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Design, Implementation, and Interpretation of Clinical Trials

  • Carol K. Redmond
  • Jong-Hyeon JeongEmail author
Chapter

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.

Keywords

Biased coin algorithm Competing risks CONSORT Data monitoring Efficacy Explanatory trial Futility Kaplan–Meier Log-rank Pragmatic trial Prediction models Proportional hazards model Randomization Safety Stopping rules Survival analysis Time-to-event 

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Copyright information

© Springer International Publishing Switzerland 2016

Authors and Affiliations

  1. 1.Department of BiostatisticsUniversity of PittsburghPittsburghUSA

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