Doctors, scientists, and other health professionals use biomarkers as tools to obtain information about a person’s health status or response to interventions. Biomarkers, defined as characteristics that indicate biological processes, are essential for monitoring the health of both individuals and communities. Some biomarkers, called surrogate endpoints, are used as substitutes for actual clinical endpoints such as incidence of disease or death. Surrogate endpoints are intended to predict benefit or harm based on scientific evidence, and they are used in practice when it is difficult to collect data based on clinical endpoints.
In 2008, the Food and Drug Administration (FDA) asked the IOM to conduct a study on the evaluation process for biomarkers, focusing on biomarkers and surrogate endpoints in chronic disease. The FDA’s Center for Food Safety and Applied Nutrition initiated this study after reviewing dozens of applications for food health claims based on stated effects on biomarkers.
In this report, the IOM recommends that the FDA adopt a consistent scientific framework for biomarker evaluation in order to achieve a rigorous and transparent process. The biomarker evaluation framework should consist of three steps:
- Analytical validation to ensure biomarker tests are reliable, reproducible, and adequately sensitive and specific
- Qualification to ensure the biomarker is associated with the clinical outcome of concern
- Utilization analysis to determine that the biomarker is appropriate for the proposed use
Additionally, the IOM recommends that the FDA use the same degree of scientific rigor for evaluating biomarker use across regulatory areas, including drugs, medical devices, biologics, foods, and dietary supplements. In the interest of ensuring the public’s health, the proposed biomarker evaluation framework recognizes that scientific information is always evolving and yet allows for the introduction of new, life-saving health interventions.