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Real-world experiences with generating real-world evidence: Case Studies from PCORI's pragmatic clinical Studies
Mark D Neuman1, Michael D Kappelman2, Elliot Israel3
1Department of Anesthesiology and Critical Care, University of Pennsylvania Perelman School of Medicine, Philadelphia, USA; Center for Perioperative Outcomes Research and Transformation, University of Pennsylvania Perelman School of Medicine, Philadelphia, USA.
Contemporary Clinical Trials
|October 10, 2020
Summary
Conducting pragmatic trials requires balancing real-world applicability with rigorous study design. Addressing implementation challenges is key for generating generalizable evidence in healthcare.
Area of Science:
- Health Services Research
- Clinical Trials Methodology
- Evidence-Based Practice
Background:
- Pragmatic trials are crucial for generating actionable evidence in real-world healthcare settings.
- Despite methodological focus, practical and logistical challenges in real-world trial implementation are under-documented.
Purpose of the Study:
- To examine common tradeoffs in the design and implementation of pragmatic trials.
- To illustrate implementation challenges using case studies of Patient Centered Outcomes Research Institute (PCORI)-funded trials.
Main Methods:
- Convened an expert panel in 2017.
- Analyzed three ongoing PCORI-funded pragmatic trials as case studies.
- Identified and summarized common implementation challenges and design tradeoffs.
Main Results:
- Investigators aimed for generalizable findings but incorporated "explanatory" elements for a "fit-for-purpose" design.
- Balancing pragmatic and explanatory perspectives involved diverse aspects of trial design and implementation.
- Specific tradeoffs varied across the examined case studies.
Conclusions:
- Generating generalizable, real-world evidence necessitates balancing pragmatic and explanatory approaches iteratively.
- Tradeoffs were guided by maintaining pragmatism and context-specific practical considerations.
- Successful pragmatic trial implementation requires adapting to research questions and study contexts.