Report at a Glance
Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease
Doctors, scientists, and other health professionals use biomarkers as tools to obtain information about a person’s health status or response to interventions. Defined as characteristics that indicate biological processes, biomarkers 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 Institute of Medicine (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. The report’s authoring committee recommends that the FDA adopt a consistent scientific process and framework for biomarker evaluation in order to achieve a rigorous and transparent process for all stakeholders. The committee tests this framework using case studies of biomarkers and surrogate endpoints in various diseases, such as low-density lipoprotein (LDL) and high-density lipoprotein cholesterol levels in cardiovascular disease.