FDA Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers
U.S. Department of Health and Human Services — Food and Drug Administration, Office of Orphan Products Development
Up to ~$500K/yr for 3 years
Partner with FDA scientists to develop drugs for the rarest cancers
FDA's Office of Orphan Products Development funds research into novel regulatory and scientific approaches to accelerate therapeutic development for ultra-rare cancers — typically defined as cancers affecting fewer than 1,000 U.S. patients. Eligible applicants include for-profit companies, small businesses, universities, hospitals, and nonprofits. Awards are structured as cooperative agreements (U01), meaning FDA program staff actively collaborate with and provide regulatory guidance to awardees throughout the project. This is a rare opportunity for small drug developers and biotech companies to partner directly with FDA scientists on early-stage cancer drug development strategy.
- Funding type
- Grant
- Level
- Federal
- Amount range
- $250,000 – $1,500,000
- Realistic amount
- FDA OOPD cooperative agreements for novel methodology development typically fall in the $300K–$500K/year range for 2–3 y…
- Deadline
- June 15, 2026 (11:59 PM Eastern)
- Status
- active
- States
- Nationwide
Who qualifies
- For-profit companies, small businesses, biotech, and pharmaceutical companies eligible
- Universities, hospitals, nonprofits, and research institutions also eligible
- Project must focus on ultra-rare cancers (typically <1,000 U.S. patients)
- Active SAM.gov registration required
- PI must have eRA Commons ID; institution must have eRA Commons registration
- U.S.-based research activity (cooperative agreement requires close FDA partnership)
What it covers
Eligible expenses
- Personnel costs (salaries and benefits for scientific, technical, and administrative staff)
- Consultant costs for specialized expertise
- Equipment necessary for the research (with justification)
- Supplies and laboratory materials
- Travel to scientific meetings and FDA coordination meetings
- Patient-related costs for clinical or translational studies
- Contractual costs for core facilities and CROs
- Indirect (facilities and administrative) costs at the institution's negotiated rate
Ineligible expenses
- Drug manufacturing or commercial-scale production costs
- Regulatory filing fees (e.g., NDA, BLA, IND application fees)
- Clinical trial costs at commercial IND scale unless specifically tied to research aims
- Marketing or commercialization activities
- Costs for activities that have already occurred (pre-award costs without prior FDA approval)
- Foreign travel without prior approval in budget justification
How to apply
-
1
Register in SAM.gov
Obtain or verify your UEI (Unique Entity Identifier) in SAM.gov. Annual renewal is required. SAM.gov registration can take 7–10 business days for new registrants. Without a valid SAM.gov registration, Grants.gov submission is blocked.
~4 hrs
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2
Register in eRA Commons and Grants.gov
Because this is an FDA-administered grant using NIH peer review systems, applicants need an eRA Commons account. The PI must have an eRA Commons ID, and the institution's grants administrator must be registered. Grants.gov system registration is also required for submission.
~4 hrs
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3
Review solicitation and contact FDA program staff
Read the full solicitation (RFA-FD-26-004) carefully. FDA OOPD program staff are available for pre-application consultations to clarify scope and scientific objectives. Contact FDA scientific review staff before investing significant time in an application — they can indicate fit with program priorities. Note that the ultra-rare cancer focus means applicants must demonstrate the target indication meets FDA's ultra-rare threshold.
~4 hrs
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4
Prepare application components
FDA OOPD cooperative agreement applications follow the SF424(R&R) format through Grants.gov. Key components: Project Summary/Abstract (30 lines), Project Narrative (2 pages), Specific Aims (1 page), Research Strategy (usually 12–15 pages for R01/U01 format), Human Subjects protection documentation, Resource and Environment, Budget and Justification (modular or detailed), Biosketches for all key personnel, Letters of Support from collaborating clinical sites or patient advocacy organizations. For U01 cooperative agreements, a Research Coordination Plan describing how FDA staff will collaborate is typically required.
~4 hrs
-
5
Submit through Grants.gov
Submit the completed application package through Grants.gov using the FORMS-H application package or the current forms version specified in the solicitation. Verify submission receipt within 2 business days. Grants.gov generates a tracking number upon submission; watch for agency validation errors that require resubmission within the submission window.
~4 hrs
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6
Peer review and funding decision
FDA OOPD applications are reviewed by a special emphasis panel (SEP) convened in partnership with NIH. Review criteria typically include: Significance (novelty and impact on rare cancer drug development), Approach (rigor, feasibility, innovation), Innovation, Investigator(s) (expertise, especially FDA regulatory science background), and Environment. Scores are assigned on the NIH 1–9 scoring system. Funding decisions are made by OOPD leadership following the review panel's recommendations.
~4 hrs
Pre-application FDA OOPD consultations are underused by industry. A 30-min call with program staff before investing 200+ hours can confirm you're targeting the right ultra-rare cancer and that your regulatory science approach aligns with OOPD's current priorities.
Deadline & timing
Single application deadline. RFA-FD-26-004 opened May 4, 2026 and closes June 15, 2026. Applications must be submitted through Grants.gov by 11:59 PM Eastern. On-time submission is required — late applications are not accepted except under documented system failures. FDA OOPD typically runs one funding cycle per solicitation; no rolling admissions.
Programs that stack well
- NIH National Cancer Institute (NCI) R01 Or R21 Grants For Complementary Research Aspects
- FDA SBIR/STTR Grants For Product Development Phases
- DOD Congressionally Directed Medical Research Programs (CDMRP) Rare Cancer Funding
- Private Foundations Supporting Rare Cancer Research (Alex'S Lemonade Stand, Pediatric Cancer Research Foundation)
- Orphan Drug Designation (Not A Grant But A Regulatory Benefit Reducing Development Costs)
Related programs
Last reviewed 2026. GrantCompass is an independent funding-discovery tool and is not affiliated with any government agency. Always confirm details on the official program page.