Who Are We Missing? A Principled Approach to Characterizing the Underrepresented Population

Fuente: arXiv
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Auteurs principaux: Parikh, Harsh, Ross, Rachael, Stuart, Elizabeth, Rudolph, Kara
Format: Preprint
Publié: 2024
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author Parikh, Harsh
Ross, Rachael
Stuart, Elizabeth
Rudolph, Kara
author_facet Parikh, Harsh
Ross, Rachael
Stuart, Elizabeth
Rudolph, Kara
contents 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.
format Preprint
id arxiv_https___arxiv_org_abs_2401_14512
institution arXiv
publishDate 2024
record_format arxiv
spellingShingle Who Are We Missing? A Principled Approach to Characterizing the Underrepresented Population
Parikh, Harsh
Ross, Rachael
Stuart, Elizabeth
Rudolph, Kara
Methodology
Computers and Society
Machine Learning
Applications
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.
title Who Are We Missing? A Principled Approach to Characterizing the Underrepresented Population
topic Methodology
Computers and Society
Machine Learning
Applications
url https://arxiv.org/abs/2401.14512