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NEI Small Business Innovation Research (SBIR) Cooperative Agreement for Early-Stage Clinical Trials with Greater than Minimal Risk (U44-Clinical Trial Required)


This Notice of Funding Opportunity (NOFO) encourages Small Business Innovation Research (SBIR) grant applications from small business concerns (SBCs) that propose to implement investigator-initiated clinical trials related to the research mission of the NEI. This program provides support for milestone-driven, commercialization-oriented clinical trials and will involve participation of NEI program staff in negotiating the final project plan before award and monitoring of research progress. Applications supported by this NOFO must meet the NIH definition of a clinical trial (see and must be greater than minimal risk trials. As defined in 45 CFR § 46.102, a minimal risk trial is one in which the probability and magnitude of harm or discomfort anticipated in the research are not greater in and of themselves than those ordinarily encountered in daily life or during the performance of routine physical or psychological examinations or tests. The mission of the NEI is to eliminate vision loss and improve quality of life through vision research. Applications considered for funding by the NEI must align with the NEI Strategic Plan. Background This NEI SBIR Cooperative Agreement (U44) for Clinical Trials is designed to support SBIR grant applications specifically from small business concerns (SBCs). Currently, the NEI uses U01 cooperative agreement mechanism to support early-stage, small-scale clinical trials that are greater than minimal risk; and UG1 to support large-scale, multi-center clinical trials, human gene-transfer and stem cell therapy trials, and other complex or high resource- or safety-risk clinical trials. Scope The scope of this NOFO is to support U44 applications for clinical trials to investigate the safety and/or efficacy of screening, diagnostic, preventative, or therapeutic interventions for eye diseases or disorders. Applicants seeking a Phase II or Phase IIB award must have received an eligible predicate award that is appropriate and relevant (e.g., pre-clinical studies, planning activities, etc.) to this NOFO. An application without an eligible predicate award must be submitted as a Direct to Phase II application. This NOFO is specifically intended for early-stage clinical trials with greater than minimal risk that meet all of the following criteria: Include strong preliminary and/or pre-clinical data Are early-stage clinical trials Have a well described Commercialization Plan (see Section IV for details) Examples of appropriate studies 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. Determining the optimal outcome measure (endpoint), its variability, and/or the optimal timing of outcome evaluations in the context of the intervention. 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 effects. Collecting information on the utility of questionnaires, rating scales, or biomarkers. This NOFO will support the conduct, completion, and analysis of a clinical trial, including activities related to the conduct of the clinical trial, which include but are not limited to the following: training of study personnel enrollment and recruitment of study subjects investigational product costs data collection, management and quality control laboratory work and data analyses study management and oversight establishment of committees to manage the complexity of the trial preparation of the final study report and other related post-trial activities regulatory activities and site monitoring can be covered if required Clinical trial planning activities should be completed prior to the time of application submission and investigators must be ready to implement the proposed trial at the time of award. If the proposed clinical trial involves the use of a drug or a device that has not been approved by the FDA for the proposed investigational use, then evidence of an Investigational New Drug (IND) application or an Investigational Device Exemption (IDE) application submission to the FDA that meets all requirements under 21 CFR 312 is required at the time of the U44 grant application submission. It is the responsibility of the applicant to provide evidence from the FDA if an IND or an IDE is not required. NEI reserves the right to deny or postpone the award until evidence of IND/IDE authorization from FDA is provided. Milestones Delineation of milestones is a key characteristic of the NEI Cooperative Agreement (U44) for Early-Stage Clinical Trials with greater than Minimal Risk. A milestone is defined as a scheduled event in the project timeline, signifying the completion of a major project stage or activity. See Section IV. Application and Submission Information for more specific instructions. Applications Not Responsive to this NOFO: The following types of activities remain outside of the scope of this NOFO, and applications proposing such activities will be considered non-responsive to this NOFO and will be withdrawn without review: Applications that do not meet NEI Mission: Applications involving a clinical experiment that is not directly intended to evaluate a screening, diagnostic, preventative, or therapeutic intervention Applications that aim to test a drug, a device, or service with limited or no commercialization potential NIH-defined clinical trial applications proposing only mechanistic and/or minimal risk studies Applications that are large-scale, multi-center clinical trials, human gene-transfer and stem cell therapy trials, and other complex or high resource clinical trials Applications that propose a project that extends beyond a three-year project period Applications that propose animal studies Applications that lack appropriate attachments (See Other Attachments in Section IV. Application and Submission Information) Data Monitoring and Safety Committee (DSMC) A DSMC is required for this mechanism. The DSMC is an independent group composed of individuals not directly involved in patient care or data collection for the study who are responsible for safeguarding the interests of all trial participants, assessing the safety and efficacy of the treatment during the trial, and for monitoring the overall conduct of the trial. The DSMC operates under the guidance of an approved Charter. Do not name individuals for the DSMC but include areas of expertise that will be pertinent in forming this committee. NEI reserves the right to specify the requirements for the establishment of a DSMC (Data and Safety Monitoring Committee). Applicants are encouraged to discuss those decisions and requirements with NEI prior to submission of the application. Programmatic Oversight NEI program staff will closely monitor progress in meeting the milestones of all projects funded by cooperative agreements under this NOFO and will also oversee the management and reporting of adverse events and have regular communications with the PD(s)/PI(s). The NEI Program Official(s) will participate in the DSMC meetings and will serve as a technical representative to the DSMC. Applicants are strongly encouraged to contact Scientific/Research staff as plans for an application are being developed (see Section VII, Agency Contacts), and no later than 12 weeks prior to the anticipated application submission date. All research and development activities associated with awards made under this NEI SBIR Cooperative Agreement for Early-Stage Clinical Trials with Greater than Minimal Risk (U44) NOFO must be performed within the United States (the United States is defined as the 50 States, the territories and possessions of the United States, the Commonwealth of Puerto Rico, the Federated States of Micronesia, the Republic of Palau, the Republic of the Marshall Islands, and the District of Columbia).
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