Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
PAR:PAR-25-450.htmlWhat this funds
- Clinical research that gets rare diseases ready for efficient, well-designed clinical trials by:
- Validating or adapting biomarkers and clinical outcome assessments (COAs) so they’re fit-for-purpose in upcoming trials.
- Defining disease presentation and natural history to inform inclusion/exclusion criteria, stratification, timing of intervention, a…
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…
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