FDA Issues Pharmacoepidemiologic Safety Study Guidance

FDA has released guidance on best practices for conducting and reporting pharmacoepidemiologic safety studies, specifically studies that use electronic healthcare data including administrative claims data and electronic medical record data.

Included in the guidance are recommendations for documenting the design, analysis, and results of pharmacoepidemiologic safety studies to optimize FDA’s review of protocols and final reports that are submitted to the agency. FDA defines a pharmacoepidemiologic safety study in relation to the guidance as “an observational study designed to assess the risk associated with a drug exposure and to test prespecified hypotheses.” The guidance does not address medical devices.

The guidance document provides industry and FDA with direction on designing, conducting, and analyzing pharmacoepidemiologic safety studies. It also creates a framework for industry to use when submitting pharmacoepidemiologic safety-study protocols and final reports to FDA.

FDA states in the guidance document that, although the guidance is not intended to address basic epidemiologic principles, many of the concepts may apply to pharmacoepidemiologic safety studies that use other types of data.