Biomarkers Consortium - Workshop: Developing an Evidentiary Criteria Framework for Safety Biomarkers Qualification

Ensuring drug safety by establishing standard safety evaluation criteria

The Problem
The lack of standard assessment criteria makes it difficult for FDA to consistently evaluate the safety of the wide variety of drugs and diminishes the overall quality of many drug safety submissions to FDA.
The Solution
This workshop led to proposed criteria for evaluating the safety of drugs being tested in clinical trials, which will improve the quality of drug safety submissions to FDA and make it easier for FDA to compare the safety of different drugs.

Overview

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.

Partners

Critical Path Institute – Predictive Safety Testing Consortium

Duke University

Food and Drug Administration

Genentech, Inc.

Merck Sharp & Dohme Corp.

National Cancer Institute (NCI)

National Institute of Mental Health (NIMH)

Pfizer Inc

Pharmaceutical Research and Manufacturers of America

Takeda Pharmaceuticals International, Inc.

FNIH Contact

Joseph Menetski, Associate Vice President of Research Partnerships, jmenetski@fnih.org

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

Goals

  • 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.

Scientific Publications

Guidance Document Resulting from this Workshop

Media