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Molecular Diagnostics for Melanoma pp 667–677Cite as

Statistical Design and Evaluation of Biomarker Studies

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Part of the book series: Methods in Molecular Biology ((MIMB,volume 1102))

Abstract

We review biostatistical aspects of biomarker studies, including design and analysis issues, covering the range of settings required for translational research—from early exploratory studies through clinical trials.

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References

  1. Rothberg BEG, Bracken MB, Rimm DL (2009) Tissue biomarkers for prognosis in cutaneous melanoma: a systematic review and meta-analysis. J Natl Cancer Inst 101:452–474

    Article  CAS  Google Scholar 

  2. Simon R (2010) Clinical trial designs evaluating the medical utility of prognostic and predictive biomarkers in oncology. Per Med 7:33–47

    Article  PubMed  Google Scholar 

  3. McShane LM, Altman DG, Sauerbrei W, Taube SE, Gion M, Clark GM (2005) Reporting recommendations for tumor marker prognostic studies (REMARK). J Natl Cancer Inst 97:1180–1184

    Article  PubMed  CAS  Google Scholar 

  4. Prentice RL (1989) Surrogate endpoints in clinical trials: discussion, definition and operational criteria. Stat Med 8(4):431–440

    Article  PubMed  CAS  Google Scholar 

  5. Friedman LM, Furberg CD, DeMets DL (2010) Fundamentals of clinical trials, 4th edn. Springer, New York

    Book  Google Scholar 

  6. Cook TD, DeMets DL (2008) Introduction to statistical methods for clinical trials. Chapman and Hall, New York

    Google Scholar 

  7. Fleming TR, DeMets DL (1996) Surrogate end points in clinical trials: are we being misled? Fleming TR and DeMets DL. Ann Intern Med 125(7):605–613

    Article  PubMed  CAS  Google Scholar 

  8. Buyse M, Sargent DJ, Grothey A, Matheson A, de Gramont A (2010) Biomarkers and surrogate end points: the challenge of statistical validation. Nat Rev Clin Oncol 7:309–317

    Article  PubMed  Google Scholar 

  9. Dobbin KK, Beer DG, Meyerson M, Yeatman TJ, Gerald WL, Jacobson JW, Conley B, Buetow KH, Heiskanen M, Simon RM, Minna JD, Girard L, Misek DE, Taylor JMG, Hanash S, Naoki K, Hayes DN, Ladd-Acosta C, Enkemann SA, Viale A, Giordano TJ (2005) Interlaboratory comparability study of cancer gene expression analysis using oligonucleotide microarrays. Clin Cancer Res 11:565–572

    PubMed  CAS  Google Scholar 

  10. Shoukri MM (2004) Measures of interobserver agreement. Chapman and Hall, Boca Raton

    Google Scholar 

  11. Simon RM, Paik S, Hayes DF (2009) Use of archived specimens to evaluate prognostic and predictive biomarkers. J Natl Cancer Inst USA 101(21):1446–1452

    Article  Google Scholar 

  12. Amado RG, Wolf M, Peeters M, Van Cutsem E, Siena S, Freeman DJ, Juan T, Sikorski R, Suggs S, Radinsky R, Patterson SD, Chang DD (2008) Wild-type KRAS is required for panitumumab efficacy in patients with metastatic colorectal cancer. J Clin Oncol 26:1626–1634

    Article  PubMed  CAS  Google Scholar 

  13. Karapetis CD, Khambata-Ford S, Jonker DJ, O’Callaghan CJ, Tu D, Tebbutt NC, Simes RJ, Chalchal H, Shapiro JD, Robitaille S, Price TJ, Shepherd L, Au HJ, Langer C, Moore MJ, Zalcberg JR (2008) K-RAS mutations and benefit from cetuximab in advanced colorectal cancer. N Engl J Med 359:1757–1765

    Article  PubMed  CAS  Google Scholar 

  14. Dancey JE, Dobbin KK, Groshen S, Jessup JM, Hruszkewicz AH, Koehler M, Parchment R, Ratain MJ, Shankar LK, Stadler WM, True LD, Gravell A, Grever MR, Biomarkers Task Force of the NCI Investigational Drug Steering Committee (2010) Guidelines for the development and incorporation of biomarker studies in early clinical trials of novel agents. Clin Cancer Res 16(6):1745–1755

    Article  PubMed  CAS  Google Scholar 

  15. McShane LM, Hunsberger S, Adjei AA (2009) Effective incorporation of biomarkers in phase II trials. Clin Cancer Res 15(6):1898–1905

    Article  PubMed  CAS  Google Scholar 

  16. Freidlin B, McShane LM, Korn EL (2009) Randomized clinical trials with biomarkers: design issues. J Natl Cancer Inst 102:152–160

    Article  Google Scholar 

  17. Vogel CL, Cobleigh MA, Tripathy D, Gutheil JC, Harris LN, Fehrenbacher L, Slamon DJ, Murphy M, Novotny WF, Burchmore M, Shak S, Stewart SJ, Press M (2002) Efficacy and safety of trastuzumumab as a single agent in first-line treatment of HER2-overexpressing metastatic breast cancer. J Clin Oncol 20(3):716–726

    Article  Google Scholar 

  18. Sparano JA (2006) TAILORx: trial assigning individualized options for treatment (Rx). Clin Breast Cancer 7(4):347–350

    Article  PubMed  Google Scholar 

  19. Cardoso F, Van’t Veer L, Rutgers E, Loi S, Mook S, Piccart-Gebhart MJ (2008) Clinical application of the 70-gene profile: the MINDACT trial. J Clin Oncol 26(5):729–735

    Article  PubMed  Google Scholar 

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Acknowledgment

This work was supported by the Distinguished Cancer Scholars grant program of the Georgia Research Alliance.

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Authors and Affiliations

  1. Department of Epidemiology and Biostatistics, College of Public Health, University of Georgia, Athens, GA, USA

    Kevin K. Dobbin

Authors
  1. Kevin K. Dobbin
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Editor information

Editors and Affiliations

  1. Cancer Diagnosis Program, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA

    Magdalena Thurin

  2. Sidra Medical and Research Center,Qatar Foundation, Doha, Qatar

    Francesco M. Marincola

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© 2014 Springer Science+Business Media, New York

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Dobbin, K.K. (2014). Statistical Design and Evaluation of Biomarker Studies. In: Thurin, M., Marincola, F. (eds) Molecular Diagnostics for Melanoma. Methods in Molecular Biology, vol 1102. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-62703-727-3_36

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  • DOI: https://doi.org/10.1007/978-1-62703-727-3_36

  • Published:

  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-62703-726-6

  • Online ISBN: 978-1-62703-727-3

  • eBook Packages: Springer Protocols

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