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Contemporary Biostatistics with Biopharmaceutical Applications pp 285–298Cite as

Review of Statistical Issues in Pragmatic Clinical Trials in Current Drug Development Environment

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Part of the book series: ICSA Book Series in Statistics ((ICSABSS))

Abstract

Facing increasing resource pressure, stakeholders in healthcare are seeking real-world evidence (RWE) of effectiveness and safety for informed decision-making. Increasing attention is being drawn to pragmatic clinical trials (PCTs) for their emerging roles in generating high-quality RWE. Principles of PCTs challenge some well-established guidelines in randomized clinical trials (RCTs), as open-label and treatment switch in intention-to-treat (ITT) population being the most pronounced ones. This review discusses key methodological and statistical implications of PCTs in the context of drug development and reimbursement, with emphasis on study design and analyses to maximize external validity. Many of those aspects are very challenging and are expected to crystallize over time after more research is done.

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Author information

Authors and Affiliations

  1. AbbVie Inc., North Chicago, IL, USA

    Dingfeng Jiang, Kun Chen, Saurabh Mukhopadhyay & Nareen Katta

  2. Data and Statistical Sciences, AbbVie Inc., North Chicago, IL, USA

    Lanju Zhang

Authors
  1. Dingfeng Jiang
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  2. Kun Chen
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  3. Saurabh Mukhopadhyay
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  4. Nareen Katta
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  5. Lanju Zhang
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Corresponding author

Correspondence to Dingfeng Jiang .

Editor information

Editors and Affiliations

  1. Data and Statistical Sciences, AbbVie Inc., North Chicago, IL, USA

    Lanju Zhang

  2. School of Social Work, University of North Carolina, Chapel Hill, NC, USA

    Ding-Geng (Din) Chen

  3. Department of Statistics, Northwestern University, Evanston, IL, USA

    Hongmei Jiang

  4. R&D, Janssen Pharmaceuticals, Raritan, NJ, USA

    Gang Li

  5. Sanofi US, Bridgewater, NJ, USA

    Hui Quan

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Jiang, D., Chen, K., Mukhopadhyay, S., Katta, N., Zhang, L. (2019). Review of Statistical Issues in Pragmatic Clinical Trials in Current Drug Development Environment. In: Zhang, L., Chen, DG., Jiang, H., Li, G., Quan, H. (eds) Contemporary Biostatistics with Biopharmaceutical Applications. ICSA Book Series in Statistics. Springer, Cham. https://doi.org/10.1007/978-3-030-15310-6_16

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