Online System Opens
Aug. 12, 2025, 9 am (ET)
Applicant Town Hall
Aug. 19, 2025; 12 - 1 pm (ET)
View Event
Letter of Intent Deadline
Sept. 23, 2025, 5 pm (ET)
Letter of Intent Status Notification
Oct. 21, 2025, 5 pm (ET)
Application Deadline
Jan. 13, 2026, 5 pm (ET)
Merit Review
March 2026
Awards Announced
July 2026
Earliest Start Date
November 2026
Funds Available Up To
Up to $60 million
Total Direct Costs
Up to $12 million
Maximum Project Period
Up to five years
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 below SAEs should identify the area that is best associated with their research approach:
Applications addressing cross-cutting issues that include more than one rare disease diagnosis are encouraged, but studies focusing on a single rare disease are also acceptable. Individual- or cluster-randomized controlled trials are preferred; however, well-specified natural experiments, well-designed prospective or retrospective observational studies, and other innovative CER designs suited to rare disease research will also be considered if rigorous methods are proposed. Applicants are encouraged to pay special attention to issues of intervention implementation with an aim of facilitating widespread uptake of findings after completion of the study by utilizing hybrid effectiveness-implementation approaches. Studies focused on the development of research methods will not be considered responsive. Applicants proposing retrospective observational studies must use and demonstrate access to existing data sources such as registries, patient-powered networks or data networks (e.g., PCORnet®).
Comparators can be systems and/or clinical approaches, which may be pharmacological or nonpharmacological interventions. Comparators targeted to rare disease patients and/or their caregivers are acceptable. Interventions with established efficacy in the general population or interventions developed or adapted for those with rare diseases may be acceptable. Applications proposing novel interventions and/or aiming to develop new technologies (such as mobile apps) or decision support tools/aids will be considered nonresponsive. Adaptations of efficacious interventions used in the general population or similar situation may be responsive, but the level of adaptation must be minimal, clearly described and justified a priori.
Applicants should consider, as appropriate, the full range of clinical and patient-centered outcomes data relevant to patients and other stakeholders. PCORI’s Principles for the Consideration of the Full Range of Outcomes Data in PCORI-Funded Research inform the expectations for applicants and the corresponding evaluation of applications submitted in response to this PFA.
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.
PCORI encourages all investigators interested in applying to use this inquiry form to submit questions and request to speak with a PCORI Program Officer.
This opportunity seeks to fund high-quality, patient-centered comparative clinical effectiveness research projects focused on rare diseases like ALS. PCORI is particularly interested in submissions that address approaches to symptom management, timely diagnosis, and improving care delivery for individuals with rare diseases.
Sponsor Institute/Organizations: Patient-Centered Outcomes Research Institute
Sponsor Type: Corporate/Non-Profit
Address: 1333 New Hampshire Avenue NW, Suite 1200 Washington, DC 20036 Phone: (202) 827-7700 | Fax: (202) 355-9558 info@pcori.org
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Sep 23, 2025
Jan 13, 2026
$12,000,000
Affiliation: Patient-Centered Outcomes Research Institute
Address: 1333 New Hampshire Avenue NW, Suite 1200 Washington, DC 20036 Phone: (202) 827-7700 | Fax: (202) 355-9558 info@pcori.org
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