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Addressing Rare Diseases Topical PCORI Funding Announcement

Patient-Centered Outcomes Research Institute

Key Dates

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 and Project Period

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:

Approaches to symptom management for individuals with rare disease: As few rare diseases have curative treatments and many patients with a rare disease focus their efforts on managing their disease symptoms, PCORI is interested in funding CER comparing symptomatic care, particularly for symptoms that occur in multiple rare diseases. Some examples include behavioral and symptom relief treatments/techniques for sleep disturbance, pain, pruritus and comorbid mental health diagnoses (e.g., anxiety and depression).
Approaches for timely diagnosis of rare diseases: Between three and 15 years is a common timeline for receiving a correct rare disease diagnosis, with an average of approximately five years. During this period, frequently referred to as the “diagnostic odyssey,” individuals may experience numerous care visits, a multitude of tests and procedures and inaccurate or missed diagnoses. PCORI is interested in CER studies comparing approaches to decrease the length of the diagnostic odyssey — for example, by comparing genetic testing approaches for newborns with serious illnesses of unknown cause.
Approaches to improving care delivery for individuals with rare diseases: Care delivery for those with rare diseases is influenced by many system-level factors that impact patients, families and caregivers. Challenges are not necessarily condition-specific, which allows for a focus on cross-cutting issues and outcomes to increase the potential for research impact. PCORI is interested in funding CER comparing strategies to address these challenges and improve care delivery for individuals with rare diseases. Some examples include models of care for children with rare disease transitioning to adult care; case management models, including family-based care management models designed to reduce caregiver burden or stress; and approaches to improving primary care for individuals with rare disease unable to access care in specialty centers, including care coordination models or telemonitoring/digital health tools.

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.

Specialties

Clinical Trials

Rare Diseases

Eligibility Requirements

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 Details

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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