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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. The book contains chapters encompassing common designs, along with the 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 such 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 biostatitians, but also for several members of biomedical field who are interested in applying pragmatic randomized clinical trials in their research.
- Brings typical designs and challenges of pragmatic randomized clinical trials (pRCTs)
- Encompasses analytic aspects for sample size determination of such trials
- Discusses real cases on operational challenges in launching and conducting pRCTs in electronic health records
Biostatitians; medical researchers; epidemiologists; pharmaceutical researchers
1. Introduction to pRCTs
2. The Efficacy - Effectiveness Gap
3. Studies for labeling vs reimbursement
4. Special considerations EHR and claims, or linked data (Intro)
II Patient Voice and Stakeholder Feedback
5. Formulating research question, Study Design and Methods
6. Operational (recruitment, retention, burden on patients)
8. Stakeholder Engagement Methods (Patients and Other)
9. Patient Voice in Industry
III Design and Analysis
10. What is the research question? Include issues of analysis population
11. Feasibility assessment for use of EHR
12. pRCT typical design
13. Randomization / Blinding – Randomization at what unit? Blinding of who / what?
14. Cluster Randomized Trials
15. Design and Analytic Approaches to Minimize Bias and Confounding
16. Considerations for Primary Data Collection
17. Sensitivity analyses
18. Anticipating/identifying/quantifying potential bias
19. Reporting - Consort pRCT, Precis, GRADE, Strobe ISPOR etc. ICPE/ISPOR on transparency, PCORI Methodology Standards
20. Unmeasured Confounding with randomization – Does it matter?
21. Analytic Issues and Data Analysis
IV Operational Aspects
22. Importance of blinding during eligibility assessment and enrollment
23. Validation of Outcomes
23. Computational Phenotype in practice
24. Prospective Follow-up? –primary data collection vs EHR
25. Special Considerations in EHR (including pharmacy data, EMR and claims vs unstructured data)
26. Networks of Electronic Health Data – distributed vs centralized
27. International / global issues – differences in medical practice
V Privacy and Ethics
28. Patient identifying information and protection, IRB issues
29. Clinicaltrials.gov, ENCePP
VI Interpretation, Limitations, and Strength
30. Clinical meaningfulness
31. Considering results with the totality of evidence
32. Potential for confounding, even with randomization (selection bias)
33. Missing/implausible data, value/relational conformance issues: missing encounters, elements (diagnosis, procedure), implausible values, lack of conformance to data model and to other health data
34. Replication and reproducibility
35. Communicating results to patients for shared decision-making
36. Communicating to medical community
37. Communicating to regulatory agencies
38. Communicating to reimbursement agencies
VIII Special Considerations on Interventions
41. Rare Diseases
42. Behavioral Intervention
43. Communication Intervention
IX Case Studies (This section will consist of brief case studies, 1-3 cases per area of interest. An introduction to the section will be provided by the Editor.)
44. Examples from Primary Data Collection
45. Examples from Administrative Claims Data
46. Examples from Electronic Health Records
47. Examples of pRCT for use by regulatory agencies
48. Examples of pRCT for reimbursement
49. Concluding remarks
- No. of pages:
- © Academic Press 2021
- 1st April 2021
- Academic Press
- Paperback ISBN:
Cynthia J. Girman is President and sole proprietor of CERobs Consulting, LLC, which provides consulting on study design and methodology for comparative effectiveness research and real-world randomized pragmatic clinical trials as well as the development and validation of patient reported outcomes. Previously, Dr. Girman was Executive Director and Head of Data Analytics & Observational Methods in the Center for Observational & Real World Evidence at Merck Research Laboratories, from which she retired after 33 years. She founded the Merck Center of Excellence on the development and validation of endpoints for clinical trials, including patient reported outcomes, and her work has focused on incorporating patient input into clinical trials endpoint identification. With experience in biostatistics, epidemiology and observational research methods, Dr. Girman has been involved in a range of collaborative methodology research activities with universities in the United States and Europe. She is adjunct Professor in Epidemiology at the University of North Carolina Gillings School of Public Health and a fellow of the International Society for Pharmacoepidemiology. Dr. Girman received an MS in Applied Statistics and Computer Science from Villanova University, and a DrPH. in biostatistics/epidemiology from the School of Public Health at the University of North Carolina.
President and sole proprietor of CERobs Consulting, LLC, USA
Mary Beth Ritchey is the Principal Epidemiologist for Medical Devices at RTI-HS. In her role at RTI, Dr. Ritchey performs and oversees the scientific, technical, and logistic aspects of projects including postmarketing noninterventional safety and effectiveness studies, analyses of the regulatory landscape for novel therapeutics and rare diseases, and consultation on timing, design, and methodological aspects of real-world studies for medical devices and other therapeutic products. She obtained her doctorate in Epidemiology from the UNC Gillings School of Global Public Heath with concentrations in Pharmacoepidemiology and Infectious Disease. She also has a MSPH in Epidemiology and BS in Nursing from UNC, and an undergraduate degree from Duke University. Dr. Ritchey worked at the Center for Devices and Radiologic Health (CDRH) at the Food and Drug Administration (FDA), where she served as Associate Director for Postmarket Surveillance Studies in the Division of Epidemiology. At the FDA, she managed and oversaw the Postmarket Surveillance Studies (21 CFR 822) and literature review programs and was involved in the Medical Device Epidemiology Network and FDA Sentinel Initiative. She also worked in the Comparative and Outcomes Evidence group at Merck enabling development of comparative data strategies through tools, collaborations, and establishing a continuous learning organization for comparative effectiveness research (CER). Dr. Ritchey also worked in the global safety group at Procter & Gamble, providing epidemiologic support across the therapeutic and consumer goods portfolio and enhancing the company’s signal management and risk management programs to include interpretation and synthesis across spontaneous adverse events and other safety data sources. Dr. Ritchey has experience with database analyses, literature reviews, utilization and safety surveillance of therapeutic products, registry implementation and governance, and practical application of advanced research methods.
Principal Epidemiologist for Medical Devices, RTI-HS, USA
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