Many people naturally assume that the claims made for foods and nutritional supplements have the same degree of scientific grounding as those for medication, but that is not always the case. The IOM recommends that the FDA adopt a consistent scientific framework for biomarker evaluation in order to achieve a rigorous and transparent process.

Contents

• THE NATIONAL ACADEMIES
• COMMITTEE ON QUALIFICATION OF BIOMARKERS AND SURROGATE ENDPOINTS IN CHRONIC DISEASE
• Reviewers
• Preface
• Acknowledgments
• Summary
• 1. Introduction
  • ORIGIN OF THE TASK
  • DEFINITIONS
  • RELATED IOM WORK
  • FRAMEWORK OF THE REPORT
  • REFERENCES
• 2. Review: Evaluating and Regulating Biomarker Use
  • INTRODUCTION
  • SURVEY OF BIOMARKER USES
  • EVALUATION FRAMEWORKS
  • EVOLUTION OF REGULATORY PERSPECTIVES ON SURROGATE ENDPOINTS
  • BIOMARKERS AND COMMUNICATION STRATEGIES AT THE FDA
  • FURTHER ISSUES WITH USE OF BIOMARKERS
  • REFERENCES
• 3. The Biomarker Evaluation Process
  • THE RATIONALE FOR AN INTERRELATED, THREE-STEP PROCESS
  • APPLICATION OF THE EVALUATION FRAMEWORK
  • SCIENTIFIC PROCESS HARMONIZATION
  • CONCLUSION
  • REFERENCES
• 4. Case Studies
  • INTRODUCTION
  • TUMOR SIZE AS BIOMARKER FOR CANCER CLINICAL ENDPOINTS
  • C-REACTIVE PROTEIN
  • TROPONIN
  • LDL AND HDL AS BIOMARKERS FOR CARDIOVASCULAR RISK
  • BETA-CAROTENE
  • REFERENCES
• 5. Strengthening Evidence-Based Regulation
  • CHAPTER RECOMMENDATIONS
  • FDA REGULATORY AUTHORITY
  • FEDERAL AGENCIES AND DATA COLLECTION
  • TRACKING THE EFFECTS OF BIOMARKER USE AT THE FDA
  • REFERENCES
• Acronyms
• Glossary
• REFERENCES
• Appendix A Table of Papers About Biomarker Qualification
• Appendix B Recommendations from Related IOM Reports
• Appendix C Committee Member and Consultant Biographies
• Appendix D Staff Biographies
• Appendix E Workshop Agenda
• Appendix F Speaker Biographies

This study was supported by Contract No. HHSF223200810020I between the National Academy of Sciences and the Food and Drug Administration. Any opinions, findings, conclusions, or recommendations expressed in this publication are those of the author(s) and do not necessarily reflect the view of the organizations or agencies that provided support for this project.

Suggested citation:

IOM (Institute of Medicine). 2010. Evaluation of biomarkers and surrogate endpoints in chronic disease. Washington, DC: The National Academies Press.

NOTICE: The project that is the subject of this report was approved by the Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, and the Institute of Medicine. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance.