grant

Innovative designs, extrapolation, simulation methods and evidence-tools for rare diseases addressing regulatory needs (INVENTS)

Organization UKRILocation United Kingdom
UKRIUK ResearchGrantActive
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The evaluation of new medicines for rare diseases (RD) including rare paediatric RDs is challenging for several reasons, among which are the small patient sample sizes, heterogeneity of patients and diseases and heterogeneity in disease knowledge. Due to these difficulties, access to effective treatments and the number of treatment options are…

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Innovative designs, extrapolation, simulation methods and evidence-tools for rare diseases addressing regulatory needs ( | Dev Procure