Addressing Rare Diseases PCORI Funding Announcement -- Cycle 3 2025
PCORI funds rigorous, patient-centered research comparing care, symptom management, and diagnosis strategies to improve outcomes for people with rare diseases.
Funder: Patient-Centered Outcomes Research Institute
Due Dates (Anticipated): September 2026 (Letter of Intent) | January 2027 (Full application)
Funding Amounts: Up to $12 million in direct costs per project (max 5 years); total program budget up to $60 million.
Summary: Supports high-impact, patient-centered comparative effectiveness research addressing care, symptom management, and diagnosis challenges in rare diseases.
Key Information: All dates are projected; verify on the program page for updates.
Description
This funding opportunity from the Patient-Centered Outcomes Research Institute (PCORI) supports high-quality, patient-centered comparative clinical effectiveness research (CER) focused on rare diseases, as defined by the Orphan Drug Act (affecting fewer than 200,000 individuals in the US). The initiative aims to address research gaps and critical decisional dilemmas experienced by individuals with rare diseases and their caregivers, such as difficulties in accessing information, obtaining an accurate diagnosis, and securing appropriate care. Proposals may focus on cross-cutting issues or a single rare disease and are encouraged to address care delivery, symptom management, or timely diagnosis. Studies should use rigorous methods—including randomized controlled trials, well-specified observational studies, or innovative CER designs—and must include meaningful patient and stakeholder engagement throughout the research process.
