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Generalizing Treatment Effect Estimates From Sample to Population: A Case Study in the Difficulties of Finding
Elizabeth A Stuart1,2,3, Anna Rhodes4
11 Department of Mental Health, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.
Enhancing the external validity of randomized trials is crucial for policy relevance. Limited common data elements between trial samples and target populations challenge generalization, highlighting the need for better data collection and comparability.
Area of Science:
- Health Services Research
- Biostatistics
- Public Policy
Background:
- Growing concerns exist regarding the relevance of research findings to real-world policy and practice.
- Assessing and enhancing the external validity of randomized trials is increasingly important.
- External validity determines the applicability of trial findings to specific target populations.
Purpose of the Study:
- To highlight advances in assessing and enhancing external validity of randomized trials.
- To focus on data requirements for statistical adjustments to improve applicability of findings.
- To illustrate generalization of treatment effect estimates using a case study.
Main Methods:
- A case study was used to generalize treatment effect estimates from a randomized trial sample to a target population.
- The study compared children in a Head Start supplemental program trial (Research-Based, Developmentally Informed study) to the national Head Start population (Head Start Impact Study).
Main Results:
- Generalizing findings from the trial sample to the target population was challenging.
- Limited common data elements between the trial sample and the population data were identified as a key limitation.
Conclusions:
- More publicly available data are needed to address external validity questions.
- Future studies should prioritize collecting measures comparable to other datasets.
- Enhanced measure comparability between population data and randomized trials will broaden the scope of answerable research and policy questions.
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