The Path from Biomarker Discovery to Regulatory Qualification

Edited by

  • Federico Goodsaid, PhD, Vice President, Strategic Regulatory Intelligence, Vertex Pharmaceuticals, Cambridge, MA
  • William Mattes, PhD, DABT, Senior Director of COPD Biomarkers Qualification Consortium, COPD Foundation and Founder and Principal, PharmPoint Consulting

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:

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


Book information

  • Published: August 2013
  • ISBN: 978-0-12-391496-5


"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

Table of Contents

Table of Contents

Section 1: Background

1. Biomarker Applications in the Pharmaceutical Industry - William Mattes, PharmaPoint

2. Fundamental Principles of Biomarker Qualification - Issam Zineh, CDER/FDA

3. The Impact of Biomarker Qualification Regulatory Processes on the Critical Path for Drug Development - Federico Goodsaid, Vertex

4. Regulatory Experience at the FDA, EMA, and PMDA - Shashi Amur, CDER/FDA, Spiros Vamvakas, EMA, and Yoshiaki Uyama, PMDA.

5. Biomarkers in the Co-Development of Drugs and Tests - Elizabeth Mansfield, CDRH/FDA

Section 2: Vignettes of Biomarker Development and Qualification

6. Biomarker Development and Qualification in the Pharmaceutical Industry -

Merck. John Wagner and Frank Sistare, Merck

Pfizer. Stephen Williams, Pfizer

Roche. Jackie Vonderscher, Roche

Novartis. Frank Dieterle, Novartis

Astra-Zeneca. Stephen Furlong, Astra-Zeneca

BMS. Bruce Car, BMS

Vertex. Martyn Botfield, Vertex

7. Toxicogenomic Biomarkers

Phase-1 Molecular Toxicology. Lawrence Kier

Incyte. Mary Jane Cunningham, Southern Institute

Gene Logic. Donna Mendrick, FDA

Johnson and Johnson. Peter Lord

ILSI-HESI. Cindy Afshari, Amgen

8. Pharmacogenomic Biomarkers

Warfarin. Lawrence Lesko, University of Florida

Clopidogrel. Michael Pacanowski, CDER/FDA

D-Met Chip. Richard Hockett, Affymetrix

9. Oncology Biomarkers

Herceptin. Eric Walk, Ventana

OncoDx. Kimberly Popovitz, Genomic Health

K-Ras. Scott Patterson, Amgen

CTCs. Howard Scher, Sloan-Kettering

10. Biomarkers of Drug Safety

Cardiac Safety: Troponins. Peter O’Brien, Dublin

Urinary Biomarkers of Kidney Injury. Joseph Bonventre, Harvard

Genetic Markers for Drug-Induced Liver Injury. Sally John, Pfizer/iSAEC

11. Consortia

ILSI-HESI. Michael Holsapple, ILSI-HESI

C-Path Institute. Ray Woosley, C-Path

FNIH. David Wholley, Biomarkers Consortium

iSAEC. Arthur Holden, iSAEC

Beyond QT. Jean-Phillipe Couderc, University of Rochester

Digestive System Motility. Michael Camilleri, Mayo Clinic

12: Tortuous Path to Regulatory Qualification Process Development

ECVAM. Thomas Hartung, Johns Hopkins

ICCVAM. Len Schechtman

FDA. Janet Woodcock, CDER/FDA

EMA. Eric Abadie and Marisa Papaluca-Amati, AFSSAPS/EMA

PMDA. Yoshiaki Uyama, PMDA

Section 3. Past, Present and Future of Biomarker Qualification - Federico Goodsaid, Vertex Pharmaceuticals.