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On May 7, 2024, PCORI will begin accepting Letter of Intent (LOI) submissions for the Addressing Rare Diseases Topical PCORI Funding Announcement (PFA) under Cycle 2 2024.


A male physician administers a check-up for a female patient in a medical setting
A rare disease is defined as one that affects less than 200,000 individuals, according to the National Institutes of Health. It is estimated that there are more than 10,000 rare diseases affecting about 30 million Americans.

This Topical PFA will seek applications that propose to address patient-centered comparative clinical effectiveness research (CER) questions that 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).

  • 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.

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. 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.

Learn more about this PFA and attend a virtual applicant town hall on May 14.

This PFA will open on May 7 and LOIs will be due on June 4 by 5 pm (ET).

Posted: February 6, 2024

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