Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01) Clinical Trials Required
🏛 Food and Drug Administration (HHS-FDA)
✓ Free, no account · Source: Grants.gov · Last verified Jul 16, 2026
Can you apply?
This grant is for research institutions, academic medical centers, and nonprofits conducting clinical trials focused on rare neurodegenerative diseases. Applicants must be eligible NIH grantees (typically 501(c)(3) organizations, government agencies, or other authorized entities). The program supports Phase 1–3 clinical trial investigations that address unmet therapeutic needs in rare neurodegenerative conditions. Principal investigators should have relevant expertise in clinical research, neurobiology, or related fields. This is an R01 mechanism, meaning institutional backing and research infrastructure are expected. Geographic scope is U.S.-based organizations, though certain international collaborations may be permitted. The program encourages applications from diverse research teams and institutions serving underrepresented populations in clinical research.
Not the right fit? Find grants for your organization in 5 questions →
Program description
The purpose is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare neurodegenerative diseases for children and adults. Through the support of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare neurodegenerative diseases and exert a broad and positive impact on rare disease drug development.
Who can apply
Eligible applicants
How to apply
Application links
Required documents
- NIH R01 Application Form (SF-424 R&R)
- Project Narrative (specific aims, significance, innovation, approach, impact)
- Detailed Budget and Budget Justification (3–5 year project period)
- Biographical Sketches for key personnel (4-page NIH format)
- Letters of Support from collaborating institutions and clinical trial sites
- IRB approval or approval-pending letter
- Preliminary Data and Published Literature supporting the unmet need
- Clinical Trial Protocol or detailed protocol summary
- Data and Safety Monitoring Plan
- Regulatory Documentation (FDA guidance alignment, IND status if applicable)
- Letters from patient advocacy groups or patient registry partners (recommended)
- Current and Pending Support documentation
Program contact
- 👤 Terrin Brown Grantor
- 📧 terrin.brown@fda.hhs.gov
- 📞 2404027610
Funding track record
Recent awards under CFDA 93.103 from the last 3 years — real organizations that won funding through this same program.
Top 10 Largest Recent Awards
-
$121,795,918
-
$76,105,626
-
$50,217,964
-
$47,940,304
-
$36,000,000
-
$35,573,997
-
$35,391,995
-
$30,732,300
-
$23,332,999
-
$21,347,288
Top States by Funding
- AZ 3 awards $131.4M
- MD 7 awards $108.7M
- CA 9 awards $106.5M
- VA 5 awards $96.6M
- PA 10 awards $77.4M
Source: USAspending.gov — federal spending transparency. Data covers last 3 years.
Funding history
Annual funding for this program — Federal obligations (CFDA 93.103). How funding has trended year over year.
| 2016 | $170,482,435 | |
| 2017 est. | $208,900,832 | |
| 2018 | $173,077,408 | |
| 2019 | $198,507,896 | |
| 2020 | $212,448,590 | |
| 2021 | $218,918,739 | |
| 2022 est. | $255,910,458 | |
| 2023 est. | $246,894,600 |
FAQ
What types of rare neurodegenerative diseases are eligible for this program?
The program typically supports clinical trials for less common neurological conditions including but not limited to primary progressive aphasia, behavioral variant frontotemporal dementia, progressive supranuclear palsy, corticobasal degeneration, and other conditions with limited existing treatment options. Check the specific funding announcement for the current disease priority list.
Who can serve as a principal investigator?
PIs typically must hold a doctorate (MD, PhD, DVM, or equivalent) and have demonstrated research capability. Early-stage investigators are often encouraged to apply, and collaborations with established senior scientists are supported.
What is the timeline for clinical trial completion?
R01 awards typically support 3–5 year project periods. Clinical trials may extend beyond the funding period if reasonable milestones are met during the award cycle.
How competitive is this funding mechanism?
R01 mechanisms are highly competitive, typically funded at 10–20% of applications submitted. Strong preliminary data, rigorous trial design, and institutional commitment are critical.
What is the typical funding range?
R01 awards for clinical trials commonly range from $200,000–$500,000 per year, depending on trial complexity, sample size, and multisite coordination. Budget justification must be detailed and evidence-based.
💡 Tips for applicants
- Demonstrate a clear unmet medical need: Use published literature and preliminary data to show that existing treatments are inadequate or absent for your target disease population.
- Design a rigorous, feasible trial protocol: Include detailed statistical power analyses, realistic recruitment timelines, and contingency plans for participant retention. FDA input on trial design may strengthen your application.
- Secure institutional commitment and letters of support: Include evidence of IRB approval pathways, clinical site readiness, and resource availability (lab space, equipment, personnel).
- Build a multidisciplinary team: Include clinical experts, biostatisticians, patient advocates, and if applicable, industry partners or patient registries to enhance credibility.
- Address diversity and inclusion: Demonstrate commitment to recruiting participants from underrepresented groups in your trial. Explain how trial design accommodates various populations.
⚠️ Common mistakes
Applications often fail due to weak preliminary data or insufficient evidence that the proposed intervention addresses a genuine unmet need in the rare disease population. Additionally, applicants frequently underestimate trial complexity and overestimate recruitment feasibility; reviewers are skeptical of timelines that don't account for the difficulty of enrolling patients with rare conditions. Finally, poor alignment between the proposed trial and FDA guidance on clinical trial endpoints or regulatory pathways can lead to rejection.
Similar grants
- ROLLING Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required) — Food and Drug Administration
- OPEN Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required) — Food and Drug Administration
- OPEN Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 – Clinical Trial Optional) — National Institutes of Health
- OPEN Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed) — National Institutes of Health
- OPEN NIH Research Project Grant (Parent R01 Clinical Trial Required) — National Institutes of Health