Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional)
Last verified by NonDilute: 2026-04-29. Official notice and agency instructions control.
If you have access to a rare disease patient population and can design a rigorous observational study, FDA will fund you to generate the clinical evidence that de-risks future drug development.
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What this is
The R01 Clinical Trials Optional funding mechanism from FDA/NIH supports rigorous natural history studies in rare diseases where patient data and disease progression are poorly understood. These studies generate the foundational evidence needed by pharmaceutical companies, regulators, and clinicians to design better trials and therapies. Applicants should have institutional research infrastructure, access to patient populations or registries, and expertise in rare disease clinical research or epidemiology. This is a competitive grant typically ranging from $250K–$500K+ annually for 3–5 years.
Who can apply
Academic institutions, non-profit research organizations, and eligible small businesses with institutional review board (IRB) infrastructure and demonstrated access to rare disease patient populations can apply. U.S. domestic organizations are presumed eligible; specific size caps and geographic restrictions are not detailed in this posting.
Topics: rare disease research · natural history studies · clinical data collection · FDA funding · patient registry · unmet medical needs · drug development · longitudinal study
Public-source funding discovery only. This summary is generated from public agency data and may be incomplete or stale. NonDilute is not affiliated with, endorsed by, or acting on behalf of any government agency. Official notices and agency instructions control. NonDilute does not determine eligibility, provide grant-writing advice, or guarantee funding.