Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
Department of Health and Human Services
Description
Purpose: Fund clinical research that makes rare disease studies “clinical trial–ready” by:
Developing, testing, and validating fit-for-purpose biomarkers and clinical outcome assessments (COAs)
Defining disease presentation and natural history to inform eligibility criteria, stratification, endpoints, timing of intervention, and power/sample-size calculations
Sponsor: NIH’s National Center for Advancing Translational Sciences (NCATS), …
Attaining effective therapies for rare diseases is challenging due to their low prevalence resulting in fewer patients, clinicians, researchers, and resources compared to common diseases. This leads to gaps in our understanding of a rare disease's natural history, and a dearth of suitable biomar…
Source
Grant ID
PAR:PAR-25-450.html
Agency
Department of Health and Human Services
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