37. The Use of Pharmacoepidemiology to Study Beneficial Drug Effects

  1. Brian L. Strom MD, MPH2,3,4,5,
  2. Stephen E. Kimmel MD, MSCE4,5 and
  3. Sean Hennessy PHARMD, PHD4,5
  1. Brian L. Strom MD, MPH2,3,4,5 and
  2. Kenneth L. Melmon1

Published Online: 3 JAN 2012

DOI: 10.1002/9781119959946.ch37

Pharmacoepidemiology, Fifth Edition

Pharmacoepidemiology, Fifth Edition

How to Cite

Strom, B. L. and Melmon, K. L. (2012) The Use of Pharmacoepidemiology to Study Beneficial Drug Effects, in Pharmacoepidemiology, Fifth Edition (eds B. L. Strom, S. E. Kimmel and S. Hennessy), Wiley-Blackwell, Oxford, UK. doi: 10.1002/9781119959946.ch37

Editor Information

  1. 2

    George S. Pepper Professor of Public Health and Preventive Medicine, Philadelphia, PA, USA

  2. 3

    Department of Biostatistics and Epidemiology, Philadelphia, PA, USA

  3. 4

    Center for Clinical Epidemiology and Biostatistics, Philadelphia, PA, USA

  4. 5

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

Author Information

  1. 1

    Stanford University School of Medicine, Stanford, CA, USA

  2. 2

    George S. Pepper Professor of Public Health and Preventive Medicine, Philadelphia, PA, USA

  3. 3

    Department of Biostatistics and Epidemiology, Philadelphia, PA, USA

  4. 4

    Center for Clinical Epidemiology and Biostatistics, Philadelphia, PA, USA

  5. 5

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

Publication History

  1. Published Online: 3 JAN 2012
  2. Published Print: 17 FEB 2012

ISBN Information

Print ISBN: 9780470654750

Online ISBN: 9781119959946

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Keywords:

  • beneficial drug effect;
  • confounding by indication;
  • drug effect;
  • effectiveness;
  • efficacy;
  • non-experimental study;
  • observational study;
  • premarketing study;
  • postmarketing study

Summary

In order to be approved for marketing in the United States, drugs must be proven to be safe and effective using “adequate and well-controlled investigations.” This chapter clarifies the different definitions of various types of beneficial drug effects and discusses the need for postmarketing studies of drug effectiveness. Unique methodologic problems raised by studies of beneficial drug effects, and potential solutions to these problems, are also discussed. Finally, specific examples of approaches to the study of efficacy are presented.