This funding supports efficient and innovative natural history studies aimed at advancing medical product development for rare diseases with unmet needs. The studies should generate high-quality, interpretable data to address critical knowledge gaps, remove barriers, and have a significant impact on specific or multiple rare diseases sharing similar pathophysiology, facilitating product development.
Eligible applicants include a wide range of organizations such as higher education institutions, nonprofits, for-profit organizations, government entities, tribal governments, and foreign organizations. Both domestic and non-domestic entities may apply.
