The Biomarkers Consortium:
Shaping the Future of Biomedical Research

The FNIH Biomarkers Consortium (BC) celebrated 10 years of collaboration, research and impact on regulatory science in 2016. This public-private partnership managed by the FNIH is helping to create a new era of precision medicine and to enable therapies that are better tailored for patients. In 2016, the BC launched five collaborative projects, which are designed to generate tools that will advance the development of therapies in cancer, rheumatoid arthritis, frailty and heart disease. Consortium members also continue to develop new and innovative programs in these and other disease areas ensuring the BC will provide decision-making tools for advancing therapies into the future.

In addition to its work on disease-specific projects, the BC recognized that the use of biological markers (biomarkers) in drug development has been particularly hampered by a lack of clear, predictable and specific regulatory criteria for the evidence required to qualify new markers. To resolve this, a group led by the BC developed and released a “Framework for Defining Evidentiary Criteria for Biomarker Qualification” in October 2016 as a tool to help assess the level of evidence needed to support formal regulatory qualification of biomarkers at the U.S. Food and Drug Administration (FDA). The document was a result of a multi-stakeholder effort that incorporated input from nearly 200 scientific leaders from the FDA, the National Institutes of Health (NIH), the FNIH, the Critical Path Institute (C-Path), Pharmaceutical Research and Manufacturers of America (PhRMA) and multiple pharmaceutical companies. The framework will prove useful in enabling more productive discussions between biomarker developers and the FDA, improving the quality of biomarker qualification submissions and informing future efforts to develop relevant FDA Guidances for evidentiary criteria in biomarker qualification.

Over the past decade, the BC has raised more than $70 million for precompetitive, collaborative projects that include participants from the NIH, the FDA, not-for-profit organizations, academic research organizations, and pharmaceutical and biotechnology companies. More than 20 projects have been launched in 13 different disease areas, resulting in 46 publications written on behalf of or sponsored by the BC. Its work also has been instrumental in testing new models for clinical trials. The BC sponsored one of the first trials to use genetic markers to guide the selection of treatments from among multiple drugs in a single trial, helping establish an accelerated approval pathway for new breast cancer medicines. To date, the work of the BC has supported the advancement of six therapeutics in the drug development process and helped generate four separate FDA Guidance documents.

The BC can solve these and other challenges head-on because it offers a unique environment in which the resources and scientific expertise of its partners can be freely shared. Over the past 10 years, biomarkers have become recognized as indispensable tools for effective drug development, and the BC has equally established itself as the proven model for advancing the scientific validity and practical utility of biomarkers in treating patients. Its mission is as relevant as ever.