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Skills Course: Emerging methods in pragmatic trials
Course Aim
Pragmatic randomized clinical trials (pRCTs) are trials conducted in a routine clinical practice setting with minimal disruption to clinical health care (other than randomization) to assess effectiveness and safety of interventions. They require the expertise of pharmacoepidemiologists who understand considerations and methodologic challenges that arise with generating and analyzing real world data compared with traditional “explanatory” RCTs.
This year, the course will focus on emerging methods as the need for rapid, rigorous, and generalizable evidence increases and technologies have improved for clinical data collection. The course will cover high level designs, including the typical attributes of pragmatic trials and then cover the methods for ensuring the generation of robust real world evidence and for leveraging electronic health records and other sources of RWD to improve efficiency of pRCTs and support embedding of pRCTs in health care delivery settings. Operational challenges and lessons learned from conducting pRCTs will be shared along with case examples from PCORI and other invited speakers.
Requisites Statement: Entry level, though familiarity with basic principles of observational and/or experimental study design and statistical methods is recommended. Recommended pre-course reading material will also be provided.
Course Objectives
· To understand key design considerations for pRCTs and drivers of design choices
· To understand statistical issues for the analyses of pRCTs
· To understand considerations pertaining to primary data collection vs use of secondary data sources
· To identify and address operational considerations and challenges in implementing pRCTs
· To gain insight into current and recent pragmatic trial case examples with regard to their methods, implementation, operational challenges, and intended use as real-world-evidence
Syllabus Outline
1. Overview of Pragmatic Trials-J Christian
To introduce attendees to the rationale and context for conducting pRCTs, present different design tools for researchers, share pivotal studies and articles, and examples of impact with various stakeholders.
2. Emerging methods for EHR linkage and data collection strategies for pragmatic trials – P Velentgas
The following topics related to study designs and outcome selection for pragmatic trials will be addressed: patient-level and cluster randomized designs, cross-over trials, blinding, inclusion and exclusion criteria, choice of comparators, and data collection with emphasis on trials that blend primary data collection with EHRs or other secondary data.
3. Statistical analyses for pragmatic trials – C Girman
Analytic methods related to pragmatic trials will be reviewed, including different considerations related to randomization, sample size estimation for cluster randomized and stepped wedge designs, appropriate analysis methods, and non-inferiority vs. superiority hypothesis testing.
4. Recent case examples of pragmatic trials funded by PCORI – A Trontell
Dr. Trontell will provide an overview of some of the recently completed PCORI-funded pragmatic trials, focusing on unique features of PCORI’s approach and challenges of implementation. Investigator feedback on challenges in pragmatic trials and a patient-centered approach to design of such trials will also be given.
5. The AIRWISE Study: Study Design Features and Lessons Learned: Vincent Willey
The AIRWISE study, a PCT assessing treatment in COPD patients, will be reviewed. Specific highlights will include discussion on the use of administrative claims data throughout the design, operational and analytic phases of the study as well as a discussion on the lessons learned.
6. Results and Lessons learned from the VERVE trial of the Safety and Effectiveness of Live Zoster Vaccine in Anti-TNF Users: Jeffrey Curtis
Dr. Curtis will share key learnings and challenges from the NIH funded VERVE trial, which utilized screening of EHRs for patient identification and included capabilities to link to EMR and administrative claims data for longer term outcomes assessment.
Authors
Speakers
Priscilla Velentgas
Cynthia Girman, FISPE
Patient Centered Outcomes Research Institute
Anne Trontell
Patient Centered Outcomes Research Institute
Jeffrey Curtis
jcurtis@uab.edu
University of Alabama Birmingham
Vincent Willey
vwilley@healthcore.com
HealthCore, Inc.
Skills Course: Emerging methods in pragmatic trials
Category
Skills Course
Description
Presenting Author
Jennifer Christian, FISPE
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