This opportunity invites studies that compare different approaches to prevention, diagnosis, treatment, or care delivery in ways that meaningfully improve outcomes for individuals and families facing these challenging conditions.

Rare diseases affect fewer than 200,000 people in the United States individually, yet collectively they touch nearly 30 million individuals. Many of those affected and their caregivers grapple with similar hurdles—ranging from delayed or inaccurate diagnoses to difficulty accessing reliable information or appropriate care. This funding announcement calls for proposals that directly address these shared challenges and fill critical research gaps.

PCORI is especially encouraging applications that explore three key areas: improving care delivery models such as case or care management, enhancing symptom management for conditions without curative treatments, and shortening the painfully long timeline to accurate diagnosis—the so-called “diagnostic odyssey.” Approaches that cut across multiple rare diseases are welcome and often carry greater potential for broader impact.

The program provides significant financial support, with applicants able to request direct costs of up to $12 million. At the time of contract execution, PCORI sets aside the full project budget to be available for the entire performance period. The maximum project period extends up to five years, ensuring that research teams have the time and resources needed to generate meaningful results. The budget covers all research- and peer-review-related costs associated with the project.

Eligibility for this program is broad, allowing a wide range of research organizations to apply. Applications may come from academic research institutions, private sector research entities, nonprofit or for-profit organizations, colleges and universities, and agencies or instrumentalities of the federal government. All applicants must be equipped to conduct rigorous, patient-centered comparative effectiveness research and manage funding responsibly.

This funding cycle represents a rare opportunity for researchers and healthcare institutions to advance treatments and delivery systems that can transform the lives of individuals affected by rare diseases. By supporting studies that span conditions and emphasize patient-centered outcomes, PCORI is aiming to build evidence that can be rapidly translated into real-world impact.

For more information, visit PCORI.