The Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER), as a co-sponsor with the FNIH Biomarkers Consortium, hosted a public meeting titled Developing an Evidentiary Criteria Framework for Safety Biomarkers Qualification Workshop on April 14 and 15, 2016.
This workshop aimed to create a harmonized framework among scientific stakeholders such as FDA, NIH, biopharmaceutical industry partners, academic Researchers and patient groups for determining levels of evidence required to qualify biomarkers for use in drug development, with an emphasis on biomarkers used to assess drug safety. Participants developed a general framework for biomarker qualification and applied that framework to different contexts of use related to drug safety, including assessment of specific case studies qualifying clinical markers of toxicity in different organ systems.
Critical Path Institute – Predictive Safety Testing Consortium
Food and Drug Administration
Merck Sharp & Dohme Corp.
National Cancer Institute (NCI)
National Institute of Mental Health (NIMH)
Pharmaceutical Research and Manufacturers of America
Takeda Pharmaceuticals International, Inc.
Joseph Menetski, Associate Vice President of Research Partnerships, [email protected]
The FNIH/FDA Evidentiary Framework Workshop, Core Writing Group
- Jiri Aubrecht, Senior Director, Pfizer
- Linda Brady Division Director and Biomarkers Consortium Neuroscience Steering Committee Co-Chair, NIMH/NIH
- Martha Brumfield, President & CEO, Critical Path Institute
- Bill Chin, Executive VP Scientific and Regulatory Affairs and Biomarkers Consortium Executive Committee Member, PhRMA
- Steve Hoffmann, Scientific Program Manager, Foundation for the National Institutes of Health
- Gary Kelloff, Special Advisor and Biomarkers Consortium Cancer Steering Committee Co-Chair, NCI/NIH
- Gabriela Lavezzari, Research Director, Duke-Robert J. Margolis, MD Center for Health Policy (formally PhRMA)
- Chris Leptak, Co-Director Biomarker Qualification Program and Biomarkers Consortium Executive Committee Member, FDA
- Joseph Menetski, Associate Vice President of Research Partnerships, Foundation for the National Institutes of Health
- Rajesh Ranganathan, Vice President, Scientific & Regulatory Advocacy, (formally PhRMA)
- John-Michael Sauer, Executive Director, Predictive Safety Testing Consortium, Critical Path Institute
- Frank Sistare, Associate Vice President, Merck
- John Wagner, Senior Vice President and Biomarkers Consortium Executive Committee Member, Takeda
- David Wholley, Senior Vice President Research Partnerships, Foundation for the National Institutes of Health
- Tanja Zabka, Pathologist/Senior Scientist, Genentech
- Improve the quality of drug safety biomarker submissions to the FDA.
- Enhance the predictability of the safety biomarker qualification process.
- Clarify the type and extent of evidentiary criteria needed to support the biomarker’s context of use (COU).
Results & Accomplishments
Scientific Publications and Guidance Documents
This project led to several peer-reviewed scientific publications, and to the creation of a Framework Document to inform FDA and other regulators’ creation of new guidance around qualifying drug safety biomarkers.
- What evidence do we need for biomarker qualification? C. Leptak, J. P. Menetski, J. A. Wagner, J. Aubrecht, L. Brady, M. Brumfield, W. W. Chin, S. Hoffmann, G. Kelloff, G. Lavezzari, R. Ranganathan, J.-M. Sauer, F. D. Sistare, T. Zabka, D. Wholley, Sci. Transl. Med. 9, eaal4599 (2017).
- Evidentiary Criteria Framework Final Version
- FNIH FDA April Biomarker Evidentiary Criteria Meeting Public Report
- Workshop Agenda
- Additional Information for Registered Participants