3.3MEFeb 26, 2024
Towards Generalizing Inferences from Trials to Target PopulationsMelody Y Huang, Harsh Parikh
Randomized Controlled Trials (RCTs) are pivotal in generating internally valid estimates with minimal assumptions, serving as a cornerstone for researchers dedicated to advancing causal inference methods. However, extending these findings beyond the experimental cohort to achieve externally valid estimates is crucial for broader scientific inquiry. This paper delves into the forefront of addressing these external validity challenges, encapsulating the essence of a multidisciplinary workshop held at the Institute for Computational and Experimental Research in Mathematics (ICERM), Brown University, in Fall 2023. The workshop congregated experts from diverse fields including social science, medicine, public health, statistics, computer science, and education, to tackle the unique obstacles each discipline faces in extrapolating experimental findings. Our study presents three key contributions: we integrate ongoing efforts, highlighting methodological synergies across fields; provide an exhaustive review of generalizability and transportability based on the workshop's discourse; and identify persistent hurdles while suggesting avenues for future research. By doing so, this paper aims to enhance the collective understanding of the generalizability and transportability of causal effects, fostering cross-disciplinary collaboration and offering valuable insights for researchers working on refining and applying causal inference methods.
4.3MEJan 25, 2024
Who Are We Missing? A Principled Approach to Characterizing the Underrepresented PopulationHarsh 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.