FDA: Considerations for Use of Histopathology and Its Associated Methodologies to Support Biomarker Qualification

This guidance is intended to assist submitters of a biomarker for qualification that conduct nonclinical studies for which histopathology is used as a reference or truth standard (Zhou et al. 2011). Scientifically rigorous evaluation of biomarker performance in relation to histopathologic changes is essential in these studies because they may provide direct evidence to support nonclinical biomarker qualification or supportive translational data to aid in the development and qualification of clinical biomarkers for a proposed context of use. This guidance discusses the issues that should be considered when generating histopathology data in nonclinical biomarker qualification studies and outlines the scientific standards recommended for biomarker characterization and qualification. The biomarker qualification process is described in a separate guidance, Qualification Process for Drug Development Tools.