The Path from Biomarker Discovery to Regulatory Qualification

The Path from Biomarker Discovery to Regulatory Qualification

1st Edition - July 16, 2013

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  • Editors: Federico Goodsaid, William Mattes
  • Paperback ISBN: 9780123914965
  • eBook ISBN: 9780123944085

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Description

The Path from Biomarker Discovery to Regulatory Qualification is a unique guide that focuses on biomarker qualification, its history and current regulatory settings in both the US and abroad. This multi-contributed book provides a detailed look at the next step to developing biomarkers for clinical use and covers overall concepts, challenges, strategies and solutions based on the experiences of regulatory authorities and scientists. Members of the regulatory, pharmaceutical and biomarker development communities will benefit the most from using this book—it is a complete and practical guide to biomarker qualification, providing valuable insight to an ever-evolving and important area of regulatory science.   For complimentary access to chapter 13, 'Classic' Biomarkers of Liver Injury, by John R. Senior, Associate Director for Science, Food and Drug Administration, Silver Spring, Maryland, USA, please visit the following site:  http://tinyurl.com/ClassicBiomarkers

Key Features

  • Contains a collection of experiences of different groups taking different types of biomarkers to different levels of qualification and provides insightful case studies of an important area of regulatory science
  • Focuses on practical advice, concepts, strategies and overall outcomes to support those working toward biomarker qualification for clinical use
  • Offers a valuable resource for members of the regulatory, pharmaceutical and biomarker development communities

Readership

The primary audience includes pharmaceutical scientists and pharmacologists, clinicians and drug development managers and executives involved in pharmaceutical development.

Table of Contents

  • Section 1: Introduction

    1. Biomarker Applications in the Pharmaceutical Industry

    Diagnostic Applications

    Prognostic Applications

    Intervention Management/Monitoring

    Concluding Remarks

    References

    2. Impact of Biomarker Qualification Regulatory Processes on the Critical Path for Drug Development

    Introduction

    Has the Process at the FDA (CDER) Achieved the Goals Originally Proposed by the Pharmacogenomics Guidance in 2005 and the Critical Path Opportunities List and Report in 2006?

    What are the Strengths and Weaknesses of the Different Versions of a Biomarker Qualification Process Developed in Each ICH Region?

    How Well Have the Different Versions of This Process Been Harmonized across These Regions?

    What are the Opportunities and Challenges for a Single, Universal, Biomarker Qualification Process?

    How Can We Develop Metrics with Which to Measure the Impact of These Processes on Drug Development and Their Acceptance by the Pharmaceutical Industry?

    Summary

    References

    3. Regulatory Experience of Biomarker Qualification in the EMA

    Other Informal Interactions with the EMA

    Examples of Qualification Opinions

    4. Regulatory Experience at the FDA, EMA, and PMDA: Regulatory Experience at the PMDA

    Acknowledgment

    References

    Section 2: Biomarker Development and Qualification in the Pharmaceutical Industry

    5. The Impact of Changing Context of Use on Weight of Evidence for Qualification of Biomarkers: A Case Study in Ischemic Stroke

    6. Safety Biomarker Development and Qualification in the Pharmaceutical Industry

    7. First-Ever Regulatory Biomarker Qualification – Review and Insights by a Participant

    References

    8. Metabolomics-Derived Biomarkers of Drug-Induced Skeletal Muscle Injury and Urinary Bladder Transitional Cell Carcinoma in Rats

    Metabolomic Biomarker for Drug-Induced Skeletal Muscle Injury in Rats

    Metabolomic Biomarker for Causation of Bladder Tumorigenesis in Rats

    Summary

    References

    9. Molecular Biomarkers for Patients with Castration-Resistant Prostate Cancer: Validating Assays Predictive of Tumor Response

    Unmet Needs to be Addressed with Biomarkers

    FDA-Approved Method and Demonstrated Use

    Emerging Molecular Biomarkers

    Validating Genomic Biomarkers in CTCs

    Molecular Biomarkers in a CLIA-Certified Laboratory

    Changing the Paradigm for Assessment of Recurrent Prostate Cancer: a Liquid Biopsy

    References

    Section 3: Toxicogenomic Biomarkers

    10. Gene Logic and Toxicogenomics Biomarkers

    References

    11. The When, Where and How of Toxicogenomic Submissions to Regulatory Agencies: The Deliberations of the HL7/CDISC/I3C Pharmacogenomics Data Standards Track 1 Toxicogenomics Working Group

    Acknowledgments

    Introduction and Background

    Toxicogenomic Applications in Drug Discovery and Development

    The Case Examples

    A Hypothetical Submission in Support of the Safety Claims for a Hypolipidemic Drug

    Highlights from the Discussions

    Conclusions

    References

    12. Biomarker Qualification – Past, Present and Future: Initiating a Cross-Sector Toxicogenomics Biomarker Initiative – Health and Environmental Sciences Institute (HESI) Committee on Application of Genomics to Mechanism-Based Risk Assessment

    HESI’s Contributions to the Emerging Field of Toxicogenomics

    Use of Toxicogenomics to Biological Assess Mechanisms – Case Studies

    Current Focus and Activities of the HESI Genomics Committee

    Summary and Future Horizons

    References

    Section 4: Biomarkers of Drug Safety

    13. ‘Classic’ Biomarkers of Liver Injury

    Bilirubin

    Alternative and Discarded Biomarkers

    Aminotransferases (Transaminases)

    Combined ALT and TBL Measurements

    Conclusions and Recommendations

    References

    14. Qualification of Urinary Biomarkers for Kidney Toxicity

    Regulatory Framework: Advancing the Science of Biomarkers

    Kidney Injury Molecule-1: Characteristics

    Collaborations in Consortia

    Interactions with the FDA and EMA

    Translation to Clinical Use

    Conclusions

    References

    Section 5: Consortia

    15. Renal Biomarker Qualification: An ILSI Health and Environmental Sciences Institute Perspective

    Safety Biomarker Research – the Origin of this Activity in HESI

    Creation of Biomarker Working Groups

    Biomarkers of Nephrotoxicity Committee (BNC)

    Biomarker Qualification: the Changing Landscape

    References

    16. Vignette Regarding Consortia: C-Path Institute

    17. The Telemetric and Holter ECG Warehouse to Enable the Validation and Development of Novel Electrocardiographic Markers

    Introduction

    QT Interval as a Safety Marker in Drug Development

    QT Interval: from a Biomarker to a Surrogate Marker

    The Thorough QT Studies (TQT)

    Improving the QT Marker

    QT, Heart Rate and Autonomic Regulation

    Inception of the Telemetric and Holter ECG Warehouse (THEW): a Model to Develop Academia-Regulators-Industries Partnerships

    Involving the FDA and Industry

    Participation by the National Institutes of Health

    Facilitating the Submission of New ECG Markers to the FDA

    Conclusions and Perspectives

    References

    Section VI: Path to Regulatory Qualification Process Development

    18. Path to Regulatory Qualification Program Development: A US FDA Perspective

    Acknowledgments

    The Biomarker Qualification Process at US FDA

    Current Status of Biomarker Qualification Submissions

    Future Perspectives

    References

    19. Path to Regulatory Qualification Process Development

    Acknowledgment

    References

    20. The Tortuous Path From Development to Qualification of Biomarkers

Product details

  • No. of pages: 206
  • Language: English
  • Copyright: © Academic Press 2013
  • Published: July 16, 2013
  • Imprint: Academic Press
  • Paperback ISBN: 9780123914965
  • eBook ISBN: 9780123944085

About the Editors

Federico Goodsaid

PhD, Vice President, Strategic Regulatory Intelligence, Vertex Pharmaceuticals, Cambridge, MA

Affiliations and Expertise

PhD, Vice President, Strategic Regulatory Intelligence, Vertex Pharmaceuticals, Cambridge, MA

William Mattes

William B. Mattes, PhD, DABT, Senior Director of COPD Biomarkers Qualification Consortium, COPD Foundation and Founder and Principal, PharmPoint Consulting

Affiliations and Expertise

PhD, DABT, Senior Director of COPD Biomarkers Qualification Consortium, COPD Foundation and Founder and Principal, PharmPoint Consulting

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