and treatment. More detailed understanding of disease biology has the potential to lead to more effective prevention and treatment approaches. Biomarkers are critical to progress in these areas, and it will be important that newly discovered biomarkers be adequately studied before being adopted into routine clinical management of patients.


In 2008, the Food and Drug Administration’s (FDA’s) Center for Food Safety and Applied Nutrition (CFSAN), in conjunction with the FDA’s Center for Drug Evaluation and Research, approached the IOM for advice on the topic of biomarker and surrogate endpoint evaluation, noting the limited number of surrogate endpoints available, the high cost of evaluating possible surrogate endpoints biomarkers, and the absence of an agreed-upon, systematic, transparent process for biomarker evaluation. Study developers were also interested in learning whether principles of biomarker qualification or evaluation learned in the drug development setting would also be generally applicable in other FDA-regulated product categories, such as foods and supplements. As part of its efforts within the Critical Path Initiative (CPI),1 CFSAN requested that the IOM charge an expert committee with the following task:

An Institute of Medicine (IOM) committee will be convened to generate recommendations on the qualification process for biomarkers, with a focus on risk biomarkers and surrogate endpoints in chronic disease. These recommendations will consider existing prototypes for qualification of biomarkers used in drug development. The committee will recommend a framework for qualification and test it using case studies of risk biomarkers and surrogate endpoints for coronary heart disease (CHD) such as low-density lipoprotein (LDL) and high-density lipoprotein (HDL) cholesterol levels. In particular, the committee will:

  1. Conduct a review of current approaches to qualifying biomarkers.

  2. Recommend a framework that can be used to rank biomarkers according to the types and quality of evidence, considering context of use for a range of product types.

  3. Demonstrate applications through case studies.

  4. Make ancillary recommendations for the application, enhanced development, and use of risk biomarkers and surrogate endpoints in chronic disease.2




The terminology in the statement of task differs in a few ways from the terminology of this report. As will be explained in Chapter 3, the committee’s terminology replaces qualification with evaluation in many instances, and risk biomarker with biomarker.

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