RFA-NS-26-001-Full-Announcement.html

Department of Health and Human Services

Part 1. Overview Information

Participating Organization(s)

National Institutes of Health (NIH)

Components of Participating Organizations

National Institute of Neurological Disorders and Stroke (NINDS)

Office of The Director, National Institutes of Health (OD)

Funding Opportunity Title

Amyotrophic Lateral Sclerosis (ALS) Intermediate Patient Population Expanded Access (U01 Clinical Trial Required)

Activity Code

U01 Research Project – Cooperative Agreements

Announcement Type

Reissue of

RFA-NS-25-024

Related Notices

Check for any recent Notices of NIH Policy Changes that may impact application requirements.

Funding Opportunity Number (FON)

RFA-NS-26-001

Companion Funding Opportunity

None

Number of Applications

See Section III. Eligibility Information.

Assistance Listing Number(s)

93.853

Funding Opportunity Purpose

The purpose of this Notice of Funding Opportunity (NOFO) is to solicit applications for the conduct of scientific research utilizing data from expanded access (EA) for investigational drugs or biological products as described in section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb). These applications will target intermediate size populations of patients living with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of ALS.

Funding Opportunity Goal(s)

To support extramural research funded by the National Institute of Neurological Disorders and Stroke (NINDS) including: basic research that explores the fundamental structure and function of the brain and the nervous system; research to understand the causes and origins of pathological conditions of the nervous system with the goal of preventing these disorders; research on the natural course of neurological disorders; improved methods of disease prevention; new methods of diagnosis and treatment; drug development; development of neural devices; clinical trials; and research training in basic, translational, and clinical neuroscience.

Key Dates

Posted Date

May 04, 2026

Open Date (Earliest Submission Date)

May 04, 2026

June 08, 2026

June 08, 2026

Not Applicable

September 2026

November 10, 2026

November 10, 2026

Not Applicable

July 2027

All applications are due by 5:00 PM local time of applicant organization.

Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date.

No late applications will be accepted for this Notice of Funding Opportunity (NOFO).

Due Dates for E.O. 12372

Not Applicable

Expiration Date

November 11, 2026

Required Application Instructions

It is critical that applicants follow the instructions in the Research (R) Instructions in the How to Apply - Application Guide, except where instructed to do otherwise (in this NOFO or in a Notice from NIH Guide for Grants and Contracts).

Conformance to all requirements (both in the Application Guide and the NOFO) is required and strictly enforced. Applicants must read and follow all application instructions in the Application Guide as well as any program-specific instructions noted in Section IV. When the program-specific instructions deviate from those in the Application Guide, follow the program-specific instructions.

Applications that do not comply with these instructions may be delayed or not accepted for review.

Table of Contents

Part 1. Overview Information Key Dates Part 2. Full Text of Announcement Section I. Notice of Funding Opportunity Description Section II. Award Information Section III. Eligibility Information Section IV. Application and Submission Information Section V. Application Review Information Section VI. Award Administration Information Section VII. Agency Contacts Section VIII. Other Information

Part 2. Full Text of Announcement

Section I. Notice of Funding Opportunity Description

The purpose of this Notice of Funding Opportunity (NOFO) is to solicit applications for the conduct of scientific research utilizing data from expanded access (EA) for investigational drugs or biological products as described in section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb). These applications will target intermediate size populations of patients living with amyotrophic lateral sclerosis (ALS) who are not eligible for ongoing clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of ALS ("intermediate EA protocol for ALS").

Provision of the investigational drug or biological product under an intermediate EA protocol for ALS must not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval or otherwise compromise the potential development of medical products for the prevention, diagnosis, mitigation, treatment, or cure of ALS.

Background ALS is a rapidly progressive, ultimately fatal, neurodegenerative disease causing weakness and wasting of skeletal muscles including the diaphragm. There are approximately 34,000 cases in the United States (U.S.) equivalent to a prevalence of 10.1 per 100,000 U.S. population. While considerable variability in presentation and progression exists, mean survival from symptom onset is typically only 3–5 years, and treatment options for ALS remain severely limited. Existing disease-modifying drugs for ALS have only modest effects on slowing disease progression in most ALS cases, and no known treatment prevents, halts or reverses ALS progression. Thus, development of new, effective treatments to prevent disease onset, make ALS a livable disease, or cure ALS is a pressing need.

To address this challenge, individuals with ALS may enroll in ongoing phase 3/efficacy clinical trials of investigational drugs and biological products for ALS. However, trial sponsors typically set inclusion criteria that restrict participation to only a subset of people with ALS to increase the likelihood of detecting efficacy with feasible sample size and trial duration. Thus, a potentially large segment of people with ALS may be ineligible to participate in clinical trials because they do not meet these inclusion criteria.

EA, sometimes referred to as “compassionate use”, was established to provide treatment access to investigational medical products (i.e., drugs, biological products, medical devices) for people with serious, life-threatening diseases like ALS when no comparable or satisfactory therapy is available to diagnose, monitor, or treat the disease or condition. This pathway is defined and regulated by the FDA (see https://www.fda.gov/news-events/public-health-focus/expanded-access). Further, the FDA has not yet determined if these products are safe and effective for their intended use. One requirement of the EA pathway is that providing the investigational medical product under EA must not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of a medical product for the EA use, or otherwise compromise its development. An intermediate-size patient population EA protocol is generally used when more than one patient will be treated with an investigational drug/biological product under the EA (21 CFR 312.315), and can involve hundreds or even thousands of patients if appropriate.

Section 2 of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS; P.L. 117-79) established a grant program for scientific research utilizing data from EA to investigational drugs for ALS. Since fiscal year 2022, NIH has been implementing this grant program through issuing NOFOs, including this one, and funding new EA research grants.

Objective The objective of this NOFO is to support the conduct of scientific research utilizing data from intermediate-size patient population EA protocols for ALS ("intermediate EA protocol for ALS", see https://www.fda.gov/news-events/expanded-access/expanded-access-categories-drugs-including-biologics). To be eligible, the proposed intermediate EA protocol for ALS will provide access to an investigational drug or biological product for people with ALS and also generate data to support research on the prevention, diagnosis, mitigation, treatment, or cure of ALS. The scientific research goal(s) informed by these data could include the investigational drug’s or biological product’s effect on biomarkers related to the pathophysiology of ALS or target engagement, the collection of safety or outcome information, such as survival or other significant medical events (e.g., hospitalization or need for ventilatory support), or patient experience data that are not specifically tied to assessing efficacy.

Additionally, this NOFO allows for competitive renewal of currently funded EA protocols through the NINDS. (See instructions under Section IV under Research Strategy.) Applicants are encouraged to discuss the submission of a renewal application with NINDS and seek approval to submit as necessary prior to submitting the application.

Applicants should take note of the following special requirements and considerations:

Investigators proposing NIH-defined clinical trials may refer to the Research Methods Resources website for information about developing statistical methods and study designs.

See Section VIII. Other Information for award authorities and regulations.

Section II. Award Information

Funding Instrument

Cooperative Agreement: A financial assistance mechanism used when there will be substantial Federal scientific or programmatic involvement. Substantial involvement means that, after award, NIH scientific or program staff will assist, guide, coordinate, or participate in project activities. See Section VI.2 for additional information about the substantial involvement for this NOFO.

Application Types Allowed

New

Renewal

Resubmission

The OER Glossary and the How to Apply Application Guide provide details on these application types. Only those application types listed here are allowed for this NOFO.

Clinical Trial?

Required: Only accepting applications that propose clinical trial(s).

Need help determining whether you are doing a clinical trial?

Funds Available and Anticipated Number of Awards

NINDS intends to commit up to $40,000,000 per fiscal year to fund 4 new awards per year for the duration of this RFA. This estimate is contingent upon NIH appropriations and a sufficient number of meritorious applications.

Award Budget

Application budgets need to reflect the actual needs of the proposed project.

Award Project Period

The scope of the proposed project should determine the project period. The maximum project period is 4 years.

NIH grants policies as described in the NIH Grants Policy Statement will apply to the applications submitted and awards made from this NOFO.

Section III. Eligibility Information

1. Eligible Applicants

Eligible Organizations

Nonprofits Other Than Institutions of Higher Education

For-Profit Organizations

Local Governments

Federal Governments

Eligible Agencies of the Federal Government

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

Eligible applicants must be clinical trial sites that participate in a phase 2/3 or phase 3 efficacy clinical trial supported by a U.S.-based small business concern that is the FDA-designated sponsor of a drug or biological product which is the subject of an IND under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) to prevent, diagnose, mitigate, treat, or cure ALS. The definition of a small business concern can be found in section 3(a) of the Small Business Act (15 U.S.C. 632(a)).

All activities proposed in your application and budget narrative must align with applicable law, including but not limited to statutes, executive orders, federal regulations and applicable judicial holdings. Accordingly, discretionary awards shall not be used to fund, promote, encourage, subsidize, or facilitate; racial preferences or other forms of racial discrimination by the recipient, including activities where race or intentional proxies for race will be used as a selection criterion for employment or program participation; denial by the recipient of the sex binary in humans, or the belief that sex is a chosen or mutable characteristic; illegal immigration; or any other initiatives that compromise public safety. If an application does not align, the application will not receive funding to the extent permitted by law and applicable court orders.

Foreign Organizations/International Collaborations

Non-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply.

Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply.

Foreign components, as defined in the NIH Grants Policy Statement, are not allowed.

Required Registrations

Applicant Organizations Applicant organizations must complete and maintain the following registrations as described in the How to Apply- Application Guide to be eligible to apply for or receive an award. All registrations must be completed prior to the application being submitted. Registration can take 6 weeks or more, so applicants should begin the registration process as soon as possible. Failure to complete registrations in advance of a due date is not a valid reason for a late submission, please reference the NIH Grants Policy Statement Section 2.3.9.2 Electronically Submitted Applications for additional information.

System for Award Management (SAM) – Applicants must complete and maintain an active registration, which requires renewal at least annually. The renewal process may require as much time as the initial registration. SAM registration includes the assignment of a Commercial and Government Entity (CAGE) Code for domestic organizations which have not already been assigned a CAGE Code. Foreign organizations must obtain a NATO Commercial and Government Entity (NCAGE) Code (in lieu of a CAGE code) in order to register in SAM. Unique Entity Identifier (UEI)- A UEI is issued as part of the SAM.gov registration process. The same UEI must be used for all registrations, as well as on the grant application.

Program Directors/Principal Investigators (PD(s)/PI(s)) All PD(s)/PI(s) must have an eRA Commons account. PD(s)/PI(s) should work with their organizational officials to either create a new account or to affiliate their existing account with the applicant organization in eRA Commons. If the PD/PI is also the organizational Signing Official, they must have two distinct eRA Commons accounts, one for each role. Obtaining an eRA Commons account can take up to 2 weeks.

All PD(s)/PI(s) must be registered with ORCID. The personal profile associated with the PD(s)/PI(s) eRA Commons account must be linked to a valid ORCID ID. For more information on linking an ORCID ID to an eRA Commons personal profile see the ORCID topic in our eRA Commons online help.

Eligible Individuals (Program Director/Principal Investigator)

Any individual(s) with the skills, knowledge, and resources necessary to carry out the proposed research as the Program Director(s)/Principal Investigator(s) (PD(s)/PI(s)) is invited to work with their organization to develop an application for support.

For institutions/organizations proposing multiple PDs/PIs, visit the Multiple Program Director/Principal Investigator Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the How to Apply-Application Guide.

2. Cost Sharing This NOFO does not require cost sharing as defined in the NIH Grants Policy Statement Section 1.2 Definition of Terms.

3. Additional Information on Eligibility Number of Applications

Applicant organizations may submit more than one application, provided that each application is scientifically distinct.

The NIH will not accept duplicate or highly overlapping applications under review at the same time, per NIH Grants Policy Statement Section 2.3.7.4 Submission of Resubmission Application. This means that the NIH will not accept:

A new (A0) application that is submitted before issuance of the summary statement from the review of an overlapping new (A0) or resubmission (A1) application.

A resubmission (A1) application that is submitted before issuance of the summary statement from the review of the previous new (A0) application.

An application that has substantial overlap with another application pending appeal of initial peer review (see NIH Grants Policy Statement 2.3.9.4 Similar, Essentially Identical, or Identical Applications).

Section IV. Application and Submission Information

1. Requesting an Application Package The application forms package specific to this opportunity must be accessed through ASSIST, Grants.gov Workspace or an institutional system-to-system solution. Links to apply using ASSIST or Grants.gov Workspace are available in Part 1 of this NOFO. See your administrative office for instructions if you plan to use an institutional system-to-system solution.

2. Content and Form of Application Submission It is critical that applicants follow the instructions in the Research (R) Instructions in the How to Apply - Application Guide except where instructed in this notice of funding opportunity to do otherwise (in this NOFO, in a policy notice, or other notice from NIH Guide for Grants and Contracts). Conformance to the requirements in the Application Guide is required and strictly enforced. Applications that are out of compliance with these instructions may be delayed or not accepted for review.

Page Limitations All page limitations described in the How to Apply- Application Guide and the Table of Page Limits must be followed.

Instructions for Application Submission

The following section supplements the instructions found in the How to Apply- Application Guide and should be used for preparing an application to this NOFO.

SF424(R&R) Cover All instructions in the How to Apply - Application Guide must be followed.

SF424(R&R) Project/Performance Site Locations All instructions in the How to Apply- Application Guide must be followed.

SF424(R&R) Other Project Information

SF424(R&R) Senior/Key Person Profile

R&R or Modular Budget

R&R Subaward Budget

PHS 398 Cover Page Supplement

PHS 398 Research Plan All instructions in the How to Apply - Application Guide must be followed, with the following additional instructions:

Research Strategy:

Significance: Describe how data generated through the proposed intermediate EA protocol for the investigational drug/biological product will advance research or development to support prevention, diagnosis, mitigation, treatment, or cure of ALS.

Approach:

Non-interference with Clinical Trials: Describe how the proposed intermediate EA protocol for ALS is designed to avoid 1) interfering with the initiation, conduct, or completion of clinical investigations that could support regulatory approval or 2) otherwise compromise the potential development of medical products for the prevention, diagnosis, mitigation, treatment, or cure of ALS.

Outline how data will be obtained, analyzed, and interpreted with sufficient rigor to quantitatively assess project outcomes. Describe the use of common data elements (CDEs), including those available at Amyotrophic Lateral Sclerosis | NINDS CDE, and, as applicable, other NIH or external resources to standardize the collection of clinical data such as:

As applicable, describe what non-traditional approaches to engage potential study participants for intervention delivery and data collection will be used to minimize participant burden. In addition, describe the plans, if any, to use non-traditional data collection approaches (e.g., digital/mobile/sensor technologies and web-based systems) and why these are appropriate.

As appropriate, describe the plan for stakeholder engagement, such as establishing relationships with PWLE and/or advocacy groups to solicit their input (including during study design) on recruitment, the clinical meaningfulness of the question under study, the relevance of the proposed clinical outcomes, and approaches to minimizing the burden on study participants.

The summary of the proposed research should include a discussion of potential limitations and/or challenges in the protocol and strategies to address these concerns.

Letters of Support: If there will be subcontracts or service agreements for personnel or facilities, include documentation of such commitments, co-signed by a business official and the investigator at the participating center. All activities proposed in subcontracts must align with the current Executive Orders (EOs).

If there are agreements with collaborating industry partners, include documentation of the agreements, co-signed by a business official and an appropriate official at the company.

If there are manufacturing agreements, include a letter of intent or letter of authorization from the manufacturer, co-signed by a business official and an appropriate official at the company.

Should CTSA resources be used, include letter(s) of support from each site’s CTSA program officer(s) concurring with the specific plan for usage of these resources.

If some costs for the intermediate EA protocol for ALS are to be borne by sources other than NIH, include documentation of this support, signed by individuals who have the authority to make a commitment on behalf of the organization they represent.

Applicants are encouraged to include letters or other supporting documentation from relevant stakeholders (e.g., potential study participants, referring and treating physicians, patient advocacy groups) to show that they believe the study question to be important, consider the study design to be acceptable, and that patients were included as partners in the concept development and design of the EA program.

Resource Sharing Plan: Individuals are required to comply with the instructions for the Resource Sharing Plans as provided in the How to Apply - Application Guide.

Applications proposing to collect biospecimens must use the BioSEND protocols and procedures, and all specimens collected and banked with BioSEND must come from individuals who have consented to banking and sharing broadly with academia and industry. Note that costs for collection are NOT included as a component of the NINDS Biomarkers Repository award to offset some of the cost of biospecimen banking. Therefore, costs for the biospecimen collection (collection kits, shipping kits to sites, shipping samples to BioSEND) are borne by the recipients utilizing this resource (see NOT-NS-15-046). Applicants planning projects in which biospecimens will be collected must consult the BioSEND website for more information about samples banked at the repository. In addition, applicants are advised to consult with BioSEND staff to obtain a quote for biospecimen banking costs. Please request a quote from BioSEND by filling out a quote form. BioSEND staff will contact the PI(s). Applicants should include this information in the application.

Whenever applicable, the NINDS Human Cell and Data Repository (NHCDR) must be used to bank and distribute cells collected from participants in these studies. Note that costs for collection are NOT included as a component of the NHCDR award to offset some of the cost of biospecimen banking. Therefore, costs for the biospecimen collection (collection kits, shipping kits to sites, shipping samples to NHCDR) are borne by the recipients utilizing this resource. Applicants planning projects in which biospecimens will be collected are strongly advised to consult the NHCDR website for more information about samples banked at the repository. In addition, applicants are advised to consult with NHCDR staff to obtain a quote for biospecimen banking costs.

Other Plan(s):

All instructions in the How to Apply - Application Guide must be followed, with the following additional instructions:

PHS Human Subjects and Clinical Trials Information When involving human subjects research, clinical research, and/or NIH-defined clinical trials (and when applicable, clinical trials research experience) follow all instructions for the PHS Human Subjects and Clinical Trials Information form in the How to Apply- Application Guide, with the following additional instructions:

If you answered “Yes” to the question “Are Human Subjects Involved?” on the R&R Other Project Information form, you must include at least one human subjects study record using the Study Record: PHS Human Subjects and Clinical Trials Information form or Delayed Onset Study record.

Study Record: PHS Human Subjects and Clinical Trials Information

All applications must follow the instructions G.500 – PHS Human Subjects and Clinical Trials Information, in addition to instructions specific to this NOFO.

Section 2 - Study Population Characteristics A goal of this initiative is to support studies that lead to findings applicable to all people affected by ALS. Therefore, it is important that participants appropriately represent the ALS patient population in the U.S.

Inclusion of Human Subjects Policies When the proposed project involves human subjects and/or NIH-defined clinical research, the committee will evaluate the proposed plans for inclusion. For additional information on review of the Inclusion section, please refer to the Guidelines for the Review of Inclusion in Clinical Research.

Section 3 - Protection and Monitoring Plans

3.1 Protection of Human Subjects

Discuss how the consent process will clearly inform study participants that the grant support for EA for the investigational drug/biological product is limited to the project period.

3.1.2 Adequacy of Protection Against Risks

3.1.2b Protections Against Risks A plan for interim analysis and stopping the study for evidence of harm, i.e., greater than expected mortality as well as serious adverse effects.

3.3 Data and Safety Monitoring Plan

Applicants should follow the NINDS Guidelines for Data and Safety Monitoring in Clinical Trials when developing their Data and Safety Monitoring Plan (DSMP).

3.5 Overall Structure of the Study Team

Delayed Onset Study Note: Delayed onset does NOT apply to a study that can be described but will not start immediately (i.e., delayed start). All instructions in the How to Apply- Application Guide must be followed.

PHS Assignment Request Form

3. Unique Entity Identifier and System for Award Management (SAM) See Part 2. Section III.1 for information regarding the requirement for obtaining a unique entity identifier and for completing and maintaining active registrations in System for Award Management (SAM), NATO Commercial and Government Entity (NCAGE) Code (if applicable), eRA Commons, and Grants.gov

4. Submission Dates and Times Part I. contains information about Key Dates and times. Applicants are encouraged to submit applications before the due date to ensure they have time to make any application corrections that might be necessary for successful submission. When a submission date falls on a weekend or Federal holiday, the application deadline is automatically extended to the next business day.

Organizations must submit applications to Grants.gov (the online portal to find and apply for grants across all Federal agencies). Applicants must then complete the submission process by tracking the status of the application in the eRA Commons, NIH’s electronic system for grants administration. NIH and Grants.gov systems check the application against many of the application instructions upon submission. Errors must be corrected and a changed/corrected application must be submitted to Grants.gov on or before the application due date and time. If a Changed/Corrected application is submitted after the deadline, the application will be considered late. Applications that miss the due date and time are subjected to the NIH Grants Policy Statement Section 2.3.9.2 Electronically Submitted Applications.

Applicants are responsible for viewing their application before the due date in the eRA Commons to ensure accurate and successful submission.

Information on the submission process and a definition of on-time submission are provided in the How to Apply-Application Guide.

5. Intergovernmental Review (E.O. 12372) This initiative is not subject to intergovernmental review.

6. Funding Restrictions All NIH awards are subject to the terms and conditions, cost principles, and other considerations described in the NIH Grants Policy Statement.

Pre-award costs are allowable only as described in the NIH Grants Policy Statement Section 7.9.1 Selected Items of Cost.

All activities proposed in your application and budget narrative must align with applicable law, including but not limited to statutes, executive orders, federal regulations and applicable judicial holdings. Accordingly, discretionary awards shall not be used to fund, promote, encourage, subsidize, or facilitate; racial preferences or other forms of racial discrimination by the recipient, including activities where race or intentional proxies for race will be used as a selection criterion for employment or program participation; denial by the recipient of the sex binary in humans, or the belief that sex is a chosen or mutable characteristic; illegal immigration; or any other initiatives that compromise public safety. If an application does not align, the application will not receive funding to the extent permitted by law and applicable court orders.

7. Other Submission Requirements and Information

Applications must be submitted electronically following the instructions described in the How to Apply – Application Guide. Paper applications will not be accepted.

Applicants must complete all required registrations before the application due date. Section III. Eligibility Information contains information about registration.

For assistance with your electronic application or for more information on the electronic submission process, visit How to Apply – Application Guide. If you encounter a system issue beyond your control that threatens your ability to complete the submission process on-time, you must follow the Dealing with System Issues guidance. For assistance with application submission, contact the Application Submission Contacts in Section VII.

Important reminders:

All PD(s)/PI(s) must include their eRA Commons ID in the Credential field of the Senior/Key Person Profile form. Failure to register in the Commons and to include a valid PD/PI Commons ID in the credential field will prevent the successful submission of an electronic application to NIH. See Section III of this NOFO for information on registration requirements.

The applicant organization must ensure that the unique entity identifier provided on the application is the same identifier used in the organization’s profile in the eRA Commons and for the System for Award Management. Additional information may be found in the How to Apply – Application Guide.

See more tips for avoiding common errors.

Upon receipt, applications will be evaluated for completeness and compliance with application instructions by the Center for Scientific Review and responsiveness by components of participating organizations, NIH. Applications that are incomplete, non-compliant and/or nonresponsive will not be reviewed.

Mandatory Disclosure

Recipients or subrecipients must submit any information related to violations of federal criminal law involving fraud, bribery, or gratuity violations potentially affecting the federal award. See Mandatory Disclosures, 2 CFR 200.113 and NIH Grants Policy Statement Section 4.1.35.

For financial conflicts of interest in drug development see 42 eCFR Part 50.

Send written disclosures to the NIH Chief Grants Management Officer listed on the Notice of Award for the IC that funded the award and to the HHS Office of Inspector Grant Self Disclosure Program at grantdisclosures@oig.hhs.gov.

Post Submission Materials Applicants are required to follow the instructions for post-submission materials, as described in the policy

FDA Documentation : FDA documentation is optional and should be provided as post-submission materials only if available and not already provided in the application at the time of submission. If still unavailable at the deadline for post-submission materials, they must be provided as part of just in time materials.

Section V. Application Review Information

1. Criteria Only the review criteria described below will be considered in the review process. Applications submitted to the NIH in support of the NIH mission are evaluated for scientific and technical merit through the NIH peer review system.

Overall Impact

Reviewers will provide an overall impact score to reflect their assessment of the likelihood for the project to exert a sustained, powerful influence on the research field(s) involved, in consideration of the following scored review criteria and additional review criteria (as applicable for the project proposed). An application does not need to be strong in all categories to be judged likely to have a major scientific impact.

Scored Review Criteria

Reviewers will consider Factors 1, 2 and 3 in the determination of scientific merit, and in providing an overall impact score. In addition, Factors 1 and 2 will each receive a separate factor score.

Factor 1. Importance of the Research (Significance and Innovation)

Innovation

Specific to this NOFO:

Significance

Innovation Evaluate how the data can de-risk future rare disease drug development.

Factor 2. Rigor and Feasibility (Approach)

Rigor:

For applications involving human subjects, including clinical trials, assess the adequacy of inclusion plans as appropriate for the scientific goals of the research. Considerations of appropriateness may include disease/condition/behavior incidence, prevalence, or population burden, population representation, and/or current state of the science.

Feasibility:

Specific to this NOFO:

Approach