published by IRDiRC

Adaptative Pathway Clinical Development Clinical Trial Design Early Access Support Regulatory Affairs

The adaptive pathways approach is part of the European Medicines Agency’s (EMA) efforts to improve timely access for patients to new medicines

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Paediatric Investigation Plans

Published by IRDiRC

Early Access Support Paediatric Investigation Plan Paediatric Medicine Regulatory Affairs

A paediatric investigation plan (PIP) is a development plan aimed at ensuring that the necessary data are obtained through studies in children, to support the authorisation of a medicine for children

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AMED – Initiative on Rare and Undiagnosed Diseases

Published by IRDiRC

Agency For Medical Research and Development Genetic Analysis Initiative on Rare Disease Orphan Drug Development Guideline Regulatory Affairs

Japan Agency for Medical Research and Development (AMED) leads the Initiative on Rare and Undiagnosed Diseases (IRUD) program. The program provides patients across Japan suffering from undiagnosed diseases with a diagnostic strategy that involves genetic analysis

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From lab to patient: journey of a medicine

Published by EMA

Centralised Procedure Committee for Orphan Medicinal Products EMA Drug Development EU Marketing Authorisation Road Map Marketing Authorisation National Procedure Regulatory Affairs Scientific Advice

Follow the journey of a medicine for human use assessed by EMA in this interactive timeline. It explains all stages from initial research to patient access, including how EMA supports medicine development, assesses the benefits and risks and monitors the Safety of medicines.

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From laboratory to patient: the journey of a medicine assessed by EMA

Published by EMA

Committee for Orphan Medicinal Products EMA Drug Development EU Marketing Authorisation Road Map Marketing Authorisation Regulatory Affairs Scientific Advice

This booklet covers medicines for human use that are authorised via EMA through the EU centralised procedure.

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Guideline On Development And Manufacture Of Lentiviral Vectors

Published by EMA

Advanced Therapy ATMP Early Access Support Gene Therapy Lentiviral Vector Regulatory Affairs

This guideline describes quality aspects that are relevant for lentiviral vectors (LV). It applies to LV intended for ex vivo or in vivo application.

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Guideline On The Non-Clinical Studies Required Before First Clinical Use Of Gene Therapy Medicinal Products

Published by EMA

Advanced Therapy Early Access Support First Clinical Use Gene Therapy Non-Clinical Regulatory Affairs

This document defines scientific principles and provides guidance to applicants developing Gene Therapy medicinal products (GTMPs). It focuses on the non-clinical studies required before the first use of a GTMPs in human subjects.

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Clinical Trial Regulation

Published by EMA

Clinical Development Clinical Trial Design Regulatory Affairs

The goal of the Clinical Trial Regulation is to create an environment that is favourable to conducting clinical trials in the EU, with the highest standards of Safety for participants and increased transparency of trial information.

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Paediatric Regulation

Published by EU

Early Access Support EMA Paediatric Regulation Official Journal Paediatric Medicine Paediatric Regulation Regulatory Affairs

The Regulation aims to ensure that medicines for use in children are of high quality, ethically researched and authorised appropriately and improving the availability of information on the use of medicines for children. It aims to achieve this without subjecting children to unnecessary trials or delaying the authorisation of medicines for use in adults.

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Stakeholders interactions – Patients Affairs Staff

Published by IRDiRC

Clinical Development Early Access Support FDA Regulatory Advice Patient Advice Patient Affairs Staff Patient Engagement Patient Support Regulatory Affairs Research and Drug Development

At the FDA, the Patient Affairs Staff (PAS) is devoted to supporting cross-cutting patient engagement activities across the FDA. Its mission is to coordinate and support patient engagement activities across FDA to facilitate awareness and collaboration with patients, their advocates and the FDA

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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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