PCORI Help Center

Will PCORI consider rare disease CER studies where one of the interventions being compared is a drug being used off-label in clinical practices?

In some instances, particularly with respect to research in rare diseases, it may be the case that off-label use of drugs is a realistic choice and represents appropriately a potential alternative therapy in need of evaluation. In these instances, a specific rationale to support the inclusion of an off-label therapy should be clearly documented in the application. As part of the justification for the importance of the study, investigators should describe how often the interventions being compared are currently used in clinical practice for treatment of the condition being studied.

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