PCORI generally applies the same evaluation criteria to review all research applications. However, we understand that existing evidence regarding interventions for rare diseases is often limited. In articulating the clinical decision dilemma, investigators must describe the existing evidence on the proposed interventions, even if the data are limited. If the data have not been published, investigators should describe why they are not, as well as any plans for the public release of the data. In some instances, PCORI may consider applications that involve interventions with limited evidence if the application compares two or more interventions that are currently being used in medical practice to treat patients with a given rare disease. Please note that applicants will need to document and address the limitations and risks of using minimal efficacy data.
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