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.
1. Chapter 1. Introduction - p. 1 2. Chapter 2. The efficacy-effectiveness gap - p. 9 3. Chapter 3. Studies for labeling versus reimbursement - p. 21 4. Chapter 4. Stakeholder engagement in the design and conduct of pragmatic randomized trials - p. 33 5. Chapter 5. Patient voice in clinical trial programs in industry - p. 47 6. Chapter 6. What is the research question? - p. 71 7. Chapter 7. Evaluating the feasibility of data sources for pragmatic clinical trials - p. 85 8. Chapter 8. Important design considerations in Pragmatic Randomized Clinical Trials - p. 99 9. Chapter 9. Randomization and masking – randomization at what unit? Masking of who and what? - p. 109 10. Chapter 10. Design and analysis of cluster randomized trials - p. 119 11. Chapter 11. Approaches to mitigate bias in the design and analysis of pRCTs - p. 131 12. Chapter 12. Sensitivity analyses in pragmatic randomized clinical trials - p. 151 13. Chapter 13. Methodology and reporting guidelines - p. 165 14. Chapter 14. Unmeasured confounding with and without randomization - p. 185 15. Chapter 15. Validation of health outcomes of interest in healthcare databases - p. 207 16. Chapter 16. Special topics in electronic health data: missing data and unstructured data - p. 219 17. Chapter 17. Distributed research networks and applications to pragmatic randomized trials - p. 237 18. Chapter 18. International and global issues – differences in health systems, patient populations, and medical practice - p. 257 19. Chapter 19. Considerations for protecting research participants - p. 273 20. Chapter 20. Replication and reproducibility - p. 293 21. Chapter 21. Dissemination of pragmatic randomized clinical trials information and results to patients and community stakeholders - p. 309 22. Chapter 22. Communicating results of pRCTs to the medical community - p. 329 23. Chapter 23. The PRIDE study: A post-marketing prospective pragmatic randomized clinical trial with regulatory approval - p. 347 24. Chapter 24. Special considerations on interventions: biologics - p. 361 25. Chapter 25. Special considerations of interventions: Medical devices - p. 387 26. Chapter 26. Special topic: Rare disease - p. 397 27. Chapter 27. Pragmatic randomized trials for behavioral health or educational interventions - p. 413 28. Chapter 28. Case studies: Examples from primary data collection - p. 427 29. Chapter 29. Examples from administrative claims data and electronic health records - p. 443 30. Chapter 30. Use of pragmatic clinical trials in reimbursement decisions - p. 455 31. Chapter 31. The future of clinical trials - p. 461 32. Index - p. 467
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.
1. Chapter 1. Introduction - p. 1 2. Chapter 2. The efficacy-effectiveness gap - p. 9 3. Chapter 3. Studies for labeling versus reimbursement - p. 21 4. Chapter 4. Stakeholder engagement in the design and conduct of pragmatic randomized trials - p. 33 5. Chapter 5. Patient voice in clinical trial programs in industry - p. 47 6. Chapter 6. What is the research question? - p. 71 7. Chapter 7. Evaluating the feasibility of data sources for pragmatic clinical trials - p. 85 8. Chapter 8. Important design considerations in Pragmatic Randomized Clinical Trials - p. 99 9. Chapter 9. Randomization and masking – randomization at what unit? Masking of who and what? - p. 109 10. Chapter 10. Design and analysis of cluster randomized trials - p. 119 11. Chapter 11. Approaches to mitigate bias in the design and analysis of pRCTs - p. 131 12. Chapter 12. Sensitivity analyses in pragmatic randomized clinical trials - p. 151 13. Chapter 13. Methodology and reporting guidelines - p. 165 14. Chapter 14. Unmeasured confounding with and without randomization - p. 185 15. Chapter 15. Validation of health outcomes of interest in healthcare databases - p. 207 16. Chapter 16. Special topics in electronic health data: missing data and unstructured data - p. 219 17. Chapter 17. Distributed research networks and applications to pragmatic randomized trials - p. 237 18. Chapter 18. International and global issues – differences in health systems, patient populations, and medical practice - p. 257 19. Chapter 19. Considerations for protecting research participants - p. 273 20. Chapter 20. Replication and reproducibility - p. 293 21. Chapter 21. Dissemination of pragmatic randomized clinical trials information and results to patients and community stakeholders - p. 309 22. Chapter 22. Communicating results of pRCTs to the medical community - p. 329 23. Chapter 23. The PRIDE study: A post-marketing prospective pragmatic randomized clinical trial with regulatory approval - p. 347 24. Chapter 24. Special considerations on interventions: biologics - p. 361 25. Chapter 25. Special considerations of interventions: Medical devices - p. 387 26. Chapter 26. Special topic: Rare disease - p. 397 27. Chapter 27. Pragmatic randomized trials for behavioral health or educational interventions - p. 413 28. Chapter 28. Case studies: Examples from primary data collection - p. 427 29. Chapter 29. Examples from administrative claims data and electronic health records - p. 443 30. Chapter 30. Use of pragmatic clinical trials in reimbursement decisions - p. 455 31. Chapter 31. The future of clinical trials - p. 461 32. Index - p. 467
