Addressing Rare Diseases PCORI Funding Grant

The Patient-Centered Outcomes Research Institute (PCORI) will announce funding for patient-centered comparative clinical effectiveness research (CER) studies focusing on rare diseases. This initiative encourages collaboration and aims to fill critical research gaps affecting approximately 30 million Americans with rare diseases. Applications can request up to $12 million over five years, including coverage for patient care costs.

Notice of Upcoming PCORI Funding Announcement
The Patient-Centered Outcomes Research Institute (PCORI) intends to issue a PCORI Funding Announcement (PFA) on August 12, 2025, seeking to fund high-quality, patient-centered comparative clinical effectiveness research (CER) studies that focus on rare diseases. This preannouncement provides potential applicants additional time to identify collaborators; obtain patient and stakeholder input on potential studies; and develop responsive, high-quality proposals.
Research Initiative Highlights
PCORI uses the National Institutes of Health's definition of a rare disease, defined as a condition that affects fewer than 200,000 individuals in the United States. Though each disease is rare individually, it is estimated that there are more than 10,000 rare diseases affecting about 30 million Americans. People with many different types of rare diseases and their caregivers often experience similar challenges such as finding reliable information and relevant resources, receiving an accurate diagnosis and gaining access to adequate and appropriate care. This Topical PFA will solicit applications proposed to address patient-centered CER questions that will fill important rare disease research gaps. Applications addressing critical decisional dilemmas that span multiple rare diseases are encouraged.PCORI is particularly interested in submissions that address the following Special Areas of Emphasis (SAEs). The purpose of identifying these SAEs is to encourage submissions to these areas, not to limit submissions to these topics. Applicants addressing one of the  SAEs found here should identify the area that is best associated with their research approach.
Award
This funding announcement will accept applications with direct costs up to $12 million dollars and up to five years in duration. For this funding announcement, applicants may request coverage of patient care costs (including medical products, procedures and care services) for potential funding by PCORI. These costs are included as part of the overall direct cost maximum. For additional information, please see the full announcement upon posting.