CARE program: NCI and FDA interagency collaborations to support oncology small business entrepreneurs

The Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs, with a $1.2 billion annual budget, are major sources of seed funding for early-stage technology development in the US. The National Cancer Institute (NCI) SBIR Development Center, the largest NIH Institute, provided approximately $180 million in non-dilutive funding in 2021 to small businesses developing cancer-related technologies. While this funding is crucial, navigating FDA regulations is a significant challenge for these small businesses, often lacking regulatory expertise. This lack of expertise leads to delays and inefficient use of resources. To address this, the NCI SBIR, in collaboration with the US Food and Drug Administration (FDA), developed the Connecting Awardees with Regulatory Experts (CARE) program, focusing on providing early-stage regulatory assistance and education to bridge this gap.

The impact of NIH SBIR/STTR programs on FDA-approved drugs is significant, with reports indicating over 12% of novel drugs approved between 1996-2020 received SBIR/STTR funding. NCI SBIR portfolio companies demonstrate a similar trend, with a substantial number of FDA approvals and IND/IDE applications in 2020-2021. This highlights the importance of collaboration between the NIH and FDA to streamline the process for small businesses.

In 2018, an NCI SBIR advisory board recommended enhancements, including regulatory assistance. A survey of NCI SBIR portfolio companies revealed significant needs for regulatory guidance, particularly early in development. The CARE program was piloted in 2019 and expanded in 2020 to include all FDA centers relevant to oncology. The program involves a multi-pronged approach: 1. **Demystifying the FDA:** NCI SBIR portfolio companies apply for CARE, providing details on their technology and regulatory questions. The application is routed to the appropriate FDA center, and questions are answered by experts. 2. **Communicating FDA Resources:** The NCI identified that many companies were unaware of available FDA resources. A public website with curated FDA links and resources was created. NCI SBIR also incorporated information on these resources into their company onboarding webinars. 3. **Joint Funding Opportunities:** A collaboration with the FDA's Center for Devices and Radiological Health (CDRH) created targeted NCI funding opportunities focusing on the development of Medical Device Development Tools (MDDTs). This incentivizes the development of tools to streamline the regulatory process. From 2019-2022, 141 companies participated in CARE. Following participation, companies completed an online survey to assess the program's impact.

From 2020-2022, 81% (88/109) of companies responded to the post-CARE survey. Key findings include: * The program helped companies identify the relevant FDA center (97%), develop regulatory strategies (85%), and plan next steps (89%). * Companies planned to utilize FDA industry education offices (81%), access online resources (93%), and submit meeting requests (91%). * The program informed future SBIR/STTR project aims (81%). * 90% of respondents would recommend the program. * The regulatory resource webpage launched in 2020 has over 2000 visitors, with 93% of CARE participants finding it useful. * Awareness of FDA resources among CARE participants increased from 16% in 2019 to 70% in 2022. * Three NCI contract funding opportunities were published in collaboration with CDRH to support MDDT development, focusing on biomarker tools, evaluation datasets, and AI/ML software for oncology devices.

The CARE program successfully connects early-stage NCI-funded companies with the FDA, providing essential regulatory guidance and education. Early regulatory input mitigates risks, reduces time to market, and improves the efficient use of federal funding. The program addresses a critical gap for small businesses lacking internal regulatory expertise. Other collaborations, such as workshops on oncology drug development and various funding programs (Technical and Business Assistance, Commercialization Readiness Pilot), further support small businesses.

The NCI and FDA's CARE program offers valuable support to small businesses navigating the regulatory landscape. By connecting companies with FDA experts and providing access to resources, it accelerates technology development and efficient use of federal funding. The program's success demonstrates the benefits of interagency collaboration and serves as a model for future initiatives.

The study's limitations include the response rate of 81% for the post-CARE survey and potential selection bias among participating companies. Long-term impact data beyond the immediate post-program survey would enhance the evaluation of the program's sustained effectiveness.