The Path from Biomarker Discovery to Regulatory Qualification - 1st Edition - ISBN: 9780123914965, 9780123944085

The Path from Biomarker Discovery to Regulatory Qualification

1st Edition

Editors: Federico Goodsaid William Mattes
eBook ISBN: 9780123944085
Paperback ISBN: 9780123914965
Imprint: Academic Press
Published Date: 7th August 2013
Page Count: 206
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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:

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.


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


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


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?



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



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


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



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


Section 3: Toxicogenomic Biomarkers

10. Gene Logic and Toxicogenomics Biomarkers


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


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



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


Section 4: Biomarkers of Drug Safety

13. ‘Classic’ Biomarkers of Liver Injury


Alternative and Discarded Biomarkers

Aminotransferases (Transaminases)

Combined ALT and TBL Measurements

Conclusions and Recommendations


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



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


16. Vignette Regarding Consortia: C-Path Institute

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


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


Section VI: Path to Regulatory Qualification Process Development

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


The Biomarker Qualification Process at US FDA

Current Status of Biomarker Qualification Submissions

Future Perspectives


19. Path to Regulatory Qualification Process Development



20. The Tortuous Path From Development to Qualification of Biomarkers



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© Academic Press 2013
Academic Press
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About the Editor

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


"This book focuses on the use of biomarkers in drug development, for efficacy and targeted drug therapy as well as for predicting drug harm. It is a collection of papers that present the authors' examples of their work using biomarkers, not only for drug development but also for regulatory review.", January 3, 2014***
"Scientists from academia, drug companies, and regulatory agencies explore ways to develop biomarkers and integrate them in both developing and regulating drugs. They cover the impact of biomarker qualification regulatory processes on the critical path for drug development, biomarker development and qualification in the pharmaceutical industry, toxicogenomic biomarkers, biomarkers of drug safety, consortia, and the path to regulatory qualification process development."--Reference & Research Book News, December 2013

Ratings and Reviews