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Introduction to Pharmacoepidemiology and Its Application in Clinical Research

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Abstract

Pharmacoepidemiology, a sub-specialty of epidemiology involves the study of the use and effects (beneficial and safety) of drugs on large numbers of people using principles and methods of epidemiology and biostatistics. Originally, pharmacoepidemiology focused on the study of the adverse events associated with medication exposure. However, the field has evolved, and includes drug utilization studies such as studies to assess medication adherence, adherence to treatment guidelines, identifying diseased undertreated population as well as effectiveness assessments such as understanding the effectiveness in real-world clinical settings, dose-response relationships, and drug-drug interactions. Pharmacoepidemiology involves the application of study design methods and analytical techniques on large comprehensive databases to obtain measures of risk or effectiveness associated with drug therapies. Interpretation of these measures should be conducted in the context of the study design methodology and the limitations of the databases. In this chapter, I present a broad overview of pharmacoepidemiology methods, and the measures of risk or effectiveness associated with each study design method, highlighting the types of bias that can be introduced by conducting these analyses.

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Acknowledgments

I would like to thank the staff at the Division of Epidemiology, Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, FDA, for their contribution to the content of this chapter.

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Authors and Affiliations

  1. Division of Epidemiology, Office of Surveillance and Epidemiology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA

    Efe Eworuke

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  1. Efe Eworuke
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Corresponding author

Correspondence to Efe Eworuke .

Editor information

Editors and Affiliations

  1. Retired Senior Expert Pharmacologist at the Office of Cardiology, Hematology, Endocrinology, and Nephrology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA

    Gowraganahalli Jagadeesh

  2. Professor & Director, Research Training and Publications, The Office of Research and Development, Periyar Maniammai Institute of Science & Technology (Deemed to be University), Vallam, Tamil Nadu, India

    Pitchai Balakumar

  3. Division Cardiology & Nephrology, Office of Cardiology, Hematology, Endocrinology and Nephrology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA

    Fortunato Senatore

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This chapter reflects the views of the author and do not necessarily represent FDA’s views or policies.

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I have no conflict of interest to declare.

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© 2023 The Author(s), under exclusive license to Springer Nature Singapore Pte Ltd.

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Eworuke, E. (2023). Introduction to Pharmacoepidemiology and Its Application in Clinical Research. In: Jagadeesh, G., Balakumar, P., Senatore, F. (eds) The Quintessence of Basic and Clinical Research and Scientific Publishing. Springer, Singapore. https://doi.org/10.1007/978-981-99-1284-1_26

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