The National Institutes of Health (NIH) provides funding to conduct efficacy studies in an established rare disease preclinical model to demonstrate that a proposed therapeutic agent warrants further development.
Donor Name: National Institutes of Health (NIH)
State: All States
County: All Counties
U.S. Territory: American Samoa, Guam, Commonwealth of Puerto Rico, Commonwealth of Northern Mariana Islands, U.S. Virgin Islands
Type of Grant: Grant
Deadline: 09/01/2023
Size of the Grant: $275,000
Grant Duration: 2 years
Details:
The goal of this NOFO is to spur therapeutic development for rare diseases by advancing projects to the point where they would attract subsequent investment supporting full Investigational New Drug (IND) application development or progression to clinical trials in the case of repurposing or repositioning.
Research Objectives and Scope
- This notice of funding opportunity (NOFO) solicits applications that propose to conduct efficacy studies in an established rare disease preclinical model to demonstrate proof of concept that a proposed therapeutic agent warrants further development. In addition to preclinical efficacy, accompanying pharmacodynamic (PD) and pharmacokinetic (PK) studies would be supported. In preclinical efficacy studies, a combination of efficacy, (PD) and (PK) measures are warranted to determine the feasibility of a candidate therapeutic agent to serve as a starting point for further therapy development. Combined measures of PK and PD greatly increase the understanding of the in vivo efficacy of the therapeutic agent by exploring the relationship between the concentration of the agent at the site of action and the resulting efficacy measures.
- For the purposes of this NOFO, efficacy measures reflect the effects of the therapeutic agent on endpoints that are closely tied to the desired clinical endpoints, but do not necessarily reflect target engagement. While PD measures may also reflect the effects of the therapeutic agent on endpoints closely tied to the desired clinical endpoint, they must also reflect target engagement. PK measurements reflect the body’s effect on the absorption, metabolism, distribution and excretion of the therapeutic agent. While not required, PK to provide evidence that the therapeutic agent has the potential to be therapeutically viable and support the robustness of the efficacy data, is encouraged, if the limited R21 budget can accommodate such studies.
- Efficacy studies should be conducted in an established and validated rare disease model. These models may include both in vivo models and/or in advanced in vitro models (e.g. tissue chip/microphysiologic systems). Data supporting relevance and validation of the model to the indicated rare disease should be included in the application. Funding will not be provided for model development; evidence supporting access to the model, in suitable numbers to support rigorous efficacy studies, must be provided. A letter of support if the animals will be provided by a collaborator, or a quote for purchase must be included in the Letters of Support attachment.
- To ensure both the relevance of the proposed development of the therapeutic agent, as well as the disease model, a plan for a partnership with a rare disease steering/oversight committee is required to submit to this NOFO. However, neither the members of the committee nor the organizations that might partner should be named in the application This committee may be formed de novo with members having appropriate disease relevant expertise and/or experience and could include a potential financial backer. The plan should include only a description of the type of individuals (expertise, patients and/or advocates, foundation representatives – do NOT name the foundation, etc.) and the planned interactions with the scientific team.
- Further, developing therapeutics requires a multidisciplinary approach. It is encouraged that academic investigators should form collaborations with those knowledgeable in the therapeutic development process, as well as those familiar with the desired properties of the end therapeutic product (such as biostatisticians, technical experts and clinicians). Applicants should consider how they will identify and foster relationships with potential licensing and commercialization partners early in the therapy development process once an award is made. Investigators are expected to work closely with their institutional technology transfer officials, if applicable, to ensure that royalty agreements, patent filings, and all other necessary intellectual property (IP) arrangements are completed in a timely manner and that commercialization plans are developed and updated over the course of the project.
Funds Available and Anticipated Number of Awards
- The NIH intends to commit up to $1,200,000 in FY24, contingent upon receiving scientifically meritorious applications.
- The combined budget for direct costs for the two-year project period may not exceed $275,000. No more than $200,000 may be requested in any single year.
- The total project period may not exceed 2 years.
Eligible Organizations
Higher Education Institutions
- Public/State Controlled Institutions of Higher Education
- Private Institutions of Higher Education
The following types of Higher Education Institutions are always encouraged to apply for NIH support as Public or Private Institutions of Higher Education:
- 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 Other Than Institutions of Higher Education
- Nonprofits with 501(c)(3) IRS Status (Other than Institutions of Higher Education)
- Nonprofits without 501(c)(3) IRS Status (Other than Institutions of Higher Education)
For-Profit Organizations
- Small Businesses
- For-Profit Organizations (Other than Small Businesses)
Local Governments
- State Governments
- County Governments
- City or Township Governments
- Special District Governments
- Indian/Native American Tribal Governments (Federally Recognized)
- Indian/Native American Tribal Governments (Other than Federally Recognized)
Federal Government
- 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
Foreign Institutions
- Non-domestic (non-U.S.) Entities (Foreign Institutions) 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 allowed.
For more information, visit Grants.gov.
