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Grant Analysis

Purpose & Target

The core objective of this grant, RFA-FD-25-001, is to fund clinical trials that evaluate the efficacy and/or safety of medical products to address unmet needs in rare neurodegenerative diseases for both children and adults. The goal is to facilitate drug development, increase the number of approved treatments, and have a broad positive impact on rare disease drug development. This is a SECTOR-SPECIFIC grant, exclusively targeting the Healthcare sector, particularly focusing on research and development for rare neurodegenerative conditions. The target recipients are organizations capable of conducting clinical trials. This includes a wide range of entity types such as higher education institutions, non-profits, for-profit organizations, and various government entities. Geographically, applicants from the United States are eligible, and importantly, non-domestic (non-U.S.) entities (Foreign Organizations) are also explicitly eligible to apply. This implies a global reach for eligible applicants, though the primary context is U.S. regulatory alignment. Key filtering criteria for initial screening include a clear proposal for a clinical trial (not just research), a focus on rare neurodegenerative diseases as defined by FDA, and a commitment to supporting a new product indication or change in labeling. This grant opportunity is recurring, with application cycles expected annually, building upon the FDA's Rare Neurodegenerative Disease Grant Program established under the 'Accelerating Access to Critical Therapies for ALS Act'.

Financial Structure

The financial structure of this grant is designed to support significant clinical trial activities, with clear guidelines on budget limits and cost coverage. Budget Range and Limitations

Base Annual Budget: Applications may request up to $650,000 in total costs (direct and indirect costs) per year for up to four (4) years.
Additional Funding for Innovation: Applicants proposing innovative and efficient trial approaches (e.g., seamless, adaptive, basket, umbrella, platform trials, or data modeling/simulations) may request an additional $250,000 in total costs per year. This can bring the maximum total award to $900,000 per year for up to 4 years. Justification for this additional funding must be provided (max 3 pages).
Budget Justification: Applicants must submit a separate detailed budget breakdown and narrative justification for each year of requested financial support.

Eligible and Ineligible Costs

Eligible Costs: Generally includes all direct costs necessary for the proposed clinical trial, plus applicable indirect costs.
Ineligible Costs: Funds cannot be used for telecommunications and video surveillance services or equipment from specific entities identified by federal law (e.g., Huawei, ZTE, Hytera, Hikvision, Dahua). Payments for individual salaries exceeding the current Executive Level II of the Federal Executive Pay Scale are prohibited.

Matching Fund Requirements

No Cost Sharing: This Notice of Funding Opportunity does not require cost sharing.

Indirect Cost Policies

Negotiated Federal Rate: If an applicant organization has a current Federal indirect cost rate, a copy of the agreement must be provided.
De Minimis Rate: If no established Federal indirect cost rate, a 10% de minimis indirect cost rate of modified total direct costs (MTDC) will be allowed. MTDC excludes equipment, capital expenditures, patient care charges, rental costs, tuition, scholarships, participant support, and subawards/subcontracts over $25,000.
Foreign Organizations: Indirect costs for foreign and international organizations are funded at a fixed rate of 8% of MTDC, with exceptions for the American University of Beirut and the World Health Organization which are eligible for full F&A cost reimbursement.
Domestic with Foreign Consortium: If a domestic organization partners with a foreign component, the foreign component's indirect costs may be included at 8% of MTDC.

Financial Reporting and Audit Requirements

Financial Reporting: Annual Federal Financial Reports (FFRs) must be submitted electronically via the Payment Management System (PMS) within 90 days after the end of the calendar quarter in which the budget period ended. A Final FFR is required within 120 days after the project period expiration date.
Audit Requirements: Recipients are subject to annual audit requirements based on Federal fund expenditure thresholds (specified in 45 CFR 75.501). Foreign recipients are subject to the same audit requirements as for-profit organizations.

Financial Guarantees

No explicit financial guarantees are mentioned, but ongoing funding is contingent on satisfactory performance, compliance with regulatory requirements, and availability of federal funds. Product availability must be evidenced before award.

Eligibility Requirements

To be considered for this grant, applicants must meet the following hard, non-negotiable eligibility criteria: Eligible Organization Types

Higher Education Institutions: Public/State Controlled Institutions of Higher Education, Private Institutions of Higher Education (including Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISIs)).
Nonprofits: Nonprofits with or without 501(c)(3) IRS Status (other than Institutions of Higher Education).
For-Profit Organizations: Small Businesses, and other For-Profit Organizations.
Government Entities: Local Governments (State, County, City or Township, Special District, Indian/Native American Tribal Governments (Federally Recognized or Other than Federally Recognized)), Federal Governments (U.S. Territory or Possession).
Other: Independent School Districts, Public Housing Authorities/Indian Housing Authorities, Native American Tribal Organizations (other than Federally recognized tribal governments), Faith-based or Community-based Organizations, Regional Organizations, and Non-domestic (non-U.S.) Entities (Foreign Organizations).

Specific Qualifications and Regulatory Requirements

Clinical Trials Required: Applications must propose clinical trial(s) addressing unmet needs in rare neurodegenerative diseases, evaluating efficacy and/or safety in support of a new indication or change in labeling.
Product Focus: Must use the generic name of the proposed product(s).
IND/IDE Compliance: For drugs and biologics, the study protocol must comply with 21 CFR 312; the Investigational New Drug (IND) application must be active (not on clinical hold or exempt) at least 30 days prior to the grant application deadline. For devices, the study protocol must comply with 21 CFR 812; the Investigational Device Exemption (IDE) must be approved at least 30 days prior to the grant application deadline.
Medical Foods/NSR Devices: Medical foods not requiring pre-market approval and medical devices classified as non-significant risk (NSR) are exempt from IND/IDE requirements. For NSR devices, a letter from the FDA Center for Devices and Radiologic Health is required.
Sponsor Letter: If the IND/IDE sponsor is not the Principal Investigator (PI), a letter from the sponsor permitting access to the IND/IDE is required.

Definition of Rare Neurodegenerative Disease

The disease must be neurodegenerative: nerve cells in the brain or peripheral nervous system lose function over time and ultimately die; generally incurable, debilitating, and progressive.
The disease must be rare: defined as a condition with a prevalence of fewer than 200,000 people in the United States (or an annual incidence of less than 200,000 for acute diseases). For studies assessing multiple diseases, supportive prevalence data for each is required. An 'orphan subset' of a non-rare disease may be considered with clear justification that the product's use is limited to that subset.

Organizational and PI Registration Requirements

Applicant Organizations: Must complete and maintain active registrations with the System for Award Management (SAM) (including NATO Commercial and Government Entity (NCAGE) Code for foreign organizations), Unique Entity Identifier (UEI) (issued via SAM), eRA Commons, and Grants.gov. These registrations can take 6 weeks or more and must be completed before application submission.
Program Directors/Principal Investigators (PD(s)/PI(s)): Must have an active eRA Commons account and affiliate it with the applicant organization. Their Commons ID must be included in the application.

Exclusion Criteria and Disqualifying Factors

Applications not proposing clinical trials or those exceeding a 4-year project period will be deemed non-responsive.
Late applications will not be accepted.
Duplicate or highly overlapping applications under review at the same time will not be accepted.
Only previously submitted applications to the FDA Rare Neurodegenerative Disease Grants program that received a numeric score will be accepted as a resubmission.

Application Process

Applying for the RFA-FD-25-001 grant requires careful adherence to specific timelines, documentation, and submission procedures. It's crucial to plan ahead for registrations and material preparation. Application Deadlines and Submission Windows

Letter of Intent Due Date: September 21, 2025. While not required or binding, it's helpful for FDA staff to estimate workload.
Application Due Date: October 21, 2025, by 11:59 PM Eastern Time.
Open Date (Earliest Submission): August 22, 2025.
Late applications will not be accepted.

Application Procedure and Steps

Registration: Ensure the applicant organization and all Program Directors/Principal Investigators (PD/PIs) are fully registered with SAM.gov (System for Award Management), possess a Unique Entity Identifier (UEI), and have active accounts in eRA Commons and Grants.gov. These registrations can take 6 weeks or more, so initiate them well in advance.
Access Forms: Obtain the application package through NIH ASSIST, Grants.gov Workspace, or an institutional system-to-system (S2S) solution.
Preparation: Follow the detailed instructions in the SF424 (R&R) Application Guide, supplemented by the specific instructions in this NOFO. Pay close attention to page limitations and required sections.
Submission: Submit the completed application electronically via Grants.gov.
Tracking: After submission, actively track the application's status in eRA Commons. Correct any errors found during initial checks and resubmit by the deadline.
Viewing Application: Applicants are responsible for viewing their application in eRA Commons before the due date to ensure accurate and successful submission.

Required Documentation and Materials

Core Application Forms: SF424 (R&R) forms including Cover, Project/Performance Site Locations, Other Project Information, Senior/Key Person Profile, R&R Budget, R&R Subaward Budget, and PHS 398 Cover Page Supplement.
PHS 398 Research Plan: This is a critical section (limited to 12 pages for the Research Strategy). It must include:
- Rationale: Detailing existing knowledge, knowledge gaps, and how the study supports product approval or development.
- Rare Neurodegenerative Disease Population/Prevalence: A subsection within 'Rationale' providing documentation and prevalence calculations for the rare neurodegenerative disease.
- Support of Product Development: A subsection explaining how the study supports new product indications or labeling changes.
- Study Design: Including a clear hypothesis, aims, experimental design, and a Study Monitoring Plan (detailing monitoring parties, what will be monitored, and frequency).
- Inclusion of Patient Input: Describing plans for early and ongoing patient/stakeholder engagement.
- Investigator, Infrastructure, and Financial Resources: Details on team competence, institutional support, product availability, and sustainability.
- Ability to Advance the Current Field: Discussing potential impact and innovative aspects.
Letters of Support: Must be uploaded (specifically to PHS 398 Research Plan Form, line 9) from:
- Study Sites: Confirming site support, resources, infrastructure, and estimated eligible patient numbers.
- Product Supplier: Evidence of product availability in the required form and quantity.
- Patient Engagement: From patient(s)/caregiver(s)/patient organizations describing meaningful input.
Appendices: Only limited materials are allowed, primarily:
- Full final protocol (IND/IDE submitted protocol).
- Informed Consent forms and other subject information.
- Innovative and Efficient Approach Justification: (If additional funding is requested, up to 3 pages).
Data Sharing Plan: Required for all applications, regardless of direct costs requested.
Resubmission Specifics: An 'Introduction' (1 page max) addressing previous review critiques, with the prior Summary Statement included as an Appendix.

Support Offered

The FDA provides contact information for scientific/research inquiries (Katherine Needleman) and financial/grants management questions (Kimberly Pendleton) to assist applicants during the application process.

Project Implementation Timeline and Reporting

Grant Duration: Maximum four (4) years. Continued funding in subsequent years is contingent on satisfactory performance, regulatory compliance, and fund availability.
Annual Reporting: Recipients must submit an annual Research Performance Progress Report (RPPR) via eRA Commons 60 days before the next budget period starts, including a detailed budget.
Financial Reporting: Annual Federal Financial Report (FFR) must be submitted electronically via PMS 90 days after the budget period ends.
Closeout Reporting: A Final RPPR, invention statement, and expenditure data are required for closeout within 120 days after the project period's expiration date.

Post-Award Requirements

Compliance: Adherence to the HHS Grants Policy Statement, 45 CFR 75, and all terms and conditions specified in the Notice of Award.
Human Subject Protection: Protocols must be reviewed and approved by an Institutional Review Board (IRB) or Independent Ethics Committee (IEC). A single IRB is required.
Key Personnel Training: All key personnel must receive appropriate training in human subject protection.
Mandatory Disclosure: Recipients must report any violations of federal criminal law involving fraud, bribery, or gratuity that could affect the federal award.
Prior Approvals: Changes in grantee organization, significant re-budgeting, changes in scope or objectives, deviation from terms, changes in key personnel, or significant disengagement by PD/PI require prior written approval from FDA.

Evaluation Criteria

Applications for this grant undergo a multi-step evaluation process, starting with a responsiveness review and followed by a scientific merit review. Funding decisions are based on scientific/technical merit, fund availability, and relevance to program priorities. Program Responsiveness Review Criteria (Initial Screening) Applications are first checked against the following criteria. Failure to meet any of these will result in the application being deemed non-responsive and not reviewed: - Clinical Trial Focus: Application must propose clinical trials providing safety and/or efficacy data for rare neurodegenerative diseases, using the generic name of the product(s). - Project Duration: The requested time for the project must not exceed 4 years. - Disease Rarity Documentation: The 'Rationale' section of the 'Research Strategy' must include clear documentation (with prevalence calculations and citations) that the disease is a rare neurodegenerative disease (as defined in 21 U.S.C. 360ee). - Product Development Support: The 'Rationale' section must explain how the proposed clinical trial will support a new indication or change in labeling for a product(s). - Regulatory Compliance: The proposed protocol must comply with 21 CFR 312 (drugs/biologics) or 21 CFR 812 (devices). The IND must be active or IDE approved at least 30 days before the application deadline. For medical foods/NSR devices, appropriate documentation is needed. - Sponsor Approval: If the IND/IDE sponsor is not the PI, a letter from the sponsor permitting IND/IDE access must be included. - Required Appendices and Letters: The application must include the full protocol, informed consent forms, and justification for innovative approaches (if applicable) as appendices. Letters of support for product availability, study sites, and patient engagement are mandatory. - Format Compliance: Adherence to all page limits, font sizes, and margin requirements specified in the Application Guide. Scored Review Criteria (Weighted Equally) Responsive applications will be reviewed for scientific and technical merit by a panel of experts. An impact score will be assigned based on the likelihood of the project to exert a sustained, powerful influence on the research field, considering the following equally weighted criteria: - Rationale: Evaluates the scientific soundness of the proposal concerning the rare neurodegenerative disease(s), the current state of knowledge, and the likelihood of facilitating a clinical trial for new indications or labeling changes. This includes the importance of addressing knowledge gaps and critical barriers, and how the study provides essential data for product approval/development. - Study Design: Assesses the quality and appropriateness of the study design, research methodology, and data analyses. This includes the clarity of the hypothesis, aims, experimental design, appropriateness of eligibility criteria and endpoints, data collection/analysis plans, and potential for broad impact. Consideration is given to innovative trial designs (e.g., basket, umbrella, platform trials) and statistical power. Protection of human subjects and adherence to Good Clinical Practice are also evaluated. - Inclusion of Patient Input: Examines the extent and meaningfulness of patient and caregiver perspectives integrated into the planning and design of the clinical study, including efforts to reduce patient burden and improve trial feasibility. - Investigator, Infrastructure, and Financial Resources: Reviews the competence of the PI(s) and team, ability to successfully recruit participants, manage the project within budget and time, institutional support, existing research networks, product availability, and sustainability plans for future phases of development. - Ability to Advance the Current Field: Focuses on the project's potential to shift current research or clinical practice paradigms, significantly influence product development, and introduce novel concepts or methodologies (e.g., adaptive designs, modeling, simulations). Includes sustainability plans beyond the proposed funding period. Additional Review Criteria (Considered but Not Separately Scored)

Protections for Human Subjects: Justification for involvement, adequacy of protection against risks, potential benefits, importance of knowledge gained, and data/safety monitoring for clinical trials.
Inclusion of Women, Minorities, and Individuals Across the Lifespan: Appropriateness of plans for inclusion/exclusion based on sex/gender, race, ethnicity, and age.
Biohazards: Assessment of potential hazards and proposed protections.
Resubmissions/Renewals/Revisions: Evaluation of responses to previous critiques, progress in past funding periods, or appropriateness of scope expansion.

Compliance & Special Requirements

This grant involves rigorous compliance and unique considerations due to its focus on clinical trials and association with the FDA. Regulatory Compliance Requirements

Human Subjects Protection: All research involving human subjects must comply with 45 CFR Part 46 (Office for Human Research Protections (OHRP)) and 21 CFR Part 50 and 56 (FDA regulations). This includes obtaining appropriate IRB/IEC approval. A single IRB and Federal Wide Assurance (FWA) are required for all performance sites.
Good Clinical Practice (GCP): Clinical protocols should comply with ICH E6 Good Clinical Practice Consolidated Guidance for ethical and scientific quality.
IND/IDE Regulations: Adherence to 21 CFR 312 for drugs and biologics, and 21 CFR 812 for devices, is critical for the product development aspect of the clinical trials.
Federal Awardee Performance and Integrity Information System (FAPIIS): Applicants are subject to FAPIIS requirements, meaning information about integrity and performance under Federal awards may be reviewed.
Telecommunications and Surveillance Equipment Prohibition: Grant funds (including direct, indirect, cost share, and program income) cannot be used to procure or obtain, or extend/renew contracts for, specific telecommunications and video surveillance services or equipment from entities like Huawei, ZTE, Hytera, Hikvision, or Dahua.
Non-Discrimination: Recipients must comply with federal civil rights laws prohibiting discrimination based on race, color, national origin, age, sex, disability, and in some circumstances, religion, conscience, and sex (including gender identity, sexual orientation, and pregnancy).
Accessibility and Inclusion: Programs must provide meaningful access to persons with limited English proficiency and be accessible to individuals with disabilities.
Sexual Harassment: Administer programs in an environment free of sexual harassment, aligning with FDA's policy on extramural research.

Data Protection and Privacy Regulations

Certificates of Confidentiality: Identifiable, sensitive information collected or used in the research is automatically protected by a Certificate of Confidentiality under 42 U.S.C. 241(d).
Data Management and Sharing: A Data Management and Sharing Plan is required for all applications, consistent with the HHS Policy for Data Management and Sharing. Recipients are required to implement the approved plan.

Risk Management Expectations

Study Monitoring Plan: A comprehensive 'Study Monitoring Plan' is a required part of the 'Study Design' section. This plan must detail the parties responsible for monitoring (e.g., Data and Safety Monitoring Board (DSMB), Study Monitoring Committee (SMC), Independent Medical Monitor (IMM)), what will be monitored, and the frequency. Monitoring activities must be appropriate to the study's risks and complexity.

Unique Aspects and Strategic Opportunities

Innovation Encouraged: The grant strongly encourages innovative clinical trial designs (e.g., seamless, adaptive, basket, umbrella, platform trials) and innovative methods (e.g., data modeling and simulations). Projects utilizing these approaches are eligible for additional funding (up to $250,000 per year) and are seen as vital for expediting drug development.
Patient Engagement: A significant emphasis is placed on early and ongoing patient and caregiver engagement in trial design, aiming to improve protocol design and reduce patient burden.
Collaborative Efforts: The FDA encourages the use of established infrastructure and resources (e.g., clinical trial networks, data standardization, analytics, and sharing platforms) and collaborative efforts between diverse stakeholders (e.g., industry, academia, patient organizations).
Product Focus: The grant is specifically designed to support clinical trials that move medical products towards a new indication or change in labeling, directly contributing to regulatory approval of rare disease treatments.

Knowledge Sharing Expectations

Publications: When research findings are published, the author's final manuscript of the peer-reviewed scientific publication resulting from FDA-funded research must be submitted to the NIH National Library of Medicine's (NLM) PubMed Central (PMC).
Acknowledgment of Federal Support: Any statements, publications, or materials describing projects funded by this grant must clearly state the percentage and dollar amount of federal and non-governmental funding.

Grant Details