Kidney Toxicity Biomarker Qualification: A Journey Along the Critical Path

• Activity Number: 542
• Type: Invited
• Date/Time: Wednesday, August 1, 2012 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Dan Holder*+
• Companies: Merck Research Laboratories
• Keywords: toxicity biomarkers ; Kidney ; statistics ; PSTC

Abstract

The tremendous utility and potential of biomarkers have created a strong desire for them in the biomedical community. However, the process that leads from biomarker discovery through development and qualification can be complex and difficult. Assessment of biomarker performance is a critical task throughout the process that is best addressed with the aid of statistical reasoning. In this presentation we discuss how statistical analysis can and should play more than a confirmatory role in biomarker research. We argue that there are different stages in biomarker research and it is important that the statistical methods used are appropriately matched to the stage of development and the biomarker's intended use. We discuss sensitivity, specificity, and the receiver-operator characteristic curve as basic statistical concepts for assessing biomarker performance. We use examples from the successful qualification of kidney toxicity biomarkers as well as current efforts to extend those claims to illustrate some of the concepts and challenges.