Published by TREAT-NMD
Translational ResearchTREAT-NMDClinical DevelopmentPreclinical ResearchTemplatesAdviceResearch and Drug DevelopmentPublished by
Advisory Committee for Therapeutics2020 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.
View this resource Bookmark this resourceThis is TREAT-NMD webpage, where it can be found relevant information how to find biomaterials for The basic scientific research being carried out on Neuromuscular Diseases. It can work also as inspiration for investigators conduting reasech in others rare diseases
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AdvicePreclinical ResearchClinical Trial DesignClinical DevelopmentTREAT-NMDResearch and Drug DevelopmentTemplatesTranslational ResearchOnce 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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Patient EngagementResearch and Drug DevelopmentPatient InformationThis guide has been developed with the help of a working group comprising of patient representatives and research funders. Its main goal is to encourage fruitful, sustainable and enduring partnerships between scientists and patient organisa
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Patient InformationResearch and Drug DevelopmentPatient EngagementThe Patient Engagement Resource center is an easy to navigate platform to help researchers get started with patient engagement in research. This toolbox provides helpful resources to help researchers understand the basics of patient engagement, and guide them through the different phases of patient engagement, from planning to conducting and evaluating.
View this resource Bookmark this resourceThe EATRIS Research Management Manual provides general guidance on how to structure and run a translational research project, from project initiation to project closure, to optimize the translational feasibility of your project. It also provides the necessary tools and templates, based on best practice experience and established project management methodologies.
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Early Access SupportRegulatory AffairsAdvanced TherapyATMPs ClassificationCompanies 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 resourceThis document lists the country-specific requirements for informed consents for paediatric clinical trials in Europe
View this resource Bookmark this resourceThe 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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