Elizabeth A. Stuart

Wenqing Zhang, Trang Nguyen, Elizabeth A. Stuart et al.

Systematic reviews are crucial for synthesizing scientific evidence but remain labor-intensive, especially when extracting detailed methodological information. Large language models (LLMs) offer potential for automating methodological assessments, promising to transform evidence synthesis. Here, using causal mediation analysis as a representative methodological domain, we benchmarked state-of-the-art LLMs against expert human reviewers across 180 full-text scientific articles. Model performance closely correlated with human judgments (accuracy correlation 0.71; F1 correlation 0.97), achieving near-human accuracy on straightforward, explicitly stated methodological criteria. However, accuracy sharply declined on complex, inference-intensive assessments, lagging expert reviewers by up to 15%. Errors commonly resulted from superficial linguistic cues -- for instance, models frequently misinterpreted keywords like "longitudinal" or "sensitivity" as automatic evidence of rigorous methodological approache, leading to systematic misclassifications. Longer documents yielded lower model accuracy, whereas publication year showed no significant effect. Our findings highlight an important pattern for practitioners using LLMs for methods review and synthesis from full texts: current LLMs excel at identifying explicit methodological features but require human oversight for nuanced interpretations. Integrating automated information extraction with targeted expert review thus provides a promising approach to enhance efficiency and methodological rigor in evidence synthesis across diverse scientific fields.

Harsh Parikh, Rachael Ross, Elizabeth Stuart et al.

Randomized controlled trials (RCTs) serve as the cornerstone for understanding causal effects, yet extending inferences to target populations presents challenges due to effect heterogeneity and underrepresentation. Our paper addresses the critical issue of identifying and characterizing underrepresented subgroups in RCTs, proposing a novel framework for refining target populations to improve generalizability. We introduce an optimization-based approach, Rashomon Set of Optimal Trees (ROOT), to characterize underrepresented groups. ROOT optimizes the target subpopulation distribution by minimizing the variance of the target average treatment effect estimate, ensuring more precise treatment effect estimations. Notably, ROOT generates interpretable characteristics of the underrepresented population, aiding researchers in effective communication. Our approach demonstrates improved precision and interpretability compared to alternatives, as illustrated with synthetic data experiments. We apply our methodology to extend inferences from the Starting Treatment with Agonist Replacement Therapies (START) trial -- investigating the effectiveness of medication for opioid use disorder -- to the real-world population represented by the Treatment Episode Dataset: Admissions (TEDS-A). By refining target populations using ROOT, our framework offers a systematic approach to enhance decision-making accuracy and inform future trials in diverse populations.