Funder: Patient-Centered Outcomes Research Institute (PCORI)
Due Dates: August 12, 2025 (Online Opens) | September 23, 2025 (LOI Deadline) | January 13, 2026 (Application Deadline)
Funding Amounts: Up to $12 million direct costs per project; maximum project period of 5 years.
Summary: Supports comparative clinical effectiveness research (CER) studies to address symptom management, diagnosis, and care delivery for individuals with rare diseases.
Key Information: Patient and stakeholder engagement is required; both single and cross-disease studies are eligible; randomized trials preferred but other rigorous CER designs considered.
Description
This funding opportunity from the Patient-Centered Outcomes Research Institute (PCORI) seeks to support high-quality, patient-centered comparative clinical effectiveness research (CER) focused on rare diseases. The initiative aims to address critical gaps in evidence related to symptom management, timely diagnosis, and care delivery for individuals with rare diseases. PCORI encourages studies that address cross-cutting issues spanning multiple rare diseases, but studies focused on a single rare disease are also eligible.
PCORI is particularly interested in research that:
- Compares approaches to symptom management for rare disease patients, especially for symptoms common across multiple conditions (e.g., pain, sleep disturbance, mental health).
- Evaluates strategies to reduce the time to accurate diagnosis (the “diagnostic odyssey”).
- Assesses models and strategies to improve care delivery, including care transitions, case management, and access to primary or specialty care.
Individual- or cluster-randomized controlled trials are preferred, but well-designed natural experiments, prospective or retrospective observational studies, and other innovative CER designs will be considered if rigorous methods are proposed. Retrospective studies must use and demonstrate access to existing data sources (e.g., registries, patient-powered networks).
Comparators may include pharmacological or nonpharmacological interventions, systems, or clinical approaches. Adaptations of interventions with established efficacy in the general population are allowed if the adaptation is minimal and well-justified. Projects focused on developing new interventions, technologies, or decision aids are not responsive.
All projects must include meaningful engagement of patients and stakeholders, consistent with PCORI’s Foundational Expectations for Partnerships in Research.
Due Dates
- PCORI Online System Opens: August 12, 2025
- Applicant Town Hall: August 19, 2025 (noon–1 pm ET) (Register here)
- Letter of Intent (LOI) Deadline: September 23, 2025 (5 pm ET)
- LOI Status Notification: October 21, 2025
- Application Deadline: January 13, 2026 (5 pm ET)
- Merit Review: March 2026
- Awards Announced: July 2026
- Earliest Start Date: November 2026
Funding Amount
- Total Direct Costs: Up to $12 million per project
- Maximum Project Period: Up to 5 years
- Patient Care Costs: May be included within the direct cost cap (e.g., medical products, procedures, care services)
Eligibility
- Open to U.S.-based research institutions, academic centers, healthcare organizations, and other eligible entities.
- Studies must address rare diseases as defined by the NIH/FDA (affecting fewer than 200,000 individuals in the U.S.).
- Both single- and multi-disease studies are eligible.
- Applicants must demonstrate access to necessary data sources for retrospective studies.
- Engagement of patients, caregivers, and other stakeholders is required throughout the research process.
Application Process
- Review the full funding announcement: PCORI Rare Diseases Funding Opportunity
- Attend the Applicant Town Hall (optional but recommended): August 19, 2025 (Register here)
- Submit a Letter of Intent (LOI): Due September 23, 2025
- If invited, submit a full application: Due January 13, 2026
- Engage with PCORI staff: Applicants may request 1:1 office hours to discuss project ideas or application questions (Request office hours)
- Ensure compliance with PCORI’s engagement and data sharing policies.
Additional Information
- Special Areas of Emphasis: Symptom management, timely diagnosis, and care delivery are highlighted, but other topics relevant to rare diseases are welcome.
- Study Design: Randomized trials are preferred; other rigorous CER designs are eligible.
- Nonresponsive Projects: Development of new interventions, technologies, or decision aids; studies focused solely on research methods.
- Patient Care Costs: May be included but must be justified and within the $12M direct cost cap.
- Engagement: Applicants must address PCORI’s Foundational Expectations for Partnerships in Research.
- FAQs: PCORI Rare Diseases PFA FAQs
External Links
Contact Information