biostudies-literatureUnknownThangaraj PMNHLBI NIH HHS<h4>Background</h4>Randomized clinical trials (RCTs) define evidence-based medicine, but quantifying their generalizability to real-world patients remains challenging. We propose a multidimensional approach to compare individuals in RCT and electronic health record (EHR) cohorts by quantifying their representativeness and estimating real-world effects based on individualized treatment effects (ITE) observed in RCTs.<h4>Methods</h4>We identified 65 pre-randomization characteristics of an RCT of heart failure with preserved ejection fraction (HFpEF), the Treatment of Preserved Cardiac Function Heart Failure with an Aldosterone Antagonist Trial (TOPCAT), and extracted those features from patients with HFpEF from the EHR within the Yale New Haven Health System. We then assessed the real-world medRxiv : the preprint server for health sciences2024.05.15.24306285https://www.ebi.ac.uk/biostudies/studies/S-EPMC11118629biostudies-literatureComputational Phenomapping of Randomized Clinical Trials to Enable Assessment of their Real-world Representativeness and Personalized Inference.PMC11118629F32 HL170592T35 HL007649R01 HL167858K23 HL153775T32 HL155000Dhingra LSSuchard MAOikonomou EKKhera RJayaram RThangaraj PMAminorroaya AfalseComputational Phenomapping of Randomized Clinical Trials to Enable Assessment of their Real-world Representativeness and Personalized Inference.<h4>Background</h4>Randomized clinical trials (RCTs) define evidence-based medicine, but quantifying their generalizability to real-world patients remains challenging. We propose a multidimensional approach to compare individuals in RCT and electronic health record (EHR) cohorts by quantifying their representativeness and estimating real-world effects based on individualized treatment effects (ITE) observed in RCTs.<h4>Methods</h4>We identified 65 pre-randomization characteristics of an RCT of heart failure with preserved ejection fraction (HFpEF), the Treatment of Preserved Cardiac Function Heart Failure with an Aldosterone Antagonist Trial (TOPCAT), and extracted those features from patients with HFpEF from the EHR within the Yale New Haven Health System. We then assessed the real-world 2025-01-01T00:00:00Z2025 Jan2026-05-28T03:19:18.138Z2025-04-04T01:40:17.808ZS-EPMC111186293879845710.1101/2024.05.15.24306285