Research and Drug Development

The information and resources in this section can help you navigate in the Research and Drug Development area to identify issues classified for:
Disease models,
Disease/Translational resources,
Drug Discovery,
Clinical Development,

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  2. Research and Drug Development
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Initiatives for undiagnosed diseases

Published by IRDiRC

Research and Drug DevelopmentPatient InformationUndiagnosed Disease ProgrammeGenetic ConditionGenotype and Phenotype Matchmaking

The purposes of Undiagnosed Diseases Programs (UDPs) are to provide patients with an unknown genetic condition a diagnosis and to find the correlation between genotype and phenotype; to share globally the information to facilitate the diagnosis through a matchmaking for finding possible second cases

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New-born screening programs

Published by IRDiRC

Research and Drug DevelopmentRare DiseasePatient InformationGenetic ConditionNewborn Sreening Programme

The purpose of NBS is to detect potentially fatal or disabling conditions in newborns as early as possible and possibly before onset of symptoms. Such detection allows the early treatment which may significantly modify the natural history of the disease and potentially prevent developmental delays, physical disabilities and eventually death.

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

Published by

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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Advisory Committee for Therapeutics (ACT) toolkit – Full application form

Published by TREAT-NMD

Translational ResearchResearch and Drug DevelopmentTREAT-NMDClinical DevelopmentClinical Trial DesignPreclinical ResearchTemplatesAdvice

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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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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Repurposing Medicines Toolkit – Guidance for navigating the process

Published by MRCLife Arc

GuidelineRepurposing

This toolkit aims to provide a guide for researchers, charities and others embarking on repurposing projects. It highlights common activities, issues and considerations at each development stage and signpost to other available resources. The content is mostly styled as questions – what should I be considering at each development stage? It is intended to stimulate thinking and not as a step-by-step guide, which reflects the variable nature of repurposing.

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