BOX 5-1Summary of Earlier IOM Report Recommendations for Effective Biomarker Evaluation

The Evaluation Framework


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


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


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


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.


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


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


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

SOURCE: IOM, 2010a.

From: 5, Development of New Therapeutic Drugs and Biologics for Rare Diseases

Cover of Rare Diseases and Orphan Products
Rare Diseases and Orphan Products: Accelerating Research and Development.
Institute of Medicine (US) Committee on Accelerating Rare Diseases Research and Orphan Product Development; Field MJ, Boat TF, editors.
Washington (DC): National Academies Press (US); 2010.
Copyright © 2010, National Academy of Sciences.

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