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Extending Inferences from a Randomized Trial to Trial-eligible and Treatment-candidate Target Populations: Examples
Jessica C Young1,2, Issa J Dahabreh3,4,5, Stefan James6
1From the Unit of Epidemiology, Institute of Environmental Medicine, Karolinska Institutet, Stockholm, Sweden.
Epidemiology (Cambridge, Mass.)
|May 4, 2026
Summary
Extending randomized trial findings to broader populations is crucial. This study found similar risk ratios for bivalirudin vs. heparin in trial-eligible and treatment-candidate groups compared to the index trial.
Area of Science:
- Cardiovascular Medicine
- Clinical Trial Methodology
- Health Services Research
Background:
- Randomized trials inform healthcare decisions, but trial participants may not represent real-world populations.
- The VALIDATE-SWEDEHEART trial compared bivalirudin and heparin during percutaneous coronary intervention.
- Generalizing trial results to wider patient groups is a key challenge in evidence-based medicine.
Purpose of the Study:
- To extend inferences from the VALIDATE-SWEDEHEART trial to two broader, clinically relevant populations.
- To compare the effectiveness of bivalirudin versus heparin in trial-eligible and treatment-candidate populations.
- To assess the generalizability of randomized controlled trial (RCT) findings.
Main Methods:
- Logistic regression models were used to estimate outcomes at 180 days based on treatment assignment.
- Risk estimates were standardized to the baseline covariate distributions of the target populations.
- Swedish healthcare registry data characterized the trial-eligible and treatment-candidate populations.
Main Results:
- The estimated risk difference for bivalirudin vs. heparin was -1.1% in the trial-eligible and -1.0% in the treatment-candidate population.
- Risk ratios were similar to the index trial (0.92 and 0.93, respectively).
- Absolute risks were slightly higher (0.8-1.2 percentage points) in these broader populations compared to the index trial.
Conclusions:
- Risk ratios for bivalirudin vs. heparin align between the index trial and broader populations.
- Evidence from RCTs can be extended to wider populations using registry data.
- This approach helps address questions about treatment effects in populations beyond trial participants.
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