A decade of optimizing drug development for rare neuromuscular disorders through TACT

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Advisory Committee for Therapeutics

2020 marks the tenth anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT), a group of multidisciplinary experts that evaluates drug development programmes for rare neuromuscular diseases and identifies pitfalls. Here, we discuss the experience with TACT based on its reviews of more than 50 applications and its potential as a model for other rare disorders.

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Once the pre-application form has been accepted, the applicant will be asked to submit a more detailed application form. This will ensure that the review panel will have sufficient information to be able to comprehensively review the application for advice. Depending on the disease area, the core committee should agree consensus on the questions to be included as part of an ACT application form.

This report to the applicant template can be used in conjunction with the ACT toolkit. For further information, please see the ACT toolkit. 

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Informed Consent for Paediatric Trials in Europe

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This document lists the country-specific requirements for informed consents for paediatric clinical trials in Europe

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Guideline on pharmaceutical development of medicines for paediatric use

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The principles of this guideline should be considered during the pharmaceutical development of all

paediatric medicines as proposed in marketing-authorisation applications (MAAs) or applications to

extend or vary marketing authorisations to the paediatric population (MAVs)

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Guideline on good pharmacovigilance practices (GVP) Product- or Population-Specific Considerations IV: Paediatric population Guideline on good pharmacovigilance practices (GVP)

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Guidance on the conduct of pharmacovigilance for medicines used by paediatric population. It is directed towards marketing authorisation holders and competent authorities. It is also

of relevance to all those involved in the conduct of paediatric clinical trials.

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Assent / Informed Consent Guidance for Paediatric Clinical Trials with Medicinal Products in Europe

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Developed by Enpr-EMA’s Working Group on Ethics, this document is intended to be used as an overview tool of the contents for assent/informed consent forms for all stakeholders (such as patients, sponsors and

investigators) to support the conduct of high quality paediatric clinical trials in Europe across all paediatric age groups, from birth to less than 18 years of age.

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EnprEMA Network Database

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This database includes research networks and centres with recognised expertise in performing clinical studies in children. It is part of the European network of paediatric research at the European Medicines Agency (Enpr-EMA).

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European YPAG Network

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The network was established to support the development of new Young Person Advisory Groups (YPAGs) within Europe. The main aim of eYPAGnet is to provide researchers with a variety of opportunities to work with children and young people in the design and conduct of paediatric clinical trials.  Here are just some of the services we offer.

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SPIRIT PRO Extension for inclusion of patient-reported outcomes in clinical trials protocols

Published by ECRIN

Clinical Trials ToolkitPCOM

Extension of the SPIRIT (Standard protocol items: recommendations for interventional trials) guideline, SPIRIT PRO provides guidelines for inclusion of patient-reported outcomes in clinical trial protocols.

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