Proceedings of the University of Pennsylvania 5th annual conference on statistical issues in clinical trials: emerging statistical issues in biomarker validation for clinical trials.

Abstract

This issue of Clinical Trials contains the proceedings of the fifth in a series of symposia held at the University of Pennsylvania on current statistical issues in clinical trials. This symposium, held in spring 2012, focused on emerging statistical issues in biomarker validation for clinical trials, a topic highly relevant to the ever-increasing concerns about clinical trial efficiency While the titles of the papers in the Table are self explanatory, the presentations and panel discussions offer a very broad spectrum of viewpoints and emphases from both long-time and more recent researchers. Simple questions which create difficult statistical issues such as how to calibrate new biomarkers against imperfect gold standards; regulatory perspective on when biomarkers are ‘good’ enough to be used in clinical trials; how to quantify treatment effect explained by biomarkers subject to measurement error are addressed (see Table). Other topics include fraud in Omics biomarker validation, and the approach to assessing clinical utility along with statistical validation. Expert panels provided thoughtful commentary on the presentations, and there were provocative and insightful questions and comments from the floor; these discussions are included along with the papers. Table 2012 Conference on Emerging Statistical Issues in Biomarker Validation for Clinical Trials The Penn conference series is aimed at facilitating advances in approaches to the statistical design, implementation and analysis of clinical trials. Earlier conferences in this series focused on proof-of-concept studies and ‘go–no go’ decision making [3], development of targeted therapies [4], comparative effectiveness studies [5] and emerging statistical issues in the conduct and monitoring of clinical trials [6]