BOX S-1

Summary of Recommendations for Effective Biomarker Evaluation

The Evaluation Framework

1.

The biomarker evaluation process should consist of the following three steps:

1a.

Analytical validation: analyses of available evidence on the analytical performance of an assay;

1b.

Qualification: assessment of available evidence on associations between the biomarker and disease states, including data showing effects of interventions on both the biomarker and clinical outcomes; and

1c.

Utilization: contextual analysis based on the specific use proposed and the applicability of available evidence to this use. This includes a determination of whether the validation and qualification conducted provide sufficient support for the use proposed.

2a.

For biomarkers with regulatory impact, the Food and Drug Administration (FDA) should convene expert panels to evaluate biomarkers and biomarker tests.

2b.

Initial evaluation of analytical validation and qualification should be conducted separately from a particular context of use.

2c.

The expert panels should reevaluate analytical validation, qualification, and utilization on a continual and a case-by-case basis.

Scientific Process Harmonization

3.

The FDA should use the same degree of scientific rigor for evaluation of biomarkers across regulatory areas, whether they are proposed for use in the arenas of drugs, medical devices, biologics, or foods and dietary supplements. Congress may need to strengthen FDA authority to accomplish this goal.

4.

The FDA should take into account a nutrient or food’s source as well as any modifying effects of the food or supplement that serves as the delivery vehicle and the dietary patterns associated with consumption of the nutrient or food when reviewing health-related label claims and the safety of food and supplements. Congress may need to strengthen FDA authority to accomplish this goal.

Biomarkers are measurements that indicate biological processes (see Box S-2 for definitions of key terms). Biomarkers include physiological measurements, blood tests, and other chemical analyses of tissue or bodily fluids, genetic or metabolic data, and measurements from images. Cholesterol and blood sugar levels are biomarkers, as are blood pressure, enzyme levels, measurements of tumor size from MRI or CT, and the biochemical and genetic variations observed in age-related macular degeneration. Emerging technologies have also enabled the use of simul-



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