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.
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Table of Contents
AcknowledgementsPreface
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 Sturmer
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