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Randomized Controlled Trials 4: Biomarkers and Surrogate Outcomes

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Clinical Epidemiology

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1281))

Abstract

Biomarkers are defined as anatomic, physiologic, biochemical, molecular, or genetic parameters associated with the presence, absence, or severity of a disease process. As such, biomarkers may be useful as prognostic and diagnostic tests. Establishing the utility of a given biomarker as a prognostic or diagnostic test requires the conduct of carefully designed cohort studies in which the biomarker and the outcome of interest are measured independently. The design and analysis of such studies is discussed. Surrogate outcomes in clinical trials consist of events or biomarkers intended to reflect important clinical outcomes. Surrogate outcomes may offer advantages in providing statistically robust estimates of treatment effects with smaller sample sizes. However, to be useful, surrogate outcomes have to be validated to ensure that the effect of therapy on them truly reflects the effect of therapy on the important clinical outcomes of interest.

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Author information

Authors and Affiliations

  1. Seven Oaks General Hospital/SBGH 2PD02, University of Manitoba, 2300 McPhillips Street, Winnipeg, MB, Canada, R2V 3M3

    Claudio Rigatto

  2. Memorial University of Newfoundland, St. John’s, NL, Canada

    Brendan J. Barrett

Authors
  1. Claudio Rigatto
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  2. Brendan J. Barrett
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Corresponding author

Correspondence to Claudio Rigatto .

Editor information

Editors and Affiliations

  1. Memorial University of Newfoundland, St. John's, Newfoundland and Labrador, Canada

    Patrick S. Parfrey

  2. Memorial University of Newfoundland, St. John's, Newfoundland and Labrador, Canada

    Brendan J. Barrett

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© 2015 Springer Science+Business Media New York

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Rigatto, C., Barrett, B.J. (2015). Randomized Controlled Trials 4: Biomarkers and Surrogate Outcomes. In: Parfrey, P., Barrett, B. (eds) Clinical Epidemiology. Methods in Molecular Biology, vol 1281. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-2428-8_12

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  • DOI: https://doi.org/10.1007/978-1-4939-2428-8_12

  • Published:

  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-2427-1

  • Online ISBN: 978-1-4939-2428-8

  • eBook Packages: Springer Protocols

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