Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers

The funding opportunity titled Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers is offered by the U.S. Food and Drug Administration (FDA), specifically through its Oncology Center of Excellence (OCE). The OCE was established to accelerate oncology product development and regulatory science by integrating scientific expertise across multiple FDA centers. This initiative reflects the agency’s broader commitment to addressing unmet medical needs in oncology, particularly in ultra-rare cancers, which are defined here as cancers with an annual U.S. incidence of approximately 300 to 400 individuals or fewer. These conditions present unique challenges including limited patient populations for trials, insufficient biological understanding, and reduced commercial incentives for drug development. The primary purpose of this funding opportunity is to support innovative approaches that facilitate therapeutic development for ultra-rare pediatric and adult cancers, including molecularly defined subsets of more common cancers. The program emphasizes the development of broadly applicable tools, methods, and frameworks that can improve drug development pipelines and regulatory decision-making. Rather than focusing on creating a single therapeutic product, the funding is intended to advance enabling technologies, infrastructure, and scientific methodologies that can be leveraged across multiple ultra-rare cancer contexts. Funding will support projects that align with specific research priorities such as building data-sharing infrastructures, validating novel clinical endpoints, leveraging real-world data through registries, and repurposing previously approved or discontinued drugs. Additional areas include improving trial accessibility through telemedicine, developing advanced delivery systems for targeted therapies, and conducting molecular-level analyses to identify novel therapeutic targets. Importantly, clinical trials are not allowed under this opportunity; however, observational studies and methodological innovations related to clinical trials are permitted. Awardees must also comply with federal funding restrictions and align all proposed activities with applicable laws and policies. The funding mechanism for this opportunity is a cooperative agreement (U01), indicating substantial involvement from FDA staff throughout the project lifecycle. Awards will provide one year of initial funding with the possibility of up to two additional years contingent on performance and available appropriations, for a maximum project period of three years. The FDA anticipates committing up to $1,500,000 in fiscal year 2026 to fund approximately one to three awards. Individual project budgets must not exceed $500,000 per year in total costs, inclusive of both direct and indirect expenses. Eligibility for this funding opportunity is broad and includes a wide range of U.S.-based entities such as higher education institutions, nonprofit organizations, for-profit companies, small businesses, and various levels of government including tribal organizations. Foreign entities and foreign components of U.S. organizations are not eligible. Applicants must complete several mandatory registrations including SAM, Grants.gov, and eRA Commons prior to submission. Individuals serving as Principal Investigators must demonstrate sufficient expertise and leadership capacity to manage the proposed research program. The application process includes an optional but strongly encouraged letter of intent due on May 18, 2026, followed by a full application submission deadline of June 15, 2026. Applications must be submitted electronically through Grants.gov and validated through eRA Commons. Required components include a detailed research strategy, data management and sharing plan, and compliance with all human subjects or animal research requirements if applicable. Applications will be evaluated based on criteria such as significance, investigator qualifications, innovation and approach, alignment with regulatory science priorities, and research environment. Successful applicants will be notified following review and selection processes, and will be required to submit regular progress reports and comply with FDA oversight and collaboration requirements throughout the project period.

Focus on regulatory science relevance and demonstrate broad applicability beyond a single therapy Ensure strong methodological rigor and feasibility Avoid proposing clinical trials as they are not allowed