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

View this resource Bookmark this resource

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

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

European Network for Health Technology Assessment

Published by IRDiRC

EUnetHTA HTA Bodies HTA Network Regulatory Affairs

EUnetHTA was established to create an effective and sustainable network for HTA across Europe – we work together to help develop reliable, timely, transparent, and transferable information to contribute to HTA in European countries.

View this resource Bookmark this resource

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.

View this resource Bookmark this resource

PMDA consultations

Published by IRDiRC

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

PMDA offers consultations to give guidance and advice on clinical trials of drugs, medical devices, and regenerative medical products as well as on data for regulatory submissions. For designated orphan drugs, PMDA offers Priority consultation.

View this resource Bookmark this resource

Sakigake Designation System

Published by IRDiRC

Early Access Support Japan MHLW or PMDA Regulatory Orphan Drug Regulatory Affairs Sakigake

SAKIGAKE aims at shortening premarket review period for innovative new medical products that satisfy certain criteria, such as severity of intended indication, by designating such products during the early stages of development, and providing prioritized consultation services and premarket pharmaceutical affairs review.

View this resource Bookmark this resource

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.

IMT Feedback

Did the IMT help you in finding the resource you needed?