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Notice of Upcoming PCORI Funding Announcement

Key Dates

  • PFA Opens
    May 7, 2024
  • Applicant Town Hall
    May 14, 2024; 12 pm (ET)
  • Letter of Intent (LOI) Deadline
    June 4, 2024; 5 pm (ET)
  • LOI Status Notification
    July 2, 2024
  • Application Deadline
    Sept. 4, 2024; 5 pm (ET)

The Patient-Centered Outcomes Research Institute (PCORI) intends to issue a Topical PCORI Funding Announcement (PFA) on May 7, 2024, seeking to fund high-quality, patient-centered comparative clinical effectiveness research (CER) projects 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 less 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 an SAE should identify the area that is best associated with their research approach.

  1. 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, pruritis and comorbid mental health diagnoses (e.g., anxiety and depression).
  2. Approaches for timely diagnosis of rare diseases: Between 3 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.
  3. 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 strongly encouraged to assess issues of intervention implementation to facilitate widespread uptake of findings after completion of the study. Toward that end, hybrid type I and hybrid type II designs may be appropriate. Applicants proposing 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. 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 propose 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. 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. For more information please visit the Patient-Centered Economic Outcomes page on our website.

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.

Key Dates

Online System Opens
May 7, 2024
Applicant Town Hall
-
View Event
Letter of Intent Deadline
June 4, 2024, 5 pm (ET)
Letter of Intent Status Notification
July 2, 2024
Application Deadline
Sept. 4, 2024, 5 pm (ET)
Merit Review
November 2024
Awards Announced
April 2025
Earliest Start Date
August 2025

Funds and Project Period

Funds Available Up To
Approximately $100 million
Total Direct Costs
Up to $12 million
Maximum Project Period
Up to five years

Tags

Year
Health Conditions Health Conditions These are the broad terms we use to categorize our funded research studies; specific diseases or conditions are included within the appropriate larger category. Note: not all of our funded projects focus on a single disease or condition; some touch on multiple diseases or conditions, research methods, or broader health system interventions. Such projects won’t be listed by a primary disease/condition and so won’t appear if you use this filter tool to find them. View Glossary
Populations Populations PCORI is interested in research that seeks to better understand how different clinical and health system options work for different people. These populations are frequently studied in our portfolio or identified as being of interest by our stakeholders. View Glossary
Award Types
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Status