Methodological issues with pharmacogenomic biomarkers in relation to clinical development and patient selection - Scientific guideline

This document highlights key principles that should be considered by stakeholders with focus on use of genomic biomarkers in relation to patient selection and associated issues with trial methodology.

Keywords: Clinical trial designs, enriched design, genomic biomarkers (GBMS), hybrid design, predictive markers, pharmacogenomics, retrospective data analyses

Current version

Reflection paper on methodological issues associated with pharmacogenomic biomarkers in relation to clinical development and patient selection

Consultation dates: 09/06/2011 to 25/11/2011Draft: consultation closedReference Number: EMA/446337/2011Summary:

The availability of techniques that facilitate study of the human genome has led to an exponential increase in investigation into genomic biomarkers (GBMs) for diagnosis of specific diseases, as a marker of response to treatment or of prognosis. Theoretically genomic BMs should offer the advantage of improved specificity and reduction of heterogeneity that is an integral part of phenotypic population grouping.