PCORI Help Center

May I propose a pharmacological intervention that is not FDA approved, such as off-label drug use in pediatric population?

In some instances, particularly with respect to research in pediatrics, it may be the case that off-label use of drugs is a realistic choice, already being prescribed in this population, 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 clearly describe how often the interventions being compared are currently used in clinical practice for treatment of the condition being studied.

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