Overview
The Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium, in partnership with the Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research, hosted a public meeting entitled Framework for Defining Evidentiary Criteria: Surrogate Endpoint Qualification Workshop on July 30th and 31st, 2018.
This workshop aimed to create alignment among scientific stakeholders including FDA, NIH, the biopharmaceutical industry, academic Researchers and patient groups regarding a proposed framework for determining the levels of evidence required to qualify biomarkers for use in drug development, with an emphasis on surrogate endpoints. A primary aim of the meeting was to elaborate the general framework for biomarker qualification along with specific application to different contexts of use (COUs), including assessment of several representative case studies involving surrogate endpoint markers of specific clinical outcome measures.
Partners
- Children’s Hospital of Philadelphia
- Critical Path Institute
- Dana-Farber Cancer Institute
- Food and Drug Administration
- National Cancer Institute (NCI)
- National Center for Advancing Translational Sciences (NCATS)
- Pfizer Inc
- University of Chicago
Contact
- Joseph Menetski, Associate Vice President, Research Partnerships, [email protected]
Surrogate Endpoint Core Program Committee:
- Stacey Adam – Associate Vice President, Research Partnerships, FNIH
- Ken Anderson -Program Director, Dana–Farber Cancer Institute
- Steve Broadbent – Chief Operating Officer, Critical Path Institute
- Martha Brumfield – President & CEO, Critical Path Institute
- Sara Eggers – Office of Strategic Programs, FDA/CDER
- Steve Hoffmann – Associate Vice President, Research Partnerships, FNIH
- Laura Jaeger – Scientific Policy Advisor, FDA/CDER
- Reza Kazemi-Tabriz – Operations Research Analyst, FDA/CDER
- Gary Kelloff – Special Advisor and Biomarkers Consortium Cancer Steering Committee Co-Chair, NCI/NIH
- Leila Lackey – Operations Research Analyst, FDA/CDER
- Chris Leptak – Director Biomarker Qualification Program and Biomarkers Consortium Executive Committee Member, FDA
- Lisa McShane – Sr. Statistician, NIH/NCI
- Joseph Menetski – Associate VP Research Partnerships, FNIH
- Theresa Mullin – Associate Director for Strategic Initiatives, FDA/CDER
- Elizabeth Ottinger – Senior Project Manager, NIH/NCATS
- James Revkin – Senior Director, Pfizer
- John-Michael Sauer – Executive Director, Predictive Safety Testing Consortium, Critical Path Institute
- Myrlene Staten – Program Director and Biomarkers Consortium Metabolic Disorders Steering Committee Co-Chair NIH/NIDDK
- Adeline Vanderver – Program Director of the Leukodystrophy CoE, Children’s Hospital of Philadelphia
- John Wagner – Senior VP and Biomarkers Consortium Executive Committee Member, Takeda
- Steve Williams – Chief Medical Officer, SomaLogic Inc
- Robert Wolk – Director, Pfizer
Presentations from the Workshop:
- Surrogate Endpoint Qualification Workshop Introduction
- Joseph Menetski, Ph.D.
- Associate Vice President of Research Partnerships
- Foundation for the National Institutes of Health
- Brief Overview of Biomarkers: Value, Limitations, and the Biomarker Qualification Program
- Peter Stein, M.D.
- Deputy Director
- Office New Drugs, CDER
- U.S. Food & Drug Administration
- 21st Century Cures Biomarker Qualification :Updates, Deliverables, and Progress Towards Defined Evidence
- Christopher Leptak, M.D., Ph.D.
- Director of the Biomarker Qualification Program
- Office New Drugs, CDER
- U.S. Food & Drug Administration
- A Framework for Defining Evidentiary Criteria for Biomarker Qualification
- John Wagner, M.D., Ph.D.
- Senior Vice President and Head
- Clinical and Translational Sciences
- Takeda Pharmaceutical International
- Thoughts on Evidentiary Criteria for Biomarker Qualification: A “Decision Science” Perspective
- Reza Kazemi-Tabriz, Ph.D.
- Performance Analysis and Data Services Staff Director
- U.S. Food & Drug Administration
- Concepts and Case Study Template for Surrogate Endpoints Workshop
- Lisa M. McShane, Ph.D.
- Acting Associate Director, Division of Cancer Treatment and Diagnosis
- National Cancer Institute
- National Institutes of Health
- LDL-C and HDL-C as Surrogate Endpoints
- James Revkin, M.D.
- Senior Director, Global Clinical Lead
- Pfizer
- Minimal Residual Disease (MRD) in Multiple Myeloma (MM)
- Ken Anderson, M.D.
- Chief, Division of Hematologic Neoplasia
- Director of the Jerome Lipper Multiple Myeloma Center and
- Vice Chair of the Joint Program in Transfusion Medicine, Dana-Farber Cancer Institute
- Total Kidney Volume in Autosomal Dominant Polycystic Kidney Disease
- Arlene Chapman, M.D.
- Professor of Medicine, Chief, Section of Nephrology, Department of Medicine
- University of Chicago
- Alexander Disease
- Adeline Vanderver, M.D., Amy Waldman, M.D., MSCE
- Leukodystrophy Center of Excellence, Children’s Hospital of Philadelphia
- Albee Messing, VMD, Ph.D.
- Waisman Center, University of Wisconsin – Madison
- Case Study Framework Summaries
Goals
- Improve the quality and standardization of submissions to the FDA
- Better characterize and define surrogate endpoints for drug development through example case studies
- Support FDA in the development of Guidance(s) for the level of evidentiary criteria sufficient for surrogate endpoint qualification
Results & Accomplishments
Guidance Documents
- FDA Facts: Biomarkers and Surrogate Endpoints
- Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics, May 2017
- Surrogate Endpoint Resources for Drug and Biologic Development
- Table of Surrogate Endpoints That Were the Basis of Drug Approval or Licensure
- Considerations for Discussion of a New Surrogate Endpoint(s) at a Type C PDUFA Meeting Request (PDF-111KB)
- BEST Resource Taxonomy
Scientific Publications
- What evidence do we need for biomarker qualification? Leptak G, Menetski JP, Wagner JA, Aubrecht J, Brady L, Brumfield M, Chin WW, Hoffmann S, Kelloff G, Lavezzari G, Ranganathan R, Sauer J-M, Sistare FD, Zabka T, Wholley D. Sci Transl Med. 2017 Nov 22;9(417).
- Evidentiary Criteria Framework Final Version
- Workshop Agenda