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This design utilizes active learning to enhance treatment effect detection in targeted patient populations, highlighting regions of efficacy.
Matched-pair experimental designs aim to detect treatment effects by pairing participants and comparing within-pair outcome differences. In many situations, the overall effect size across the entire population is small. Then, the focus naturally shifts to identifying and targeting high treatment-effect regions where the intervention is most effective. This paper proposes a matched-pair experimental design that sequentially and actively enrolls patients in high treatment-effect regions. Importantly, we frame the identification of the target region as a classification problem and propose an active learning framework tailored to matched-pair designs. Our design not only reduces the experimental cost of detecting treatment efficacy, but also ensures that the identified regions enclose the entire high-treatment-effect regions. Our theoretical analysis of the framework's label complexity and experiments in practical scenarios demonstrate the efficiency and advantages of the approach.
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Li et al. (2025) studied this question.
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