Evidence map›Paper›PMID 31732971›Full record

ArticleBiometrics2020

Statistical inference for net benefit measures in biomarker validation studies.

Tracey L Marsh, Holly Janes, Margaret S Pepe

Open access · greenAbstract read
In one paragraph

Article in Biometrics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
1.9field-weighted citation impact, top 12% 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

16 citing papers in PubMed, 21 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Article
  5. Article
  6. Expected Value of Sample Information Calculations for Risk Prediction Model Validation.Medical decision making : an international journal of the Society for Medical Decision Making · 2025
    Article
  7. Article
  8. Article
  9. Article
  10. Article
  11. Closed-Form Solution of the Unit Normal Loss Integral in 2 Dimensions, with Application in Value-of-Information Analysis.Medical decision making : an international journal of the Society for Medical Decision Making · 2023
    Article
  12. Value-of-Information Analysis for External Validation of Risk Prediction Models.Medical decision making : an international journal of the Society for Medical Decision Making · 2023
    Article
  13. Article
  14. Bayesian Statistics for Medical Devices: Progress Since 2010.Therapeutic innovation & regulatory science · 2023
    Review
  15. Review
  16. 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

3 authors at 1 institution in 1 country.

Tracey L MarshFred Hutchinson Cancer Research Center, Seattle, Washington.ORCID 0000-0002-6865-5092
Holly JanesFred Hutchinson Cancer Research Center, Seattle, Washington.
Margaret S PepeFred Hutchinson Cancer Research Center, Seattle, Washington.
Fred Hutch Cancer Center · US

Funding

The Early Detection Research Network: Data Management and Coordinating CenterU24CA086368 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI RUTH D ETZIONI, Ziding Feng · 2010 to 2026
$75.7M
EDRN--DATA MANAGEMENT AND COORDINATING CENTERU01CA086368 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI FENG, ZIDING · 2000 to 2010
$16.9M
Statistical Methods for Evaluating Markers for Treatment SelectionR01CA152089 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI JANES, HOLLY · 2010 to 2021
$3.8M
NCI NIH HHS R01 CA152089NCI NIH HHS U01 CA086368NCI NIH HHS U24 CA086368
6 · The paper itself

Abstract

Referral strategies based on risk scores and medical tests are commonly proposed. Direct assessment of their clinical utility requires implementing the strategy and is not possible in the early phases of biomarker research. Prior to late-phase studies, net benefit measures can be used to assess the potential clinical impact of a proposed strategy. Validation studies, in which the biomarker defines a prespecified referral strategy, are a gold standard approach to evaluating biomarker potential. Uncertainty, quantified by a confidence interval, is important to consider when deciding whether a biomarker warrants an impact study, does not demonstrate clinical potential, or that more data are needed. We establish distribution theory for empirical estimators of net benefit and propose empirical estimators of variance. The primary results are for the most commonly employed estimators of net benefit: from cohort and unmatched case-control samples, and for point estimates and net benefit curves. Novel estimators of net benefit under stratified two-phase and categorically matched case-control sampling are proposed and distribution theory developed. Results for common variants of net benefit and for estimation from right-censored outcomes are also presented. We motivate and demonstrate the methodology with examples from lung cancer research and highlight its application to study design.

Indexed as

Research DesignBiomarkersCase-Control StudiesHumansUncertaintyBiomarkersbiomarkerclinical decision ruleclinical impactclinical utilityrisk prediction

Identifiers

PMID31732971
PMCPMC7228830
OpenAlexW2983520337

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

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.