Clinical Evidence Supporting Pharmacogenomic Biomarker Testing Provided in US Food and Drug Administration Drug Labels
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CitationWang, Bo. 2016. Clinical Evidence Supporting Pharmacogenomic Biomarker Testing Provided in US Food and Drug Administration Drug Labels. Doctoral dissertation, Harvard Medical School.
AbstractBackground: Genetic biomarkers that predict a drug’s efficacy or likelihood of toxicity are assuming increasingly important roles in the personalization of pharmacotherapy, but there is concern that there may be insufficient evidence linking use of some biomarkers to clinical benefit. Nevertheless, information about the use of biomarkers appears in the drug label for many prescription medications. This may add confusion to the clinical decision-making process.
Objective: To evaluate the evidence supporting pharmacogenomic biomarker testing in drug labels, how frequently testing is recommended, and completeness of citation of the supporting studies.
Methods: We used publicly-available databases from the US Food and Drug Administration (FDA) to identify drug labels that described the use of a biomarker and evaluated whether the label contained or referenced convincing evidence of its clinical validity (i.e., the ability to predict phenotype) and clinical utility (i.e., the ability to improve clinical outcomes) using guidelines published by the Evaluation of Genomic Applications in Practice and Prevention (EGAPP) group. We graded the completeness of the citation of supporting studies and determined whether the label recommended incorporation of biomarker test results in therapeutic decision-making.
Results: Of the 119 drug-biomarker combinations, only 36% (n=43) had labels that provided convincing clinical validity evidence while 15% (n=18) provided convincing evidence for clinical utility. Fifty-one percent of the labels (51%, n=61) made recommendations based on the results of a biomarker test; 30% of these contained convincing clinical utility data. A full description of supporting studies was included in 11% (n=13) of the labels.
Discussion: Fewer than one-sixth of drug labels contained or referenced convincing evidence for clinical utility of biomarker testing while over half made recommendations based on biomarker test results. It may be premature to include biomarker testing recommendations in drug labels when convincing data linking testing to patient outcomes does not exist.
Citable link to this pagehttp://nrs.harvard.edu/urn-3:HUL.InstRepos:27007749