Pharmaceutical Research

, Volume 22, Issue 4, pp 499–511 | Cite as

Method Validation and Measurement of Biomarkers in Nonclinical and Clinical Samples in Drug Development: A Conference Report

  • Jean W. LeeEmail author
  • Russ S. Weiner
  • Jeff M. Sailstad
  • Ronald R. Bowsher
  • Dean W. Knuth
  • Peter J. O’Brien
  • Jean L. Fourcroy
  • Rakesh Dixit
  • Lini Pandite
  • Robert G. Pietrusko
  • Holly D. Soares
  • Valerie Quarmby
  • Ole L. Vesterqvist
  • David M. Potter
  • James L. Witliff
  • Herbert A. Fritche
  • Timothy O’Leary
  • Lorah Perlee
  • Sunil Kadam
  • John A. Wagner
Conference Report

No Heading

Biomarkers are increasingly used in drug development to aid scientific and clinical decisions regarding the progress of candidate and marketed therapeutics. Biomarkers can improve the understanding of diseases as well as therapeutic and off-target effects of drugs. Early implementation of biomarker strategies thus promises to reduce costs and time-to-market as drugs proceed through increasingly costly and complex clinical development programs. The 2003 American Association of Pharmaceutical Sciences/Clinical Ligand Assay Society Biomarkers Workshop (Salt Lake City, UT, USA, October 24–25, 2003) addressed key issues in biomarker research, with an emphasis on the validation and implementation of biochemical biomarker assays, covering from preclinical discovery of efficacy and toxicity biomarkers through clinical and postmarketing implementation. This summary report of the workshop focuses on the major issues discussed during presentations and open forums and noted consensus achieved among the participants on topics from nomenclature to best practices. For example, it was agreed that because reliable and accurate data provide the basis for sound decision making, biomarker assays must be validated in a manner that enables the creation of such data. The nature of biomarker measurements often precludes direct application of regulatory guidelines established for clinical diagnostics or drug bioanalysis, and future guidance on biomarker assay validation should therefore be adaptable enough that validation criteria do not stifle creative biomarker solutions.

Key Words:

biomarkers nonclinical and clinical drug development quantitative method development and validation 



American Association of Pharmaceutical Sciences


below quantifiable limit


Center for Drug Evaluation and Research


Centers for Medicare & Medicaid Services


Clinical Ligand Assay Society


Clinical Lab Improvement Amendments


good laboratory practices


Ligand Binding Assay Bioanalytical Focus Group


lower limit of detection


lower limit of quantification


National Committee for Clinical Laboratory Standards






Office of in Vitro Diagnostics Device Evaluation and Safety


quality controls


upper limit of quantification


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Copyright information

© Springer Science+Business Media, Inc. 2005

Authors and Affiliations

  • Jean W. Lee
    • 1
    Email author
  • Russ S. Weiner
    • 2
  • Jeff M. Sailstad
    • 3
  • Ronald R. Bowsher
    • 4
  • Dean W. Knuth
    • 5
  • Peter J. O’Brien
    • 6
  • Jean L. Fourcroy
    • 7
  • Rakesh Dixit
    • 8
  • Lini Pandite
    • 9
  • Robert G. Pietrusko
    • 10
  • Holly D. Soares
    • 11
  • Valerie Quarmby
    • 12
  • Ole L. Vesterqvist
    • 2
  • David M. Potter
    • 11
  • James L. Witliff
    • 13
  • Herbert A. Fritche
    • 14
  • Timothy O’Leary
    • 15
  • Lorah Perlee
    • 16
  • Sunil Kadam
    • 6
  • John A. Wagner
    • 8
  1. 1.MDS Pharma ServicesLincolnUSA
  2. 2.Bristol-Myers SquibbPrincetonUSA
  3. 3.Trimeris Inc.DurhamUSA
  4. 4.LINCO Diagnostic ServicesSt. CharlesUSA
  5. 5.Jasper ClinicKalamazooUSA
  6. 6.Eli Lilly and CompanyIndianapolisUSA
  7. 7.Walter Reed Army HospitalBethesdaUSA
  8. 8.Merck and Company, Inc.West PointUSA
  9. 9.GlaxoSmithKlineResearch Triangle ParkUSA
  10. 10.Millenium PharmaceuticalsCambridgeUSA
  11. 11.Pfizer Global ResearchGrotonUSA
  12. 12.Genetech, Inc.South San FranciscoUSA
  13. 13.University of LouisvilleLouisvilleUSA
  14. 14.MD Anderson HospitalUniversity of TexasAustinUSA
  15. 15.Food and Drug AdministrationRockvilleUSA
  16. 16.Molecular Staging Inc.New HavenUSA

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