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Design, Implementation, and Interpretation of Clinical Trials

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Management of Breast Diseases

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Abstract

This chapter begins with a review of highlights in the evolution of clinical trials, which originates from the Old Testament of the Bible. Important features of clinical trials including various phases of clinical trials and the difference between explanatory and pragmatic approaches are reviewed. Aspects of statistical design are considered including choice of control group, randomization, and sample size and power based on various types of study outcomes. An example of using the biased coin algorithm for randomization, controlling for important prognostic factors, is illustrated, and ethical considerations arising in the randomization process are discussed. Aspects of data and safety monitoring including stopping rules due to efficacy or futility are reviewed. Popular statistical tools are presented including Kaplan–Meier estimator, log-rank test statistic, Cox’s proportional hazards model, competing risks analysis, and building and validating prediction models. Finally, guidelines for reporting and publishing the results from clinical trials are reviewed.

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Authors and Affiliations

  1. Department of Biostatistics, University of Pittsburgh, 310 Parran Hall, 130 DeSoto Street, Pittsburgh, PA, 15261, USA

    Carol K. Redmond & Jong-Hyeon Jeong

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  1. Carol K. Redmond
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  2. Jong-Hyeon Jeong
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Correspondence to Jong-Hyeon Jeong .

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  1. Department of Surgery, University of Texas Health Science Center, San Antonio, Texas, USA

    Ismail Jatoi

  2. Obstetrics and Gynecology, University Medical Center Schleswig-Holstein, Luebeck, Germany

    Achim Rody

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Redmond, C.K., Jeong, JH. (2016). Design, Implementation, and Interpretation of Clinical Trials. In: Jatoi, I., Rody, A. (eds) Management of Breast Diseases. Springer, Cham. https://doi.org/10.1007/978-3-319-46356-8_33

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