NINDS Exploratory Clinical Trials for Small Business (R43/R44 Clinical Trial Required)

Active
Yes
Status
Open
Release Date
November 27th, 2023
Open Date
December 5th, 2023
Due Date(s)
January 5th, 2024April 5th, 2024September 5th, 2024January 5th, 2025April 5th, 2025September 5th, 2025January 5th, 2026April 5th, 2026September 5th, 2026
Close Date
September 6th, 2026
Topic No.
PAR-23-311

Topic

NINDS Exploratory Clinical Trials for Small Business (R43/R44 Clinical Trial Required)

Agency

Department of Health and Human ServicesNational Institutes of Health

Program

Type: SBIRPhase: BOTHYear: 2024

Summary

The Department of Health and Human Services, specifically the National Institutes of Health, is seeking proposals for the NINDS Exploratory Clinical Trials for Small Business (R43/R44 Clinical Trial Required) topic. This solicitation aims to support small business concerns (SBCs) in conducting exploratory clinical trials that contribute to the justification for future trials to establish definitive efficacy. The trials can include Phase 1 and 2 studies of drugs and biologics, feasibility and preliminary efficacy studies of devices, as well as early studies of surgical, behavioral, or rehabilitation therapies. The trials can be single-site or multi-site with up to three clinical sites. The applications must generate data that inform further clinical development of the proposed intervention or diagnostic, and FDA approval is required for FDA-regulated clinical trials. The goal is to advance products/technologies that require federal regulatory approval or bring complex research tools to market. The solicitation is open until September 6, 2026, and more information can be found at the provided links.

Description

NINDS is committed to advancing diagnostics and treatments for people burdened by neurological diseases, and the NINDS Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs have provided the small business community with critical seed funding to support the development of a wide variety of technologies and therapeutics for the diagnosis and treatment of neurological diseases. The SBIR/STTR Programs are structured in three phases. The main objective in SBIR/STTR Phase I is to establish the technical merit and feasibility of the proposed research and development (R&D) efforts, whereas in SBIR/STTR Phase II it is to continue the R&D efforts to advance the technology toward ultimate commercialization. At the conclusion of an SBIR/STTR Phase II, it is expected that the small business concern (SBC) will fully commercialize their product or technology using non-SBIR/STTR funds in Phase III.

The development of medical biotechnology products is often impeded by a significant funding gap (known as the “Valley of Death”). In particular, the development of regulated products such as therapeutics and medical devices often requires several years and substantial capital investments, due in part to the high costs of clinical trials.

This NOFO supports applications from Small Business Concerns (SBCs) for exploratory clinical trials that contribute to the justification for a future trial to establish definitive efficacy (such as a Phase 3 clinical trial or a Pivotal device trial). This includes Phase 1 and 2 studies of drugs and biologics, feasibility and preliminary efficacy studies of devices, as well as early studies of surgical, behavioral or rehabilitation therapies. This NOFO supports single-site and multi-site trials with up to three clinical sites. Applications must aim to generate data that inform further clinical development of the proposed intervention or diagnostic, and in the case of FDA-regulated clinical trials applications must demonstrate readiness by including FDA approval of the proposed study. The earliest clinical studies should be designed to provide important initial information regarding the intervention (e.g., safety, tolerability, dosing) or diagnostic. Midstage clinical studies will generally include randomization and blinding and should yield data that allow a clear go/no-go decision regarding whether the intervention should proceed to an efficacy trial. All applications must outline specific plans for future development in the event of promising results.

This NOFO is not intended to support the conduct of a clinical trial where the primary aim is to establish or confirm definitive efficacy. Applications to implement definitive efficacy trials (e.g., Phase 3 clinical trials of drugs or Pivotal device trials) should be submitted to NINDS Efficacy Clinical Trials (UG3/UH3). See https://grants.nih.gov/grants/guide/pa-files/PAR-21-237.html for more information.

This NOFO is not intended to support the conduct of clinical trials with more than three clinical sites. NINDS supports several clinical trial networks specifically designed to implement larger multi-site clinical trials. See https://www.ninds.nih.gov/Current-Research/Research-Funded-NINDS/Clinical-Research for more information.

Renewal applications of SBIR Phase II awards (i.e., SBIR Phase IIB) to conduct Phase I and II clinical studies should be submitted to NINDS Renewal Awards of SBIR Phase II Grants (Phase IIB) for Clinical Trials and Clinical Research (R44).

The goal of this NOFO is to assist applicants in pursuing the next appropriate milestone(s) necessary to advance a product/technology that requires Federal regulatory approval or to bring a complex research tool to market. To achieve this goal, the NOFO aims to facilitate the transition of clinical-stage projects to the commercialization stage by encouraging business relationships between NIH’s SBIR/STTR recipients and third-party investors and/or strategic partners.

Definitions

For this funding opportunity announcement Phase 1 and 2 clinical studies or trials refer to the common phases of a clinical trial. SBIR Phase I and II refer to the project phases of the SBIR program.

NIH defines a clinical trial as "a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes."

Specific Objectives

Scientific/Technical Scope

Examples of appropriate studies under this NOFO include, but are not limited to, those designed to:

Evaluate and optimize the dose, formulation, safety, tolerability or pharmacokinetics of an intervention in healthy volunteers or the target population. Evaluate whether an intervention produces sufficient evidence of short-term activity (e.g., biomarker activity, target engagement, dose-response trends, pharmacodynamic response) in a human "proof of concept" trial. Select or rank the best of two or more potential interventions or dosing regimens to be evaluated in a subsequent trial, based on tolerability, biological activity, or preliminary clinical efficacy (e.g., futility trials). For devices: Establish proof-of-principle and optimize techniques, operation, and usability of a device; inform the final device design decisions; and estimate the magnitude of treatment effect. NINDS recognizes that devices can differ greatly in terms of basic form and function, physiological bases for therapy, degree of invasiveness, etc. A Pivotal device study, for example, could potentially be used in support of an off-label indication of an existing market approved device, or to provide evidence for a novel device design in support of a Pre-Market Approval (PMA), Humanitarian Device Exemption (HDE), 510(k) or 510(k) De Novo submission. Due to the broad scope of possible medical devices and the varied nature of the regulatory path, investigators considering applications to evaluate devices are strongly encouraged to contact Scientific/Research Staff as early as possible to discuss these issues and determine the suitability of their project for this funding mechanism.

Every facet of the United States scientific research enterprise—from basic laboratory research to clinical and translational research to policy formation–requires superior intellect, creativity and a wide range of skill sets and viewpoints. NIH’s ability to help ensure that the nation remains a global leader in scientific discovery and innovation is dependent upon a pool of highly talented scientists from diverse backgrounds who will help to further NIH's mission. Research shows that diverse teams working together and capitalizing on innovative ideas and distinct perspectives outperform homogenous teams. Scientists and trainees from diverse backgrounds and life experiences bring different perspectives, creativity, and individual enterprise to address complex scientific problems. There are many benefits that flow from a diverse NIH-supported scientific workforce, including: fostering scientific innovation, enhancing global competitiveness, contributing to robust learning environments, improving the quality of the research, advancing the likelihood that underserved or health disparity populations participate in, and benefit from health research, and enhancing public trust. See the Notice of NIH's Interest in Diversity, NOT-OD-20-031. Moreover, fostering diversity by encouraging the participation of individuals from nationally underrepresented groups in the scientific research workforce is a longstanding interest of Congress, (e.g., Public Law 117–167, CHIPS and Science Act of 2022, P.L. 114-329, American Innovation and Competitiveness Act of 2017, P.L. 114-255, 21st Century Cures Act of 2016). Also, fostering and encouraging participation by socially and economically disadvantaged and women-owned small businesses in technological innovation is one of the goals of the SBIR and STTR programs (https://www.sbir.gov/sites/default/files/SBA%20SBIR_STTR_POLICY_DIRECTIVE_May2023.pdf).

Plan for Enhancing Diverse Perspectives

The NIH recognizes that diverse teams working together and capitalizing on innovative ideas and distinct perspectives outperform homogeneous teams. There are many benefits that flow from a diverse scientific workforce, including: fostering scientific innovation, enhancing global competitiveness, contributing to robust learning environments, improving the quality of the research, advancing the likelihood that underserved populations participate in, and benefit from research, and enhancing public trust.

To support the best science, the NIH encourages inclusivity in research. Examples of structures that promote diverse perspectives include but are not limited to:

Transdisciplinary research projects and collaborations among neuroscientists and researchers from fields such as computational biology, physics, engineering, mathematics, computer and data sciences, as well as bioethics. Engagement from different types of institutions and organizations (e.g., research-intensive, undergraduate-focused, minority-serving, community-based). Individual applications and partnerships that enhance geographic and regional heterogeneity. Investigators and teams composed of researchers at different career stages. Participation of individuals from diverse backgrounds, including groups traditionally underrepresented in the biomedical, behavioral, and clinical research workforce (see NOT-OD-20-031), such as underrepresented racial and ethnic groups, those with disabilities, those from disadvantaged backgrounds, and women. Opportunities to enhance the research environment to benefit early- and mid-career investigators. This NOFO requires a Plan for Enhancing Diverse Perspectives (PEDP), as described in NOT-MH-21-310, submitted as Other Project Information as an attachment (see Section IV). Applications that fail to include a PEDP will be considered incomplete and will be withdrawn.

Applicants are strongly encouraged to read the NOFO instructions carefully and view the available PEDP guidance materia. The PEDP lwill be assessed as part of the scientific and technical peer review evaluation.

Independent Third-Party Partners

As significant funding is often needed to take regulated clinical projects to market beyond initial clinical studies, we encourage applicants to develop business relationships between applicant SBCs and third-party investors/strategic partners who can provide substantial financing to help accelerate the commercialization of promising new products and technologies initiated with NIH SBIR or STTR funding. Third-party partners include, but are not limited to, another company, a venture capital firm, an “angel” investor, a foundation, a university, a research institution, a state or local government, or any combination of the above. In light of these goals, the NINDS strongly encourages applicants to establish business relationships with investors and/or strategic partners that have appropriate prior experience in the commercialization of emerging biomedical technologies.

That said, NINDS recognizes that companies developing products that have small potential revenue streams or that target small patient populations face additional barriers to market entry that make them less attractive to investors and strategic partners at preclinical or early clinical stages of development. Many of these technologies require complex clinical trial designs because of small and geographically dispersed patient populations.

For the purposes of this NOFO, NINDS has defined small markets as development of novel products that:

Address a rare disease, defined in the Rare Diseases Act of 2002 (Public Law 107-280) as a condition affecting fewer than 200,000 individuals in the United States; or Qualify as a Humanitarian Use Device, defined as a medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in fewer than 4,000 persons in the United States per year; or Target a young pediatric population defined as including neonates (0-28 days), infants (

Similar Opportunities

NINDS Exploratory Clinical Trials for Small Business (R43/R44 Clinical Trial Required)
Department of Health and Human Services
The Department of Health and Human Services, specifically the National Institutes of Health, is seeking proposals for the NINDS Exploratory Clinical Trials for Small Business (R43/R44 Clinical Trial Required). This solicitation aims to support Small Business Concerns (SBCs) in conducting exploratory clinical trials that contribute to the justification for future trials to establish definitive efficacy. The trials can include Phase 1 and 2 studies of drugs and biologics, feasibility and preliminary efficacy studies of devices, as well as early studies of surgical, behavioral, or rehabilitation therapies. The trials can be single-site or multi-site with up to three clinical sites. The applications must generate data that inform further clinical development of the proposed intervention or diagnostic, and FDA approval is required for FDA-regulated clinical trials. The goal is to advance products/technologies that require Federal regulatory approval or bring complex research tools to market. The solicitation is open until September 6, 2026, and more information can be found at the provided links.
NINDS Exploratory Clinical Trials for Small Business (R41/R42 Clinical Trial Required)
Department of Health and Human Services
The Department of Health and Human Services, specifically the National Institutes of Health, is seeking proposals for the NINDS Exploratory Clinical Trials for Small Business (R41/R42 Clinical Trial Required). This solicitation aims to support small business concerns (SBCs) in conducting exploratory clinical trials that contribute to the justification for future trials to establish definitive efficacy. The trials can include Phase 1 and 2 studies of drugs and biologics, feasibility and preliminary efficacy studies of devices, as well as early studies of surgical, behavioral, or rehabilitation therapies. The trials can be single-site or multi-site with up to three clinical sites. The applications must generate data that inform further clinical development of the proposed intervention or diagnostic, and FDA approval is required for FDA-regulated clinical trials. The goal is to advance products/technologies that require federal regulatory approval or bring complex research tools to market. The funding opportunity is open until September 6, 2026. For more information and to apply, visit the solicitation agency URL: [link](https://grants.nih.gov/grants/guide/pa-files/PAR-24-044.html).
NINDS Exploratory Clinical Trials for Small Business (R41/R42 Clinical Trial Required)
Department of Health and Human Services
The Department of Health and Human Services, specifically the National Institutes of Health, is seeking proposals for the NINDS Exploratory Clinical Trials for Small Business (R41/R42 Clinical Trial Required). This solicitation aims to support small business concerns (SBCs) in conducting exploratory clinical trials that contribute to the justification for future trials to establish definitive efficacy. The trials can include Phase 1 and 2 studies of drugs and biologics, feasibility and preliminary efficacy studies of devices, as well as early studies of surgical, behavioral, or rehabilitation therapies. The trials can be single-site or multi-site with up to three clinical sites. The applications must generate data that inform further clinical development of the proposed intervention or diagnostic, and FDA approval is required for FDA-regulated clinical trials. The goal is to advance products/technologies that require federal regulatory approval or bring complex research tools to market. The funding opportunity is open until September 6, 2026. For more information and to apply, visit the solicitation agency URL: [link](https://grants.nih.gov/grants/guide/pa-files/PAR-24-044.html).
NIAID SBIR Phase II Clinical Trial Implementation Cooperative Agreement (U44 Clinical Trial Required)
Department of Health and Human Services
The Department of Health and Human Services, specifically the National Institutes of Health, is seeking proposals for the NIAID SBIR Phase II Clinical Trial Implementation Cooperative Agreement (U44 Clinical Trial Required). This solicitation encourages Small Business Innovation Research (SBIR) grant applications from small business concerns (SBCs) that propose to implement investigator-initiated clinical trials, including mechanistic studies associated with clinical trials. The program supports milestone-driven, hypothesis-driven clinical trials related to the research mission of the NIAID that address high-priority research areas. The solicitation encourages high-risk clinical studies, which involve non-routine interventions, administration of unlicensed products, or administration of licensed products for unapproved indications. Mechanistic studies are also encouraged. The funding supports the conduct, completion, and analysis of a single clinical trial, including activities such as training of study personnel, enrollment and recruitment of study subjects, data collection and management, laboratory work, study management and oversight, safety monitoring, and regulatory activities. The project duration is not specified, but the solicitation is open until January 14, 2027. The funding specifics can be found on the grants.nih.gov website.