What type of rare disease research will PCORI fund?

PCORI is interested in funding patient-centered comparative effectiveness research (CER) that addresses outstanding questions about the prevention, diagnosis, and treatment of rare diseases. Investigators applying for PCORI funding must make the case that the study addresses a clinical choice and decisional dilemma faced by patients and healthcare providers. As part of the justification for the importance of the study, investigators should describe how the interventions being studied are currently used in clinical practice for the diagnosis, treatment, or management of the condition, both in terms of how widely they are used and any particular clinical and population considerations. Additionally, PCORI encourages investigators to use validated outcome measures, including patient-reported outcomes, and provide preliminary data that support the proposed measures.