2015Journal of the Royal Statistical Society Series A (Statistics in Society)Open access

From Sample Average Treatment Effect to Population Average Treatment Effect on the Treated: Combining Experimental with Observational Studies to Estimate Population Treatment Effects

Erin Hartman, Richard Grieve, Roland R. Ramsahai, Jasjeet S. Sekhon

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Abstract

Summary Randomized controlled trials (RCTs) can provide unbiased estimates of sample average treatment effects. However, a common concern is that RCTs may fail to provide unbiased estimates of population average treatment effects. We derive the assumptions that are required to identify population average treatment effects from RCTs. We provide placebo tests, which formally follow from the identifying assumptions and can assess whether they hold. We offer new research designs for estimating population effects that use non-randomized studies to adjust the RCT data. This approach is considered in a cost-effectiveness analysis of a clinical intervention: pulmonary artery catheterization.

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Summary Randomized controlled trials (RCTs) can provide unbiased estimates of sample average treatment effects. However, a common concern is that RCTs may fail to provide unbiased estimates of population average treatment effects. We derive the assumptions that are required to identify population average treatment effects from RCTs. We provide placebo tests, which formally follow from the identifying assumptions and can assess whether they hold. We offer new research designs for estimating population effects that use non-randomized studies to adjust the RCT data. This approach is considered in a cost-effectiveness analysis of a clinical intervention: pulmonary artery catheterization.

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Available abstract

Summary Randomized controlled trials (RCTs) can provide unbiased estimates of sample average treatment effects. However, a common concern is that RCTs may fail to provide unbiased estimates of population average treatment effects. We derive the assumptions that are required to identify population average treatment effects from RCTs. We provide placebo tests, which formally follow from the identifying assumptions and can assess whether they hold. We offer new research designs for estimating population effects that use non-randomized studies to adjust the RCT data. This approach is considered in a cost-effectiveness analysis of a clinical intervention: pulmonary artery catheterization.

Key concepts: Observational study, Randomized controlled trial, Sample size determination, Population, Average treatment effect, Medicine, Treatment effect, Randomized experiment

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