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

Published by EATRIS

NIHResearch and Drug DevelopmentDrug Discovery GuidelineSmall MoleculeDrug RepurposingTarget Validation Drug DevelopmentNCATS

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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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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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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EMA Framework for stakeholder’s engagement – patients, academia, healthcare professionals

Published by IRDiRC

Research and Drug DevelopmentRegulatory AffairsEarly Access SupportScientific AdviceStakeholder EngagementHealthcare Professional

The Agency has been interacting with its stakeholders since its inception. These stakeholder relations have evolved over time and the type and degree of interaction is varied depending on the stakeholder groups and fields of Agency activity.

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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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