Evidence map›Paper›PMID 28742260›Full record

ArticleBiometrics2018

Incorporating Patient Preferences into Estimation of Optimal Individualized Treatment Rules.

Emily L Butler, Eric B Laber, Sonia M Davis, Michael R Kosorok

Abstract read
In one paragraph

Article in Biometrics, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing 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

20 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Review
  5. Article
  6. Article
  7. Article
  8. Article
  9. Article
  10. Article
  11. When the Ends do not Justify the Means: Learning Who is Predicted to Have Harmful Indirect Effects.Journal of the Royal Statistical Society. Series A, (Statistics in Society) · 2022
    Article
  12. Article
  13. Article
  14. Article
  15. Article
  16. Robust Q-learning.Journal of the American Statistical Association · 2021
    Article
  17. Learning Individualized Treatment Rules for Multiple-Domain Latent Outcomes.Journal of the American Statistical Association · 2021
    Article
  18. Estimation and Optimization of Composite Outcomes.Journal of machine learning research : JMLR · 2021
    Article
  19. Article
  20. Precision Medicine.Annual review of statistics and its application · 2019
    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

4 authors.

Emily L ButlerDepartment of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, U.S.A.
Eric B LaberDepartment of Statistics, North Carolina State University, Raleigh, North Carolina, U.S.A.ORCID 0000-0003-2640-7696
Sonia M DavisDepartment of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, U.S.A.
Michael R KosorokDepartment of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, U.S.A.ORCID 0000-0002-6070-9738

Funding

Statistical Methods for Cancer Clinical TrialsP01CA142538 · NCI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI DAVIDIAN, MARIE, KOSOROK, MICHAEL R · 2010 to 2019
$21.3M
Statistical methods for healthcare in complex patients with diabetesR01DK108073 · NIDDK · FRED HUTCHINSON CANCER RESEARCH CENTER · PI ZHAO, YINGQI · 2015 to 2019
$1.5M
NCI NIH HHS P01 CA142538NIDDK NIH HHS R01 DK108073
6 · The paper itself

Abstract

Precision medicine seeks to provide treatment only if, when, to whom, and at the dose it is needed. Thus, precision medicine is a vehicle by which healthcare can be made both more effective and efficient. Individualized treatment rules operationalize precision medicine as a map from current patient information to a recommended treatment. An optimal individualized treatment rule is defined as maximizing the mean of a pre-specified scalar outcome. However, in settings with multiple outcomes, choosing a scalar composite outcome by which to define optimality is difficult. Furthermore, when there is heterogeneity across patient preferences for these outcomes, it may not be possible to construct a single composite outcome that leads to high-quality treatment recommendations for all patients. We simultaneously estimate the optimal individualized treatment rule for all composite outcomes representable as a convex combination of the (suitably transformed) outcomes. For each patient, we use a preference elicitation questionnaire and item response theory to derive the posterior distribution over preferences for these composite outcomes and subsequently derive an estimator of an optimal individualized treatment rule tailored to patient preferences. We prove that as the number of subjects and items on the questionnaire diverge, our estimator is consistent for an oracle optimal individualized treatment rule wherein each patient's preference is known a priori. We illustrate the proposed method using data from a clinical trial on antipsychotic medications for schizophrenia.

Indexed as

Models, StatisticalAntipsychotic AgentsHumansPatient PreferencePrecision MedicineSchizophreniaSurveys and QuestionnairesTreatment OutcomeAntipsychotic AgentsCompeting outcomesIndividualized treatment rulesItem response theoryPrecision medicine

Identifiers

PMID28742260
PMCPMC5785589

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.