PCORI Funding

Addressing Rare Diseases

New Patient-Centered Outcomes Research Institute (PCORI) funding opportunity: Up to $12 million in direct costs for studies up to five years duration, researching rare diseases.

Studies should:

• Compare two or more approaches for rare disease prevention, diagnosis, treatment, or care delivery
• Address a critical clinical or care delivery problem
• Have well-documented evidence of efficacy and/or widespread use, as demonstrated by prior research, systematic reviews, and/or clinical guidelines
• Include evidence-based, impactful, patient-centered outcomes as indicated by patients, families, and caregivers (particularly those with lived experience) from the population of interest, as well as relevant members of the broader health and healthcare community
• Examine diverse populations with an overall sample size that will allow precision in the estimation of hypothesized effect sizes and, as appropriate, analysis of heterogeneity of treatment effect

PCORI is particularly interested in submissions that address the following Special Areas of Emphasis (SAEs), although applicants are not limited to these areas:

• Approaches to symptom management for individuals with rare disease
• Approaches for timely diagnosis of rare diseases
• Approaches to improving care delivery for individuals with rare diseases

For this funding announcement, applicants may request coverage of patient care costs (including medical products, procedures, and care services) for potential funding.

Highlights

• Up to $60 million in total available funding
• Up to $12 million direct costs available per application
• Maximum project period: 5 years
• August 12 – Application opens
• August 19 – Town Hall
• September 23 – Letters of Intent due

How to Apply

To apply for this funding opportunity, there are four steps you must follow.

• Read the full funding announcement, available online.
• Follow the process outlined in the submission instructions.
• Submit a Letter of Intent using the template on the application page.
• Submit an application (if invited).

Important Dates

August 12 – Applications open
August 19 – Town Hall
September 23 – Letters of Intent due
October 21 – LOI status notification
January 13, 2026 – Applications due
March 2026 – Merit review
July 2026 – Awards announced
November 2026 – Earliest start date

Special Office Hours

All investigators interested in applying are encouraged to request an appointment with PCORI staff to discuss their proposals.

Harvard Catalyst PCORI Consultation

For more information or to request a brief consultation, please contact Leslie MacGregor, PCORI Funding Specialist: Leslie_MacGregor@hms.harvard.edu