Evidence map›Paper›PMID 29589296›Full record

ArticleApplied health economics and health policy2018

Understanding Treatment Effect Estimates When Treatment Effects Are Heterogeneous for More Than One Outcome.

John M Brooks, Cole G Chapman, Mary C Schroeder

Abstract read
In one paragraph

Article in Applied health economics and health policy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing 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

12 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

3 authors.

John M BrooksUniversity of South Carolina and the Center for Effectiveness Research in Orthopaedics, 915 Greene Street, Room 303D, Columbia, SC, 29208, USA. john-brooks@sc.edu.ORCID 0000-0001-7365-4417
Cole G ChapmanUniversity of South Carolina and the Center for Effectiveness Research in Orthopaedics, 915 Greene Street, Room 303B, Columbia, SC, 29208, USA.ORCID 0000-0002-1132-3767
Mary C SchroederUniversity of Iowa College of Pharmacy, 115 S Grand Ave, Room S525, Iowa City, IA, 52242, USA.ORCID 0000-0002-1043-8140

Funding

Interpreting IV Estimates with Treatment Effect Heterogeneity: ACE/ARBs & RaceRC4AG038635 · NIA · UNIVERSITY OF IOWA · PI BROOKS, JOHN M · 2010 to 2010
$1.5M
NIA NIH HHS RC4 AG038635Patient-Centered Outcomes Research Institute ME-1303-6011
6 · The paper itself

Abstract

backgroundPatient-centred care requires evidence of treatment effects across many outcomes. Outcomes can be beneficial (e.g. increased survival or cure rates) or detrimental (e.g. adverse events, pain associated with treatment, treatment costs, time required for treatment). Treatment effects may also be heterogeneous across outcomes and across patients. Randomized controlled trials are usually insufficient to supply evidence across outcomes. Observational data analysis is an alternative, with the caveat that the treatments observed are choices. Real-world treatment choice often involves complex assessment of expected effects across the array of outcomes. Failure to account for this complexity when interpreting treatment effect estimates could lead to clinical and policy mistakes.

objectiveOur objective was to assess the properties of treatment effect estimates based on choice when treatments have heterogeneous effects on both beneficial and detrimental outcomes across patients.

methodsSimulation methods were used to highlight the sensitivity of treatment effect estimates to the distributions of treatment effects across patients across outcomes. Scenarios with alternative correlations between benefit and detriment treatment effects across patients were used. Regression and instrumental variable estimators were applied to the simulated data for both outcomes.

resultsTrue treatment effect parameters are sensitive to the relationships of treatment effectiveness across outcomes in each study population. In each simulation scenario, treatment effect estimate interpretations for each outcome are aligned with results shown previously in single outcome models, but these estimates vary across simulated populations with the correlations of treatment effects across patients across outcomes.

conclusionsIf estimator assumptions are valid, estimates across outcomes can be used to assess the optimality of treatment rates in a study population. However, because true treatment effect parameters are sensitive to correlations of treatment effects across outcomes, decision makers should be cautious about generalizing estimates to other populations.

Indexed as

Treatment OutcomeAlgorithmsCost-Benefit AnalysisHumansObservationQuality-Adjusted Life YearsRegression Analysis

Identifiers

PMID29589296
PMCPMC6437676

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Registered trials

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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.