Evidence map›Paper›PMID 35227445›Full record

ArticleValue in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research2022

A Framework for Using Real-World Data and Health Outcomes Modeling to Evaluate Machine Learning-Based Risk Prediction Models.

Patricia J Rodriguez, David L Veenstra, Patrick J Heagerty, Christopher H Goss, Kathleen J Ramos, Aasthaa Bansal

Open access · greenAbstract read
In one paragraph

Article in Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
1.0field-weighted citation impact, top 21% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 9 citations in OpenAlex.

  1. Article
  2. Review
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  8. The lung allocation score and other available models lack predictive accuracy for post-lung transplant survival.The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation · 2022
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 1 institution in 1 country.

Patricia J RodriguezThe Comparative Health Outcomes, Policy & Economics (CHOICE) Institute, University of Washington, Seattle, WA, USA. Electronic address: prodrig@uw.edu.
David L VeenstraThe Comparative Health Outcomes, Policy & Economics (CHOICE) Institute, University of Washington, Seattle, WA, USA.
Patrick J HeagertyDepartment of Biostatistics, University of Washington, Seattle, WA, USA.
Christopher H GossDivision of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, University of Washington, Seattle, WA, USA; Division of Pulmonology, Department of Pediatrics, University of Washington, Seattle, WA, USA.
Kathleen J RamosDivision of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, University of Washington, Seattle, WA, USA.
Aasthaa BansalThe Comparative Health Outcomes, Policy & Economics (CHOICE) Institute, University of Washington, Seattle, WA, USA. Electronic address: abansal@uw.edu.
University of Washington · US

Funding

Institute of Translational Health SciencesUL1TR000423 · NCATS · UNIVERSITY OF WASHINGTON · PI DISIS, MARY L. · 2012 to 2016
$49.4M
Translational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI PRADEEP k SINGH · 2010 to 2026
$21.4M
Proof of Principle Evaluation of IV Gallium Nitrate in Patients with CFUM1HL119073 · NHLBI · UNIVERSITY OF WASHINGTON · PI GOSS, CHRISTOPHER HOOPER, SINGH, PRADEEP K · 2013 to 2017
$4.8M
OPTIMIZE - DCCU01HL114589 · NHLBI · SEATTLE CHILDREN'S HOSPITAL · PI HAMBLETT, NICOLE MAYER · 2013 to 2017
$3.3M
A Phase 1b, Multi-center Study of IV Gallium Nitrate in Patients with Cystic Fibrosis who are colonized with Nontuberculosis Mycobacterium (The ABATE Study).R01FD006848 · FDA · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss, JERRY A NICK · 2020 to 2026
$3.1M
Personalized Risk-AdaptIve Surveillance strategies in cancEr -- PRAISER37CA218413 · NCI · UNIVERSITY OF WASHINGTON · PI BANSAL, AASTHAA · 2018 to 2024
$3.0M
Phase 2 Study of Intravenous Gallium Nitrate in the Treatment of Cystic FibrosisR01FD003704 · FDA · UNIVERSITY OF WASHINGTON · PI GOSS, CHRISTOPHER HOOPER · 2009 to 2018
$2.0M
Predicting survival and planning for lung transplantation in severe cystic fibrosisK23HL138154 · NHLBI · UNIVERSITY OF WASHINGTON · PI RAMOS, KATHLEEN JESSICA · 2017 to 2021
$947k
FDA HHS R01 FD003704FDA HHS R01 FD006848NCATS NIH HHS UL1 TR000423NCI NIH HHS R37 CA218413NHLBI NIH HHS K23 HL138154NHLBI NIH HHS U01 HL114589NHLBI NIH HHS UM1 HL119073NIDDK NIH HHS P30 DK089507
6 · The paper itself

Abstract

objectivesWe propose a framework of health outcomes modeling with dynamic decision making and real-world data (RWD) to evaluate the potential utility of novel risk prediction models in clinical practice. Lung transplant (LTx) referral decisions in cystic fibrosis offer a complex case study.

methodsWe used longitudinal RWD for a cohort of adults (n = 4247) from the Cystic Fibrosis Foundation Patient Registry to compare outcomes of an LTx referral policy based on machine learning (ML) mortality risk predictions to referral based on (1) forced expiratory volume in 1 second (FEV

resultsOnly 12% of patients (95% confidence interval 11%-13%) were referred for LTx over 5 years under UC, compared with 19% (18%-20%) under FEV

conclusionsHealth outcomes modeling with RWD may help to identify novel ML risk prediction models with high potential real-world clinical utility and rule out further investment in models that are unlikely to offer meaningful real-world benefits.

Indexed as

Machine LearningCystic FibrosisData CollectionForced Expiratory VolumeHumansLongitudinal StudiesLung TransplantationOutcome Assessment, Health CareReferral and ConsultationResearch DesignRisk AssessmentSurvival AnalysisTissue and Organ Procurementmachine learningmicrosimulationreal-world data

Identifiers

PMID35227445
PMCPMC9311314
OpenAlexW4200448566

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.