PCORI Funding

Addressing Rare Diseases

A PCORI Funding Announcement (PFA) will open on May 7, 2024 to fund studies addressing patient-centered comparative effectiveness research questions that will fill important rare disease research gaps. Applications addressing critical decisional dilemmas that span multiple rare diseases are encouraged, but studies focusing on one rare disease are also acceptable. For this funding announcement, applicants may request coverage of patient care costs (including medical products, procedures, and care services) for potential funding by PCORI.

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

Studies should address patient-centered comparative effectiveness research questions that will fill important rare disease research gaps. Applications addressing critical decisional dilemmas that span multiple rare diseases are encouraged, but studies focusing on one rare disease are also acceptable. For this funding announcement, applicants may request coverage of patient care costs (including medical products, procedures, and care services) for potential funding by PCORI.

Interventions/Comparators

Comparators can be systems and/or clinical approaches, which may be pharmacological or nonpharmacological interventions. Interventions with established efficacy in the general population or interventions developed or adapted for those with rare diseases may be acceptable.

Outcomes

Well-justified outcomes that are clinically meaningful and considered important by patients and that can be impacted by the proposed intervention within the study duration.

Funding

• Total allocated for this PFA: Up to $100 million
• Total direct funding: Up to $12 million

Maximum project duration

Five years

Important dates

May 7 – Applications open
May 14 – Town Hall
June 4 – Letters of Intent due (please use template on application page)
September 4 – Applications due
November 2024 – Merit review
March 2025 – Awards announced
July 2025 – Earliest start date

Special Office Hours

Potential applicants can also request an opportunity to speak with PCORI staff for more complex questions during virtual “office hours.” Submit your project information and questions on the website.

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