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Introduction

Biomarkers are characteristics that are objectively measured and evaluated as indicators of normal biological processes, pathogenic processes, or pharmacologic responses to an intervention. Cholesterol and blood sugar levels are biomarkers, as are blood pressure, enzyme levels, measurements of tumor size from magnetic resonance imaging (MRI) or computed tomography (CT), and the biochemical and genetic variations observed in age-related macular degeneration. Biomarkers can enable faster, more efficient clinical trials for life-saving and health-promoting interventions. They can help improve understanding of healthy dietary choices, and they can help public health professionals to identify and track health concerns. Biomarkers help health care practitioners and their patients make decisions about patient care. (IOM, 2010)

Due to the absence of an agreed-upon process for biomarker evaluation, the Food and Drug Administration (FDA) requested that the Institute of Medicine (IOM) recommend a framework for the evaluation of biomarkers in the chronic disease setting and make ancillary recommendations for its application. In a report published in May 2010, the IOM Committee on Qualification of Biomarkers and Surrogate Endpoints in Chronic Disease (henceforth, “the committee”) recommended such a framework, comprising three critical components: analytical validity, evidentiary qualification, and utilization analysis (IOM, 2010). This framework is intended to bring consistency and transparency to the previously nonuniform process of biomarker evaluation. The full summary of the committee’s report is included in Appendix B.

On June 21 and 22, 2010, the IOM convened a 2-day discussion forum in Washington, DC, in order to provide an opportunity for stakeholders to learn about, react to, and discuss the report, Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease (IOM, 2010; see Appendix A). The discussion forum was attended by representatives of several FDA centers, including the Center for Food Safety and Applied Nutrition (CFSAN), the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and the Center for Devices and Radiological Health (CDRH); by representatives of the National Institutes of Health (NIH), including the National Heart, Lung, and Blood Institute (NHLBI) and the Office of Dietary Supplements; by representatives of industry and industrial professional societies, including food, dietary



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1 Introduction Biomarkers are characteristics that are objectively measured and evaluated as indicators of normal biological processes, pathogenic processes, or pharmacologic responses to an intervention. Cholesterol and blood sugar levels are biomarkers, as are blood pressure, enzyme levels, measurements of tumor size from magnetic resonance imaging (MRI) or computed tomography (CT), and the biochemical and genetic variations observed in age-related macular degeneration. Biomarkers can enable faster, more efficient clinical trials for life-saving and health-promoting interventions. They can help improve understanding of healthy dietary choices, and they can help public health professionals to identify and track health con - cerns. Biomarkers help health care practitioners and their patients make deci - sions about patient care. (IOM, 2010) Due to the absence of an agreed-upon process for biomarker eval- uation, the Food and Drug Administration (FDA) requested that the Institute of Medicine (IOM) recommend a framework for the evaluation of biomarkers in the chronic disease setting and make ancillary recom - mendations for its application. In a report published in May 2010, the IOM Committee on Qualification of Biomarkers and Surrogate Endpoints in Chronic Disease (henceforth, “the committee”) recommended such a framework, comprising three critical components: analytical validity, evidentiary qualification, and utilization analysis (IOM, 2010). This frame- work is intended to bring consistency and transparency to the previously nonuniform process of biomarker evaluation. The full summary of the committee’s report is included in Appendix B. On June 21 and 22, 2010, the IOM convened a 2-day discussion forum in Washington, DC, in order to provide an opportunity for stakeholders to learn about, react to, and discuss the report, Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease (IOM, 2010; see Appendix A). The discussion forum was attended by representatives of several FDA centers, including the Center for Food Safety and Applied Nutrition (CFSAN), the Center for Drug Evaluation and Research (CDER), the Center for Bio- logics Evaluation and Research (CBER), and the Center for Devices and Radiological Health (CDRH); by representatives of the National Institutes of Health (NIH), including the National Heart, Lung, and Blood Institute (NHLBI) and the Office of Dietary Supplements; by representatives of industry and industrial professional societies, including food, dietary 1

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2 BIOMARKER AND SURROGATE ENDPOINT EVALUATION supplements, pharmaceuticals, medical diagnostics, and devices; and by representatives of public health, consumer, and nutrition consulting orga- nizations, as well as by members of the committee and IOM staff. Presentations reviewed the committee’s work process, recommenda- tions, and provided perspectives on the report from the point of view of participants from the FDA, the NIH, and from a diverse group of industry stakeholders; all such sessions were followed by panel discussions. Many presenters emphasized that the views they expressed were theirs and not necessarily those of their organizations or institutions. Thomas Fleming, professor of biostatistics and statistics at the University of Washington, gave a keynote presentation on the critical issues in the validation of sur- rogate endpoints, a specific use of a biomarker. This document recounts the discussion forum proceedings, focusing in turn on each represented sector. A summary of Dr. Fleming’s presen- tation then sets the committee’s report within the context of biomarker utilization. Lastly, this summary examines the main themes raised by stakeholders, and the challenges and opportunities presented to stake- holders by the report’s recommendations.