NINDS Renewal Awards of SBIR Phase II Grants (Phase IIB) for Clinical Trials and Clinical Research (R44 Clinical Trial Optional)

Purpose NINDS is committed to advancing treatments, tools, and diagnostics that benefit people burdened by neurological diseases through the NINDS Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs. 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). To achieve commercialization, some projects initiated with SBIR or STTR funding require considerable grant-in-aid financing beyond the SBIR/STTR Phase II award to achieve follow-on investment or commercialization. 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 or studies 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, first-in-human studies (as defined by the Food and Drug Administration), 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. Mid-stage 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. In addition, 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 disparate patient populations. For the purposes of this NOFO, NINDS has defined small markets as development of novel products that: Address a rare disease as defined in the Orphan Drug Act Amendment of 1984 as any disease or condition affecting fewer than 200,000 persons in the United States 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 Target a young pediatric population defined as including neonates (0-28 days), infants (