Pragmatic Trials and Real World Evidence


The early (possible pre-launch) implementation of pragmatic trials to investigate the effectiveness of medications in the real world can raise many different operational challenges than traditional, more explanatory trials. In this series of papers we address how to balance the methodology of pragmatic trials with overcoming these operational challenges, and give an overview of trial characteristics to consider when designing and executing a pragmatic trial. We aim to help pragmatic trial designers to be aware of consequences of their choices and maximize the pragmatic nature of the study design while ensuring operational feasibility. In this series, we integrate the theoretical design options with the practice of conducting pragmatic trials. The series starts with an introductory paper on the concept of pragmatic trials, their value, what pragmatic trials could look like in a perfect world and where challenges arise in practice. Each subsequent paper focusses on a specific trial area such as patient selection and recruitment or the choice of comparator. For each of these areas we discuss the pragmatic design choices; their specific operational challenges and their impact on operational feasibility and (ethical) acceptability of the trial; as well as generalizability, validity, and precision of the trial findings.
Contains articles from 11 May 2017 to 01 October 2017

  • Series: Pragmatic trials and real world evidence: Paper 1. Introduction

    Mira G.P. Zuidgeest, Iris Goetz, Rolf H.H. Groenwold, Elaine Irving, Ghislaine J.M.W. van Thiel, Diederick E. Grobbee On behalf of GetReal Work Package 3
    Vol. 88
    Published online: May 23, 2017
    Open Access
  • Series: Pragmatic trials and real world evidence: Paper 2. Setting, sites, and investigator selection

    Sally D. Worsley, Katrien Oude Rengerink, Elaine Irving, Stephane Lejeune, Koen Mol, Sue Collier, Rolf H.H. Groenwold, Catherine Enters-Weijnen, Matthias Egger, Thomas Rhodes On behalf of GetReal Work Package 3
    Vol. 88
    Published online: May 11, 2017
    Open Access
  • Series: Pragmatic trials and real world evidence: Paper 3. Patient selection challenges and consequences

    Katrien Oude Rengerink, Shona Kalkman, Susan Collier, Antonio Ciaglia, Sally D. Worsley, Alison Lightbourne, Laurent Eckert, Rolf H.H. Groenwold, Diederick E. Grobbee, Elaine A. Irving on behalf of Work Package 3 of the GetReal consortium
    Vol. 89
    Published online: May 11, 2017
    Open Access
  • Series: Pragmatic trials and real world evidence: Paper 4. Informed consent

    Shona Kalkman, Ghislaine J.M.W. van Thiel, Mira G.P. Zuidgeest, Iris Goetz, Boris M. Pfeiffer, Diederick E. Grobbee, Johannes J.M. van Delden on behalf of Work Package 3 of the IMI GetReal Consortium
    Vol. 89
    Published online: May 11, 2017
    Open Access
  • Series: Pragmatic trials and real world evidence: Paper 5. Usual care and real life comparators

    Mira G.P. Zuidgeest, Paco M.J. Welsing, Ghislaine J.M.W. van Thiel, Antonio Ciaglia, Rafael Alfonso-Cristancho, Laurent Eckert, Marinus J.C. Eijkemans, Matthias Egger on behalf of WP3 of the GetReal consortium
    Vol. 90
    Published online: July 7, 2017
    Open Access
  • Series: Pragmatic trials and real world evidence: Paper 6. Outcome measures in the real world

    Paco M. Welsing, Katrien Oude Rengerink, Sue Collier, Laurent Eckert, Maarten van Smeden, Antonio Ciaglia, Gaelle Nachbaur, Sven Trelle, Aliki J. Taylor, Matthias Egger, Iris Goetz on behalf of Work Package 3 of the GetReal Consortium
    Vol. 90
    Published online: May 11, 2017
    Open Access
  • Series: Pragmatic trials and real world evidence: Paper 7. Safety, quality and monitoring

    Elaine Irving, Rutger van den Bor, Paco Welsing, Veronica Walsh, Rafael Alfonso-Cristancho, Catherine Harvey, Nadia Garman, Diederick E. Grobbee on behalf of GetReal Work Package 3
    Vol. 91
    Published online: May 11, 2017
    Open Access
  • Series: Pragmatic trials and real world evidence: Paper 8. Data collection and management

    Anna-Katharina Meinecke, Paco Welsing, George Kafatos, Des Burke, Sven Trelle, Maria Kubin, Gaelle Nachbaur, Matthias Egger, Mira Zuidgeest on behalf of work package 3 of the GetReal consortium
    Vol. 91
    Published online: July 14, 2017
    Open Access
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