USE OF BIOMARKERS IN CLINICAL PRACTICE

To promote appropriate, evidence-based use of biomarkers in clinical practice; further,

To encourage research that evaluates the clinical and safety implications of biomarkers in the care of patients and to guide clinical practice; further,

To promote Food and Drug Administration qualified biomarkers in drug development, regulation, and use in clinical practice; further,

To foster the development of timely and readily available resources about biomarkers and their evidence-based application in clinical practice.

Rationale

Developed jointly by the Food and Drug Administration (FDA) and National Institutes of Health (NIH) through the FDA-NIH Biomarker Working Group, The Biomarkers, EndpointS, and other Tools (BEST) resource serves as a living document to clarify and promote consistent terminology surrounding the use of biomarkers. As defined by the FDA-NIH Biomarker Working Group, a biomarker is “a defined characteristic that is measured as an indicator of normal biological processes, or responses to an exposure or intervention, including therapeutic interventions. Molecular, histologic, radiographic, or physiologic characteristics are types of biomarkers. A biomarker is not an assessment of how a patient feels, functions, or survives.” In comparison to a clinical endpoint, a biomarker is strictly objective and quantifiable, whereas a clinical endpoint reflects the subject’s well-being and health status from the subject’s perspective. Biomarkers are classified by BEST in the following seven categories: susceptibility/risk biomarker, diagnostic biomarker, monitoring biomarker, prognostic biomarker, predictive biomarker, pharmacodynamic/response biomarker, and safety biomarker.

Further, the FDA and its Center for Drug Evaluation and Research are involved in regulating biomarkers in drug development, regulation, and use in clinical practice. Under the FDA Biomarker Qualification Program, researchers can request qualification of a biomarker in the use of drug development. The FDA’s involvement in biomarker qualifications allows for the development of a regulatory process to investigate the safety and efficacy of biomarkers. Innovative and newly discovered biomarkers are investigated or found unexpectedly in clinical research. Recently published articles demonstrate newly discovered biomarkers that potentially show clinical efficacy; however, there is debate about how to conduct further research to establish a biomarker’s clinical efficacy.

This growth in discovery and application of established biomarkers in practice presents several practice issues, including use of recognized biomarkers, collaborating with practitioners concerning newly discovered or rising biomarkers, conducting research on the outcomes of the use of various biomarkers, and integrating use of biomarkers into practice.