Reissue of RFA-FD-22-001 - Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases
Funds natural history studies that track how a rare disease unfolds over time, to close data gaps blocking new treatments for it.
Before anyone can design a trial for a rare disease, someone has to know how that disease actually behaves over time — how fast it progresses, what changes first, what a meaningful improvement even looks like. For many rare diseases nobody has collected that information yet.
This notice funds building it: natural history studies that produce high-quality, interpretable data on a specific rare dis…
Natural history studies producing high-quality, interpretable data on a rare disease or a group of related rare diseases with shared underlying biology, aimed at closing knowledge gaps and feeding into medical product development. Clinical trials are optional under this activity code.
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