Pragmatic Randomized Clinical Trials

Pragmatic Randomized Clinical Trials

Using Primary Data Collection and Electronic Health Records

1st Edition - April 8, 2021

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  • Editors: Cynthia Girman, Mary Ritchey
  • Paperback ISBN: 9780128176634
  • eBook ISBN: 9780128176641

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Pragmatic Randomized Clinical Trials Using Primary Data Collection and Electronic Health Records addresses the practical aspects and challenges of the design, implementation, and dissemination of pragmatic randomized trials, also sometimes referred to as practical or hybrid randomized trials. While less restrictive and more generalizable than traditional randomized controlled trials, such trials have specific challenges which are addressed in this book. The book contains chapters encompassing common designs along with advantages and limitations of such designs, analytic aspects in planning trials and estimating sample size, and how to use patient partners to help design and operationalize pragmatic randomized trials. Pragmatic trials conducted using primary data collection and trials embedded in electronic health records - including electronic medical records and administrative insurance claims - are addressed.This comprehensive resource is valuable not only for pharmacoepidemiologists, biostatisticians and clinical researchers, but also across the biomedical field for those who are interested in applying pragmatic randomized clinical trials in their research.

Key Features

  • Addresses typical designs and challenges of pragmatic randomized clinical trials (pRCTs)
  • Encompasses analytic aspects of such trials
  • Discusses real cases on operational challenges in launching and conducting pRCTs in electronic health records


Biostatitians; medical researchers; epidemiologists; pharmaceutical researchers

Table of Contents

  • Acknowledgements
    Robert Califf and Joe Selby

    I: Introduction
    1. Introduction to pRCTs
    Cynthia J. Girman and Mary Elizabeth Ritchey
    2. The Efficacy–Effectiveness Gap
    Rolf H.H. Groenwold
    3. Studies for labeling vs reimbursement
    Robert Epstein

    II: Patient Voice and Stakeholder Feedback

    4. Stakeholder Engagement in pRCT Design and Conduct
    Thomas Concannon
    5. Patient Voice in Clinical Trial Programs in Industry
    Jeanne M. Regnante

    III: Design & Analysis
    6. What is the research question?
    Elizabeth A. Suarez and Soko Setoguchi
    7. Feasibility assessment for use of EHR
    Mary Elizabeth Ritchey
    8. Important design considerations in pRCTs
    Kevin Thorpe
    9. Randomization and Blinding – Randomization at what unit? Blinding of who and what?
    Jennifer Christian
    10. Cluster Randomized Trials
    Katherine Harris
    11. Design and Analytic Approaches to Minimize Bias and Confounding
    Michele Jonsson Funk
    12. Sensitivity analyses
    Elizabeth A. Suarez and Cynthia J. Girman
    13. Methodology and Reporting Guidelines
    Aisling Caffrey
    14. Unmeasured Confounding with and without randomization
    Phyo Than Htoo and Til Stűrmer

    IV: Operational Aspects
    15. Validation of Electronic Health Record and Patient-Reported Outcomes
    Vincent Lo Re III
    16. Special Considerations in EHR
    Leah McGrath and Jenna Wong
    17. Distributed Networks of Electronic Health Record Data
    Catherine A. Panozzo
    18. International and global issues – differences in medical practice
    Ju-Young (Judy) Shin, Kenneth Man and Wei Zhou

    V: Privacy and Ethics
    19. Considerations for Protecting Research Participants
    Aisling Caffrey

    VI: Interpretation, Limitations, and Strengths
    20. Replication and reproducibility
    Kristy Iglay and Mary Elizabeth Ritchey

    VII: Dissemination
    21. Communicating results to patients for shared decision-making
    Wendy Camelo Castillo
    22. Communicating Results of pRCTs to the Medical Community
    Joe Selby and Timothy Carey
    23. Communicating to regulatory agencies
    Larry D. Alphs

    VIII: Special Considerations on Interventions

    24. Biologics
    Jaclyn L. F. Bosco and Priscilla Velentgas
    25. Medical Devices
    Mary Elizabeth Ritchey
    26. Rare Diseases
    Cunlin Wang and Lina Titievsky
    27. Behavioral Interventions
    Andrea Troxel

    IX: Case Studies

    28. Examples from Primary Data Collection
    Kourtney J. Davis and Jeanne Marie Pimenta
    29. Examples from Electronic Health Records and Administrative Claims Data
    Ehab Hasan
    30. Use of Pragmatic Randomized Clinical Trials in Reimbursement Decisions
    Kati Copley-Merriman

    X: Conclusions

    31. Concluding remarks
    Cynthia J. Girman, Mary Elizabeth Ritchey and Elizabeth A. Suarez

Product details

  • No. of pages: 498
  • Language: English
  • Copyright: © Academic Press 2021
  • Published: April 8, 2021
  • Imprint: Academic Press
  • Paperback ISBN: 9780128176634
  • eBook ISBN: 9780128176641

About the Editors

Cynthia Girman

Cynthia Girman, DrPH, FISPE, is founder and President of CERobs Consulting, LLC, which provides consulting services to the pharmaceutical and healthcare industry on study design and scientific methods for real world observational studies, comparative effectiveness research and pragmatic randomized clinical trials. Dr. Girman is a seasoned epidemiologist with strong interest in biostatistical and observational methods, and the bulk of her 40+ year career has been in the pharmaceutical industry. Her undergraduate and doctoral training was at UNC and her MS in Applied Statistics and Computer Science is from Villanova University. She has led multi-institution academic collaborations, published extensively and reviewed for numerous peer-reviewed medical journals. As Adjunct Professor of Epidemiology at the University of North Carolina (UNC) Gillings School of Global Public Health, she collaborates on methodology research, gives guest classroom lectures and mentors numerous doctoral candidate graduate research assistants and post-doctoral fellows. Dr. Girman was an elected Board member 2015-2020 and is a fellow of the International Society for Pharmacoepidemiology (ISPE) and she annually teaches in the pragmatic randomized clinical trials (pRCT) pre-conference course. In September 2014, Dr. Girman was the first industry member nominated to the Patient-Centered Outcomes Research Institute (PCORI) Methodology Committee, on which she actively contributes along with their Clinical Trials Advisory Panel. Currently, Dr. Girman consults on study design, protocol development and analytic methods for real world evidence, such as registries, post-authorization safety studies, natural history studies, comparative effectiveness research, pragmatic randomized clinical trials, and external control groups for rare diseases. She also consults on endpoint strategies for randomized clinical trials, including patient reported outcomes, clinician assessments and expert review panels, as well as patient engagement and qualitative research.

Affiliations and Expertise

Founder and President; Real World Evidence, Pharmacoepidemiology and Patient Outcomes; CERobs Consulting, LLC; Wrightsville Beach, NC USA

Mary Ritchey

Mary E Ritchey, PhD, FISPE is Principal and Owner, Med Tech Epi, LLC a consulting group which provides real-world evidence strategy development, study design and implementation for medical devices, biotech, and medicines. She is an Associate Research Professor in the Center for Pharmacoepidemiology and Treatment Sciences at Rutgers University and a Senior Executive Pharmacoepidemiologist for CERobs Consulting, LLC. Dr. Ritchey obtained her masters and doctorate degrees in epidemiology from University of North Carolina Gillings School of Global Public Health. She is a Fellow and board member (2019-2022) of the International Society of Pharmacoepidemiology (ISPE), a member of the Medical Device Epidemiology (MDEpiNet) Scientific Oversight Committee, and Associate Editor for Pharmacoepidemiology and Drug Safety. Her career spans more than 15 years in government, industry, consulting, and academia focused on better understanding medical product safety and effectiveness when used in actual clinical practice. She is adept with scientific, technical, and logistical aspects of feasibility, utilization, safety, and effectiveness real-world studies for postmarketing requirements, labeling expansion, clinical trial support studies, and pragmatic clinical trials. She enjoys engaging with clinical and research teams to gather knowledge, generate evidence, inform decision-makers, and communicate meaningful results to regulators, payers, clinicians, and patients.

Affiliations and Expertise

Principal and Owner, Med Tech Epi, LLC, Philadelphia, PA USA; Associate Research Professor, Center for Pharmacoepidemiology and Treatment Sciences, Rutgers University, New Brunswick, NJ USA; Senior Executive Pharmacoepidemiologist, Real World Evidence, Pharmacoepidemiology and Patient Outcomes, CERobs Consulting, LLC; Wrightsville Beach, NC USAPrincipal and Owner, Med Tech Epi, LLC, Philadelphia, PA USA; Associate Research Professor, Center for Pharmacoepidemiology and Treatment Sciences, Rutgers University, New Brunswick, NJ USA; Senior Executive Pharmacoepidemiologist, Real World Evidence, Pharmacoepidemiology and Patient Outcomes, CERobs Consulting, LLC; Wrightsville Beach, NC USA

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