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Published on: September 20, 2019
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Efficacy and effectiveness trials have different goals, use different tools, and generate different messages
Franz Porzsolt1, Natália Galito Rocha2, Alessandra C Toledo-Arruda2
1Health Care Research, Department of General and Visceral Surgery, University Hospital Ulm, Ulm, Germany; Institute of Clinical Economics (ICE) eV, Ulm, Germany.
Pragmatic and Observational Research
|October 25, 2016
Summary
Scientists and clinicians agree that two distinct clinical trial types are essential. Explanatory trials (RCTs) demonstrate efficacy under ideal conditions, while pragmatic trials (PCTs) show effectiveness in real-world settings.
Area of Science:
- Clinical trial design
- Medical research methodology
- Evidence-based medicine
Background:
- Discrepancy exists between theory-based scientists' observations and practice-based clinicians' needs regarding clinical trial design.
- Scientists view clinical trials as a continuum from explanatory to pragmatic.
- Clinicians seek trials that address real-world problems and provide applicable results.
Purpose of the Study:
- To reconcile the perspectives of scientists and clinicians on optimal clinical trial design.
- To identify the distinct roles of explanatory and pragmatic trials in medical research.
- To propose a framework for demonstrating both efficacy and effectiveness through appropriate trial types.
Main Methods:
- Summarized discussions between scientists and clinicians regarding clinical trial design.
- Analyzed the differing viewpoints and expectations of each group.
- Synthesized findings to propose a dual-trial approach for efficacy and effectiveness.
Main Results:
- Scientists observe a continuum from explanatory to pragmatic trials.
- Clinicians desire separate trials for demonstrating efficacy (ideal conditions) and effectiveness (real-world conditions).
- A single trial cannot simultaneously assess both efficacy and effectiveness.
Conclusions:
- Two distinct trial types are necessary: Randomized Controlled Trials (RCTs) for efficacy and Pragmatic Controlled Trials (PCTs) for effectiveness.
- RCTs provide insights into expected healthcare outcomes under ideal circumstances.
- PCTs reflect actual observed outcomes in real-world clinical practice.
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