Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers
This grant funds innovative research and infrastructure to accelerate the development, evaluation, and access to therapies for ultra-rare pediatric and adult cancers, including those defined by unique molecular features.
Funder: Food and Drug Administration
Due Dates: May 18, 2026 (Letter of Intent) | June 15, 2026 (Full application, all applicants)
Funding Amounts: Up to $500,000 per year for up to 3 years; total program funding up to $1,500,000; 1–3 awards expected.
Summary: Supports innovative approaches to advance therapeutic development for ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers.
Description
This opportunity from the FDA Oncology Center of Excellence (OCE) seeks to fund new methods, tools, and strategies to facilitate the development of therapies for ultra-rare cancers. Ultra-rare cancers are defined for this program as those with an annual U.S. incidence of 300–400 people or less, a more stringent criterion than the Orphan Drug Act. The program encourages projects that address barriers in drug development for these cancers, such as difficulties in patient recruitment, limited financial incentives, and gaps in disease understanding. Areas of interest include infrastructure for data sharing, novel endpoints for clinical studies, leveraging real-world data, drug repurposing, pragmatic trial design, and innovative delivery technologies. Both pediatric and adult ultra-rare cancers are within scope, including molecularly-defined subsets of more common cancers.
