Clarifying What We Mean When We Talk About Biomarkers: An NIH/FDA Joint Leadership Council Success

By: Melissa A. Robb, B.S.N., M.S. (RegSci), and Robert M. Califf, M.D.

What if there was a more uniform way to convey key technical terms to help advance scientific progress? Thanks to the Biomarkers, Endpoints, and other Tools (BEST) Resource, we’re one step closer to that goal.

Melissa Robb

Melissa A. Robb, B.S.N., M.S. (RegSci), FDA’s Associate Director for Regulatory Affairs, Office of Medical Policy, Center for Drug Evaluation and Research

Now available on the National Center for Biotechnology Information’s Bookshelf, the BEST Resource was developed through a collaboration of the Food and Drug Administration (FDA) and the National Institutes of Health (NIH). It includes a glossary of terms and definitions that will ensure the consistency and clarity needed to drive progress in biomedical research and clinical care.

Why is this textbook so important? In the spring of 2015, the FDA-NIH Joint Leadership Council identified a problem: Confusion about the definitions and inconsistent use of key terms–including biomarkers, surrogates, and clinical outcome assessments. This can deter progress in developing medical products and thereby potentially compromise efficiency in achieving public health benefits.

Accordingly, the council identified a high priority: harmonizing terms—or making sure that everyone is “speaking the same language”–that describe and categorize types of endpoints.

Members from multiple FDA Centers and NIH institutes formed a working group to focus on creating a glossary. This was the first step to a publicly available and open access textbook that could be continuously updated and expanded.

Robert M. Califf, MD, MACC; Commissioner, U.S. Food and Drug Administration

Robert M. Califf, M.D., Commissioner of the U.S. Food and Drug Administration

As the basis of their work, the group considered existing terminology and definitions. Those include FDA guidance documents and other literature, especially a seminal FDA-sponsored Institute of Medicine study.

The use of biomarkers has recently expanded widely to include fields such as mechanistic biomedical research, clinical trials, drug discovery, medical product development, clinical care, and regulatory science. Recognizing this broad influence and the accepted vernacular of these varied fields, the group sought to first reach consensus around biomarker taxonomy.

For example, there’s misunderstanding about the various types of biomarkers and the distinction between biomarkers and surrogate endpoints. One challenge was to settle upon definitions that were broad enough to be used by diverse communities, including biomedical scientists, translational researchers, clinical researchers, medical product developers, and clinicians, and also across diverse types of products.

Where possible, to provide more context and insight into important terms, examples are given alongside many definitions in the BEST Resource. 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. FDA’s Biomarker Working Group, with representation from all of our Centers, contributed to developing these definitions.

Now we need your help. We need your feedback and comments on the glossary. You can provide them at the BEST (Biomarkers, EndpointS, and other Tools) Resource.

In the meantime, we’ll continue to work on adding context to terms related to regulatory science, clinical trials, and laboratory science.

Effective, unambiguous communication is essential for efficient translation of promising scientific discoveries into approved medical products. Once we are all speaking the same language, we can tackle other challenges to bring the promises of biomedical research and clinical care to fruition.

The FDA-NIH Biomarker Working Group members include: from FDA – Shashi Amur, Robert L. Becker, Robert Califf, Aloka G. Chakravarty, David S. Cho, Nina L. Hunter, Ilan Irony, Christopher Leptak, Kathryn M. O’Callaghan, Michael A. Pacanowski, Elektra J. Papadopoulos, Vasum Peiris, Melissa Robb, Hobart L. Rogers, Rachel E. Sherman, Robert J. Temple, Ann Marie Trentacosti, and Sue Jane Wang; and from the NIH – Holli Hamilton, Pamela McInnes, Lisa M. McShane, and Monica R. Shah.

Melissa A. Robb, B.S.N., M.S. (RegSci), is FDA’s Associate Director for Regulatory Affairs, Office of Medical Policy, Center for Drug Evaluation and Research

Robert M. Califf, M.D., previously FDA’s Deputy Commissioner for Medical Products and Tobacco, became FDA’s Commissioner of Food and Drugs on Feb. 25, 2016

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