FDA Office of Orphan U01 RFA Rare Neurodegenerative Disease User Manual
- June 1, 2024
- FDA Office of Orphan
Table of Contents
FDA Office of Orphan U01 RFA Rare Neurodegenerative Disease
Natural History, Clinical Outcome Assessment, and Biomarker Studies of Rare Neurodegenerative Diseases:
Application Instructions and Helpful Hints
OOPD has posted general instructions and helpful hints for applying for natural history and clinical trial grants which provide more details to help navigate the application process. Specific items of importance or those regarding this RFA explicitly are noted below.
Application Due Date
The application submission deadline for this Request for Application (RFA) is May 6, 2023 by 11:59 PM Eastern Time. Late applications will not be accepted.
Pre-Application Registrations
- Helpful Hints: Required registrations for System for Award Management (SAM) , eRA Commons , and Grants.gov can take 6 weeks or more, so applicants are encouraged to begin this process as outlined in the RFA well before the application submission date.
General Application Instructions
Applicants should refer to the SF424 (R &R) Application Guide posted by NIH for detailed instructions on completing the SF424 (R&R) forms.
SF424 (R &R) “APPLICATION FOR FEDERAL ASSISTANCE” (Pages 1-2):
- Only “New” applications will be accepted.
- The title field is limited to 200 characters, including the spaces between words and punctuation. Use abbreviations as needed to ensure that the title is descriptive.
- Total Federal Funds Requested: Enter total (direct and indirect) federal funds requested from OOPD for the entire project period for a maximum of 4 years of support.
SF424 “RESEARCH & RELATED Other Project Information”: Budget:
FDA’s Orphan Products Grants Programs use the Research & Related (R&R) Budget Component. The duration of the project period is anticipated to be up to four years. Application budgets are not limited but should reflect the actual needs of the proposed project including both direct and indirect costs for each year requested.
Helpful Hint: Applicants must provide a detailed budget for each requested year and attach a budget justification. Failure to include a well justified budget is a frequent weakness of grant applications.
SF424 “PHS 398 Research Plan”: Research Strategy:
This section is limited to 12 pages. FDA does not follow the order/headings that are included in the NIH’s 424 R&R Application Guide. The following sections should be included under the Research Strategy section (see RFA for additional details on each scoring criteria):
- Rationale
- Study Design/Data Quality and Interpretability
- Inclusion of Patient Input
- Investigator(s), Infrastructure and Financial Resources
- Ability to Advance the Current Field
Helpful Hint: In the Rationale Section of the Research Strategy portion of the grant proposal, applicants should also include subsections with the specific headings Rare Disease Population/Prevalence and Support of Product Development. Under the subsection Rare Disease Population/Prevalence , applicants should include a description of why the rare disease is a neurodegenerative disease. As part of the programmatic review, FDA will evaluate this rationale to ensure the rare disease is a neurodegenerative disease.
Helpful Hint: The Study Design/Data Quality and Interpretability section of the Research Strategy should include a subsection with the heading Study Monitoring Plan.
Letters of support – The following should be included (see RFA for additional details):
- Study Sites
- Patient Engagement
- Intellectual Property (if applicable)
Appendix – The following should be included (see RFA for additional details):
- Protocol: The full final protocol must be provided.
- Informed Consents: Consent forms, assent forms, and any other information given to a subject must be provided and must comply with all elements of Human Subject Research per 21 CFR 50.25.
Useful Links
- FDA Office of Orphan Products Development
- FDA Orphan Products Grants Program
- FDA Rare Neurodegenerative Disease Grant Program
- Grants Forms Library
- Grants.gov Applicant FAQs
- Grants.gov Submitting Your Application
- Grants 101
- Rare Diseases: Natural History Studies for Drug Development, Guidance for Industry
- Rare Diseases: Common Issues in Drug Development
- FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making
- Guidance for Industry: E11, Clinical Investigation of Medicinal Products in the Pediatric Population.
- About Biomarkers and Qualification
- Digital biomarkers: Convergence of digital health technologies and biomarkers
- Draft Guidance for Industry, Investigators, and Other Stakeholders: Digital Health Technologies for Remote Data Acquisition in Clinical Investigations
FAQ
What is the application deadline for the RFA? The application submission deadline is May 6, 2023, by 11:59 PM Eastern Time. Are there any pre- application registrations required? Yes, registrations for SAM, eRA Commons, and Grants.gov are required and can take 6 weeks or more. What should be included in the Research Strategy section? The Research Strategy section should include Rationale, Study Design/Data Quality and Interpretability, Inclusion of Patient Input, Investigator(s), Infrastructure and Financial Resources, and Ability to Advance the Current Field. Is there a page limit for the Research Strategy section? Yes, the Research Strategy section is limited to 12 pages. Where can I find additional details on each scoring criteria and application instructions? Additional details can be found in the RFA document provided by OOPD.