would enable drug companies and regulators to decide what level of evidence may be required for a particular biomarker application, and acknowledged some aspects of the committee’s recommended framework were steps in the right direction. Jack Zakowski agreed with the committee’s biomarker evaluation framework, especially the focus on the interdependence of the three steps of the framework, and Guy Johnson noted that the framework was comprehensive. While there was general agreement on the need for a biomarker evaluation framework, several speakers expressed differing opinions on specific aspects of the framework and its implications.

James Mayne and Dr. Williams were concerned that the report did not specify criteria that should be applied to biomarkers at each step of the evaluation framework. “I eagerly tore through the document … looking for the actual elements of the decision framework, by what criteria would decisions be made in the regulatory space,” said Dr. Mayne. “That was not provided, at least not in the detail I was looking for.” John R. Ball noted that the committee didn’t view their recommendations as the last word in biomarker evaluation but as a fulfillment of their charge to develop a framework for biomarker evaluation across the Food and Drug Administration (FDA) regulatory spectrum. He added that although the Center for Food Safety and Applied Nutrition’s (CFSAN’s) work would be simplified if the Institute of Medicine (IOM) committee had developed a five-item checklist of criteria that every biomarker used in a health claim had to fulfill, the committee found this notion unrealistic. Given limited understanding of chronic disease and the biological significance of existing biomarkers, the committee concluded that evaluation must be conducted by expert panels on a case-by-case basis, Dr. Ball said. For further discussion of the committee’s evaluation framework and related recommendations, see Chapter 3 of its report (IOM, 2010).

The effect of the biomarker evaluation framework on innovation generated additional discussion. By employing a rigorous biomarker evaluation framework, there were concerns that this may unintentionally discourage research in the area of biomarkers. However, many speakers noted that the biomarker evaluation framework will not have a chilling effect on biomarker research and development. Thomas Fleming suggested that a lack of clarity, both from the regulatory and scientific perspective, on biomarker evaluation standards is much more likely to inhibit innovation. Implementation of Recommendation 3 of the report could help FDA to bring scientific and regulatory clarity to biomarker evaluation across the FDA’s centers and regulated product categories. David DeMets added that the inappropriate use of a biomarker would have negative effects on innovation. Dr. Mayne said that the report clearly stated that the framework is not intended for biomarkers used in the discovery space: “I think



The National Academies | 500 Fifth St. N.W. | Washington, D.C. 20001
Copyright © National Academy of Sciences. All rights reserved.
Terms of Use and Privacy Statement