Section 3011 of the 21st Century Cures Act established section 507 of the Federal Food, Drug, and Cosmetic Act (FD&C Act), on qualification of drug development tools. Section 3011(b)(3)(A) of the 21st Century Cures Act states; “For purposes of informing guidance under this subsection, the Secretary shall, in consultation with biomedical research consortia and other interested parties through a collaborative public process, establish taxonomy for the classification of biomarkers (and related scientific concepts) for use in drug development”. The eBook posting on the National Library of Medicine website responds to this legislative requirement.
In the Spring of 2015 the FDA-NIH Joint Leadership Council identified the harmonization of terms used in translational science and medical product development as a priority need, with a focus on terms related to study endpoints and biomarkers. Working together with the goals of improving communication, aligning expectations, and improving scientific understanding, the two agencies developed the BEST (Biomarkers, EndpointS, and other Tools) Resource. The first phase of BEST comprises a glossary that clarifies important definitions and describes some of the hierarchical relationships, connections, and dependencies among the terms it contains.
The BEST glossary aims to capture distinctions between biomarkers and clinical assessments and to describe their distinct roles in biomedical research, clinical practice, and medical product development. Because the glossary is intended to be broadly applicable to multiple communities of users and stakeholders, its definitions address nuances of usage and interpretation for a wide variety of terms currently in use. Further, based on differing stakeholder needs, it has built in flexibility, when possible and appropriate, to accommodate those interests. NIH and FDA intend to use the definitions included in this glossary when communicating on topics related to its contents (e.g., biomarkers) to ensure a consistent use of the terms and therefore, a common understanding of the issues.
The BEST glossary is meant to be a “living” resource that will be periodically updated with additional terms and clarifying information. We welcome feedback, including specific proposed edits with rationale, from all stakeholders, including the scientific and medical communities, patients, providers, industry, and regulators, so that as we refine and elaborate on these terms, they will remain relevant, thus fostering consistent usage and ultimately help to accelerate development and refinement of medical products which lead to improvements in health outcomes. Suggested revisions will be considered on a regular basis.