Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease

摘要

Biomarkers are characteristics that are objectively measured and evaluated as indicators of normal biological processes, pathogenic processes, or pharmacologic responses to an intervention. Cholesterol and blood sugar levels are biomarkers, as are blood pressure, enzyme levels, measurements of tumor size from magnetic resonance imaging (MRI) or computed tomography (CT), and the biochemical and genetic variations observed in age-related macular degeneration. Biomarkers can enable faster, more efficient clinical trials for life-saving and health-promoting interventions. They can help improve understanding of healthy dietary choices, and they can help public health professionals to identify and track health concerns. Biomarkers help health care practitioners and their patients make decisions about patient care. The use of biomarkers depends on the quality of data that supports their use and on the context in which they are applied. Evaluation of the quality of the measurements and data linking the biomarkers to clinical outcomes is important for assessing biomarker utility. The Food and Drug Administration (FDA) requested the Institute of Medicine (IOM) to recommend a framework for the evaluation of biomarkers. The committee has recommended such a framework, with critical components of analytical validity, evidentiary qualification, and utilization analysis. The framework is intended to bring consistency and transparency to a previously non-uniform process. During its deliberations, the committee identified a need for the FDA to evaluate biomarker use with the same degree of scientific rigor across the product categories regulated by the agency, including drugs, biologics, devices, foods, and supplements. The committee has also recommended strategies for implementing the evaluation framework, supporting the use of evidence-based regulation and the protection and promotion of public health.