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Estimation and Hypothesis Testing

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Principles and Practice of Clinical Trials

Abstract

This chapter presents basic elements of parameter estimation and hypothesis testing. The reader will learn how to form confidence intervals for the mean, and more generally, how to calculate confidence intervals for the one parameter setting and for the difference between two groups. Principles of hypothesis testing are detailed, including the choice of the null and alternative hypotheses, the significance level, and implications for choosing a one-sided versus two-sided test. The p-value is defined and a discussion of controversies that have arisen over its use are included. After reading this chapter, the reader will have a better understanding of the necessary steps to set up a hypothesis test and make valid inference about the quantity of interest. Other topics in this chapter include exact hypothesis tests, which may be preferable for small sample settings, and the choice of a parametric versus nonparametric test. The chapter also includes a brief discussion of the implications of multiple comparisons on hypothesis testing and considerations of hypothesis testing in the setting of noninferiority trials.

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Author information

Authors and Affiliations

  1. University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA

    Pamela A. Shaw

  2. National Institute of Allergy and Infectious Diseases, Bethesda, MD, USA

    Michael A. Proschan

Authors
  1. Pamela A. Shaw
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  2. Michael A. Proschan
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Corresponding author

Correspondence to Pamela A. Shaw .

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. Department of Biostatistics and Bioinformatics, Basic Science Division, Duke University School of Medicine, Durham, NC, USA

    Stephen L. George

  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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Shaw, P.A., Proschan, M.A. (2020). Estimation and Hypothesis Testing. 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_114-1

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  • DOI: https://doi.org/10.1007/978-3-319-52677-5_114-1

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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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