TRIALSCOPE: A Unifying Causal Framework for Scaling Real-World Evidence Generation with Biomedical Language Models

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Main Authors: González, Javier, Ueno, Risa, Wong, Cliff, Gero, Zelalem, Bagga, Jass, Chien, Isabel, Oravkin, Eduard, Kiciman, Emre, Nori, Aditya, Weerasinghe, Roshanthi, Leidner, Rom S., Piening, Brian, Naumann, Tristan, Bifulco, Carlo, Poon, Hoifung
Format: Preprint
Published: 2023
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author González, Javier
Ueno, Risa
Wong, Cliff
Gero, Zelalem
Bagga, Jass
Chien, Isabel
Oravkin, Eduard
Kiciman, Emre
Nori, Aditya
Weerasinghe, Roshanthi
Leidner, Rom S.
Piening, Brian
Naumann, Tristan
Bifulco, Carlo
Poon, Hoifung
author_facet González, Javier
Ueno, Risa
Wong, Cliff
Gero, Zelalem
Bagga, Jass
Chien, Isabel
Oravkin, Eduard
Kiciman, Emre
Nori, Aditya
Weerasinghe, Roshanthi
Leidner, Rom S.
Piening, Brian
Naumann, Tristan
Bifulco, Carlo
Poon, Hoifung
contents The rapid digitization of real-world data presents an unprecedented opportunity to optimize healthcare delivery and accelerate biomedical discovery. However, these data are often found in unstructured forms such as clinical notes in electronic medical records (EMRs), and is typically plagued by confounders, making it challenging to generate robust real-world evidence (RWE). Therefore, we present TRIALSCOPE, a framework designed to distil RWE from population level observational data at scale. TRIALSCOPE leverages biomedical language models to structure clinical text at scale, employs advanced probabilistic modeling for denoising and imputation, and incorporates state-of-the-art causal inference techniques to address common confounders in treatment effect estimation. Extensive experiments were conducted on a large-scale dataset of over one million cancer patients from a single large healthcare network in the United States. TRIALSCOPE was shown to automatically curate high-quality structured patient data, expanding the dataset and incorporating key patient attributes only available in unstructured form. The framework reduces confounding in treatment effect estimation, generating comparable results to randomized controlled lung cancer trials. Additionally, we demonstrate simulations of unconducted clinical trials - including a pancreatic cancer trial with varying eligibility criteria - using a suite of validation tests to ensure robustness. Thorough ablation studies were conducted to better understand key components of TRIALSCOPE and establish best practices for RWE generation from EMRs. TRIALSCOPE was able to extract data cancer treatment data from EMRs, overcoming limitations of manual curation. We were also able to show that TRIALSCOPE could reproduce results of lung and pancreatic cancer clinical trials from the extracted real world data.
format Preprint
id arxiv_https___arxiv_org_abs_2311_01301
institution arXiv
publishDate 2023
record_format arxiv
spellingShingle TRIALSCOPE: A Unifying Causal Framework for Scaling Real-World Evidence Generation with Biomedical Language Models
González, Javier
Ueno, Risa
Wong, Cliff
Gero, Zelalem
Bagga, Jass
Chien, Isabel
Oravkin, Eduard
Kiciman, Emre
Nori, Aditya
Weerasinghe, Roshanthi
Leidner, Rom S.
Piening, Brian
Naumann, Tristan
Bifulco, Carlo
Poon, Hoifung
Machine Learning
Artificial Intelligence
Methodology
The rapid digitization of real-world data presents an unprecedented opportunity to optimize healthcare delivery and accelerate biomedical discovery. However, these data are often found in unstructured forms such as clinical notes in electronic medical records (EMRs), and is typically plagued by confounders, making it challenging to generate robust real-world evidence (RWE). Therefore, we present TRIALSCOPE, a framework designed to distil RWE from population level observational data at scale. TRIALSCOPE leverages biomedical language models to structure clinical text at scale, employs advanced probabilistic modeling for denoising and imputation, and incorporates state-of-the-art causal inference techniques to address common confounders in treatment effect estimation. Extensive experiments were conducted on a large-scale dataset of over one million cancer patients from a single large healthcare network in the United States. TRIALSCOPE was shown to automatically curate high-quality structured patient data, expanding the dataset and incorporating key patient attributes only available in unstructured form. The framework reduces confounding in treatment effect estimation, generating comparable results to randomized controlled lung cancer trials. Additionally, we demonstrate simulations of unconducted clinical trials - including a pancreatic cancer trial with varying eligibility criteria - using a suite of validation tests to ensure robustness. Thorough ablation studies were conducted to better understand key components of TRIALSCOPE and establish best practices for RWE generation from EMRs. TRIALSCOPE was able to extract data cancer treatment data from EMRs, overcoming limitations of manual curation. We were also able to show that TRIALSCOPE could reproduce results of lung and pancreatic cancer clinical trials from the extracted real world data.
title TRIALSCOPE: A Unifying Causal Framework for Scaling Real-World Evidence Generation with Biomedical Language Models
topic Machine Learning
Artificial Intelligence
Methodology
url https://arxiv.org/abs/2311.01301