Evidence map›Paper›PMID 36700492›Full record

ArticleStatistics in medicine2023

Measuring the performance of prediction models to personalize treatment choice.

Orestis Efthimiou, Jeroen Hoogland, Thomas P A Debray, Michael Seo, Toshiaki A Furukawa, Matthias Egger, Ian R White

Abstract read
In one paragraph

Article in Statistics in medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed
–field-weighted citation impact
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

19 citing papers in PubMed.

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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

7 authors.

Orestis EfthimiouInstitute of Social and Preventive Medicine (ISPM), University of Bern, Bern, Switzerland.ORCID 0000-0002-0955-7572
Jeroen HooglandJulius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.ORCID 0000-0002-2397-6052
Thomas P A DebrayJulius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.ORCID 0000-0002-1790-2719
Michael SeoInstitute of Social and Preventive Medicine (ISPM), University of Bern, Bern, Switzerland.ORCID 0000-0002-5229-590X
Toshiaki A FurukawaDepartments of Health Promotion and Human Behavior and of Clinical Epidemiology, Kyoto University Graduate School of Medicine/School of Public Health, Kyoto, Japan.
Matthias EggerInstitute of Social and Preventive Medicine (ISPM), University of Bern, Bern, Switzerland.
Ian R WhiteMRC Clinical Trials Unit at UCL, University College London, London, UK.ORCID 0000-0002-6718-7661

Funding

Medical Research Council MC_UU_00004/07
6 · The paper itself

Abstract

When data are available from individual patients receiving either a treatment or a control intervention in a randomized trial, various statistical and machine learning methods can be used to develop models for predicting future outcomes under the two conditions, and thus to predict treatment effect at the patient level. These predictions can subsequently guide personalized treatment choices. Although several methods for validating prediction models are available, little attention has been given to measuring the performance of predictions of personalized treatment effect. In this article, we propose a range of measures that can be used to this end. We start by defining two dimensions of model accuracy for treatment effects, for a single outcome: discrimination for benefit and calibration for benefit. We then amalgamate these two dimensions into an additional concept, decision accuracy, which quantifies the model's ability to identify patients for whom the benefit from treatment exceeds a given threshold. Subsequently, we propose a series of performance measures related to these dimensions and discuss estimating procedures, focusing on randomized data. Our methods are applicable for continuous or binary outcomes, for any type of prediction model, as long as it uses baseline covariates to predict outcomes under treatment and control. We illustrate all methods using two simulated datasets and a real dataset from a trial in depression. We implement all methods in the R package predieval. Results suggest that the proposed measures can be useful in evaluating and comparing the performance of competing models in predicting individualized treatment effect.

Indexed as

Models, StatisticalPrecision MedicineRandomized Controlled Trials as TopicClinical Decision RulesHumansTreatment Outcomeheterogeneous treatment effectspersonalized medicineprediction modelling

Identifiers

PMID36700492
PMCPMC7615726

What OpenQuestion holds

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LicenceCC BY
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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.