Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)

What the official notice requires

Pulled from the official notice Foa_Content_of_RFA-FD-25-020.pdf (36 pages) so you can see the requirements at a glance. Each item shows the notice's own words. Open the page to read it in context.

Award size and project length

• Award amounts here are per year · p. 6

  “The additional funding request shall not exceed an additional $250,000 total costs per year (to a maximum total award cost of $900,000 per year) for up to 4 years.” Open page 6

Registrations you need first (3)

• Grants.gov, eRA Commons · p. 3

  “Applicants should be aware that on-time submission means that an application is submitted error free (of both Grants.gov and eRA Commons errors) by 11:59 PM Eastern Time on the application due date.” Open page 3

• SAM.gov · p. 7

  “System for Award Management (SAM) Applicants must complete and maintain an active registration, which requires renewal at least annually.” Open page 7

• Unique Entity ID (UEI) · p. 8

  “o NATO Commercial and Government Entity (NCAGE) Code Foreign organizations must obtain an NCAGE code (in lieu of a CAGE code) in order to register in SAM. o Unique Entity Identifier (UEI) - A UEI is issued as part of the SAM.gov registration process.” Open page 8

Registration is free but can take weeks, so start now, not when the deadline is close. Get-ready checklist

Steps before the deadline

• Letter of intent · p. 2

  “Key Dates Posted Date July 11, 2025 Open Date (Earliest Submission Date) August 22, 2025 Letter of Intent Due Date(s) September 22, 2025 (Optional) September 21, 2026 (Optional) September 20, 2027 (Optional) Application Due Date(s) October 21, 2025, by 11:59…” Open page 2

• Letter of intent · p. 10

  “Letter of Intent Although a letter of intent is not required, is not binding, and does not enter into the review of a subsequent application, the information that it contains allows FDA staff to estimate the potential review workload and plan the review.” Open page 10

What to submit

• Form SF-424 · p. 8

  “Information for the Contact PD/PI should be entered in item 14 of the SF424 Research & Related (R&R) form.” Open page 8

• Audit or financial statements · p. 33

  “Reporting When multiple years are involved, recipients will be required to submit the Research Performance Progress Report (RPPR) annually and financial statements as required in the HHS Grants Policy Statement.” Open page 33

Length limits

• Research Strategy: 12 pages max · p. 10

  “For this specific NOFO, the Research Strategy section is limited to 12 pages.” Open page 10

• Research Strategy: 1 page max · p. 10

  “A resubmission application must include an Introduction Section of the Research Strategy (1 page maximum) addressing the most recent objective review critique (Summary Statement).” Open page 10

• Appendix Section: 3 pages max · p. 11

  “Applications requesting additional funding (up to $250,000 total costs per year) for innovative and efficient trial approaches must submit a clear description and justification as to how they have met the requirements as outlined in the Appendix Section…” Open page 11

• Page limit (see quote): 3 pages max · p. 14

  “Applications requesting additional funding (up to $250,000 total costs per year) for innovative and efficient trial approaches must submit a clear description and justification as to how the requirements as outlined below are met (limited to 3 pages):” Open page 14

Dates in the notice

• July 11, 2025 · p. 2

  “Key Dates Posted Date July 11, 2025 Open Date (Earliest Submission Date) August 22, 2025 Letter of Intent Due Date(s) September 22, 2025 (Optional) September 21, 2026 (Optional) September 20, 2027 (Optional) Application Due Date(s) October 21, 2025, by 11:59…” Open page 2

• May 31, 2028 · p. 3

  “June 2027, 2028 Earliest Start Date July 2026 Expiration Date May 31, 2028 Advisory Council Review Not Applicable Due Dates for E.O. 12372 Not Applicable Required Application Instructions Conformance to all requirements, both in the the Research (R)…” Open page 3

• January 25, 2023 · p. 13

  “Effective for due dates on or after January 25, 2023, the Data Management and Sharing Plan will be attached in the Other Plan(s) attachment in FORMS -H application forms packages.” Open page 13

Mistakes that get applications rejected (6)

• Late or not validated: Late applications will not be accepted for this FOA Applicants are responsible for viewing their... · p. 15

  “Late applications will not be accepted for this FOA Applicants are responsible for viewing their application before the due date in the eRA Commons to ensure accurate and successful submission.” Open page 15

• Sent the wrong way or format: Paper applications will not be accepted. · p. 16

  “Paper applications will not be accepted.” Open page 16

• Missing a required part: Applications that are incomplete, non-compliant and/or nonresponsive will not be reviewed. · p. 17

  “Applications that are incomplete, non-compliant and/or nonresponsive will not be reviewed.” Open page 17

• Outside what the program funds: Applications found to be non-responsive will receive notice that the application will not be reviewed. · p. 17

  “Applications found to be non-responsive will receive notice that the application will not be reviewed.” Open page 17

• Could get your application rejected: Appeals of objective review will not be accepted for applications submitted in response to this NOFO. · p. 27

  “Appeals of objective review will not be accepted for applications submitted in response to this NOFO.” Open page 27

• Sent the wrong way or format: Paper expenditure/FFR reports will not be accepted. · p. 29

  “Paper expenditure/FFR reports will not be accepted.” Open page 29