- Montreal Accord Paper 9: Anonymization and Ethics Considerations for Capturing and Sharing Patient-Reported Outcomes
- Montreal Accord Paper 8: Patient Reported Outcomes (PRO) in Electronic Health Records Can Inform Clinical and Policy Decisions
- Montreal Accord Paper 6: Creating National Initiatives to Support Patient Reported Outcomes (PRO) Development and Use
- Montreal Accord Paper 5: Patient-Reported Outcomes Can Be Linked to Epidemiologic Measures to Monitor Populations and Inform Public Health Decisions
- Montreal Accord Paper 4: Patient-Reported Outcomes Can Inform Clinical Decision Making in Chronic Care
- Montreal Accord Paper 3: Patient Reported Outcomes (PRO) Can Facilitate Shared Decision-Making and Guide Self-Management
- Montreal Accord Paper 2: Terminology Proposed to Measure What Matters in Health
- Montreal Accord Paper 1: Pragmatic Trials and Real-World Evidence
Pragmatic Trials and Real-World Evidence
Paper 1: An Introduction to the Series
Abstract
This is the introductory paper to a series of another eight papers. In this series, we integrate the theoretical design options with the practice of conducting pragmatic trials. For most new market-approved treatments, the clinical evidence is insufficient to fully guide physicians and policy makers in choosing the optimal treatment for their patients.
Pragmatic trials can fill this gap, by providing evidence on the relative effectiveness of a treatment strategy in routine clinical practice, already in an early phase of development, while maintaining the strength of randomized controlled trials. Selecting the setting, study population, mode of intervention, comparator, and outcome are crucial in designing pragmatic trials. In combination with monitoring and data collection that does not change routine care, this will enable appropriate generalization to the target patient group in clinical practice.
To benefit from the full potential of pragmatic trials, there is a need for guidance and tools in designing these studies while ensuring operational feasibility.
This paper introduces the concept of pragmatic trial design. The complex interplay between pragmatic design options, feasibility, stakeholder acceptability, validity, precision, and generalizability will be clarified. In this way, balanced design choices can be made in pragmatic trials with an optimal chance of success in practice.
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