Evidence map›Paper›PMID 30533053›Full record

ArticlePloS one2018

Sensitivity analyses for effect modifiers not observed in the target population when generalizing treatment effects from a randomized controlled trial: Assumptions, models, effect scales, data scenarios, and implementation details.

Trang Quynh Nguyen, Benjamin Ackerman, Ian Schmid, Stephen R Cole, Elizabeth A Stuart

Open access · goldAbstract read
In one paragraph

Article in PloS one, 2018. 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
3.7field-weighted citation impact, top 6% 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

19 citing papers in PubMed, 34 citations in OpenAlex.

  1. Trial
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  3. Article
  4. Data Fusion for Partial Identification of Causal Effects.Advances in neural information processing systems · 2025
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  8. Transporting: What Is It and How Do You Do It?Current epidemiology reports · 2025
    Article
  9. Synthesis estimators for transportability with positivity violations by a continuous covariate.Journal of the Royal Statistical Society. Series A, (Statistics in Society) · 2025
    Article
  10. Article
  11. Article
  12. Causal Inference Methods for Combining Randomized Trials and Observational Studies: A Review.Statistical science : a review journal of the Institute of Mathematical Statistics · 2024
    Article
  13. Generalizability and Transportability of the National Lung Screening Trial Data: Extending Trial Results to Different Populations.Cancer epidemiology, biomarkers & prevention : a publication of the American Association for Cancer Research, cosponsored by the American Society of Preventive Oncology · 2021
    Article
  14. Article
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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

5 authors at 2 institutions in 1 country.

Trang Quynh NguyenDepartment of Mental Health, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, United States of America.
Benjamin AckermanDepartment of Biostatistics, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, United States of America.ORCID 0000-0003-2522-6623
Ian SchmidDepartment of Mental Health, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, United States of America.
Stephen R ColeDepartment of Epidemiology, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC, United States of America.
Elizabeth A StuartDepartment of Mental Health, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, United States of America.
Johns Hopkins University · USUniversity of North Carolina at Chapel Hill · US

Funding

Virology, Immunology, and Microbiology CoreP30AI050410 · NIAID · UNIV OF NORTH CAROLINA CHAPEL HILL · PI DAVID M. MARGOLIS · 2001 to 2026
$76.9M
Structural models for treatment and exposure effects in clinical HIV cohortsR01AI100654 · NIAID · UNIV OF NORTH CAROLINA CHAPEL HILL · PI COLE, STEPHEN R · 2013 to 2016
$1.6M
Generalizing RCT Efficacy Evidence: Application to NIDA Clinical Trials NetworkR01DA036520 · NIDA · JOHNS HOPKINS UNIVERSITY · PI MOJTABAI, RAMIN · 2014 to 2016
$723k
NIAID NIH HHS P30 AI050410NIAID NIH HHS R01 AI100654NIDA NIH HHS R01 DA036520
6 · The paper itself

Abstract

backgroundRandomized controlled trials are often used to inform policy and practice for broad populations. The average treatment effect (ATE) for a target population, however, may be different from the ATE observed in a trial if there are effect modifiers whose distribution in the target population is different that from that in the trial. Methods exist to use trial data to estimate the target population ATE, provided the distributions of treatment effect modifiers are observed in both the trial and target population-an assumption that may not hold in practice.

methodsThe proposed sensitivity analyses address the situation where a treatment effect modifier is observed in the trial but not the target population. These methods are based on an outcome model or the combination of such a model and weighting adjustment for observed differences between the trial sample and target population. They accommodate several types of outcome models: linear models (including single time outcome and pre- and post-treatment outcomes) for additive effects, and models with log or logit link for multiplicative effects. We clarify the methods' assumptions and provide detailed implementation instructions. ILLUSTRATION: We illustrate the methods using an example generalizing the effects of an HIV treatment regimen from a randomized trial to a relevant target population.

conclusionThese methods allow researchers and decision-makers to have more appropriate confidence when drawing conclusions about target population effects.

Indexed as

Models, TheoreticalRandomized Controlled Trials as TopicResearch DesignHumansOutcome Assessment, Health Care

Identifiers

PMID30533053
PMCPMC6289424
OpenAlexW2905563699

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.