Research and Drug Development

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Advisory Committee for Therapeutics (ACT) toolkit

Published by UNEW

Translational ResearchResearch and Drug DevelopmentTREAT-NMDClinical DevelopmentClinical Trial DesignPreclinical ResearchTemplatesAdvice

The Advisory Committee for Therapeutics (ACT) toolkit provides procedural advice and guidance to replicate the successful TREAT-NMD Advisory Committee for Therapeutics (TACT) model in other rare disease communities. An ACT can provide independent and objective advice to industry and academia, on a real-life therapeutic pathway of drug development programmes. An ACT review can help an applicant to position a candidate compound along a realistic and well-informed pathway to clinical trial and eventual registration. 

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Early Drug Discovery and Development Guidelines: For Academic Researchers, Collaborators, and Start- up Companies

Published by EATRIS

Research and Drug DevelopmentDrug Discovery GuidelineSmall MoleculeDrug RepurposingTarget Validation Drug DevelopmentNCATSNIH

This manual contains guidelines to develop therapeutic hypotheses, target and pathway validation, proof of concept criteria and generalized cost analyses at various stages of early drug discovery. Various decision points in developing a New Chemical Entity (NCE), description of the exploratory Investigational New Drug (IND) and orphan drug designation, Drug Repurposing and drug delivery technologies are also described and geared toward those who intend to develop new drug discovery and development programs.

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AMED Clinical Innovation network – Diseases-related Registries

Published by IRDiRC

Research and Drug DevelopmentClinical DevelopmentPatient RegistryAgency For Medical Research and DevelopmentJapanInitiative on Rare DiseasesClinical Innovation NetworkCinical Trial

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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EURORDIS’ Community Advisory Boards

Published by IRDiRC

Research and Drug DevelopmentRare DiseasePatient InformationPatient OrganisationEurordisCommunity Advisory BoardPatient AdvocateMedical ResearchOrphan Drug Development

Patient Community Advisory Boards are groups established and operated by patient advocates. They facilitate discussions in a safe harbour setting and under strict rules, an MOU and CA on the latest developments and challenges related to medical research and procedures in a disease area with the company or body conducting the research. The CAB can cover from preclinical to post marketing topics. The CAB depending on the areas of discussion can invite scientific expert to serve as advisors.

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