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Inferential Frameworks for Clinical Trials

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Abstract

Statistical inference is the process of using data to draw conclusions about unknown quantities. Statistical inference plays a large role both in designing clinical trials and in analyzing the resulting data. The two main schools of inference, frequentist and Bayesian, differ in how they estimate and quantify uncertainty in unknown quantities. Typically, Bayesian methods have clearer interpretation at the cost of specifying additional assumptions about the unknown quantities. This chapter reviews the philosophy behind these two frameworks including concepts such as p-values, Type I and Type II errors, confidence intervals, credible intervals, prior distributions, posterior distributions, and Bayes factors. Application of these ideas to various clinical trial designs including 3 + 3, Simon’s two-stage, interim safety and efficacy monitoring, basket, umbrella, and platform drug trials is discussed. Recent developments in computing power and statistical software now enable wide access to many novel trial designs with operating characteristics superior to classical methods.

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Author information

Authors and Affiliations

  1. Department of Biostatistics, University of Texas MD Anderson Cancer Center, Houston, TX, USA

    James P. Long

  2. Department of Biostatistics, Associate Vice President, Quantitative Sciences, University of Texas MD Anderson Cancer Center, Houston, TX, USA

    J. Jack Lee

Authors
  1. James P. Long
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  2. J. Jack Lee
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Corresponding author

Correspondence to J. Jack Lee .

Editor information

Editors and Affiliations

  1. Samuel Oschin Comprehensive Cancer Insti, WEST HOLLYWOOD, CA, USA

    Steven Piantadosi

  2. Bloomberg School of Public Health, Johns Hopkins Center for Clinical Trials Bloomberg School of Public Health, Baltimore, MD, USA

    Curtis L. Meinert

Section Editor information

  1. University of Iowa, Iowa, IA, USA

    Christopher S Coffey Ph.D

  2. The Johns Hopkins Center for Clinical Trials and Evidence Synthesis, Johns Hopkins University, Baltimore, MD, USA

    Curtis L. Meinert

  3. Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA

    Steven Piantadosi

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Long, J.P., Lee, J.J. (2021). Inferential Frameworks for Clinical Trials. In: Piantadosi, S., Meinert, C.L. (eds) Principles and Practice of Clinical Trials. Springer, Cham. https://doi.org/10.1007/978-3-319-52677-5_271-1

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  • DOI: https://doi.org/10.1007/978-3-319-52677-5_271-1

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  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-319-52677-5

  • Online ISBN: 978-3-319-52677-5

  • eBook Packages: Springer Reference MathematicsReference Module Computer Science and Engineering

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