Pragmatic Randomized Clinical Trials Using Primary Data Collection and Electronic Health Records

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. They are less restrictive and more generalizable than traditional randomized controlled trials, however they 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 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 trialsDiscusses real cases on operational challenges in launching and conducting pRCTs in electronic health records INDICE: I Introduction 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) 7. Dissemination 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 VII Dissemination 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 39. Biologics 40. Devices 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 X Conclusions 49. Concluding remarks

• ISBN: 978-0-12-817663-4
• Editorial: Academic Press
• Encuadernacion: Rústica
• Páginas: 500
• Fecha Publicación: 01/06/2020
• Nº Volúmenes: 1
• Idioma: Inglés