Evidence map›Paper›PMID 36608311›Full record

ArticleJCO clinical cancer informatics2023

Target Trial Emulation: A Design Tool for Cancer Clinical Trials.

Sandi A Kwee, Linda L Wong, Christina Ludema, Chris K Deng, Deborah Taira, Todd Seto, Douglas Landsittel

Open access · hybridAbstract read
In one paragraph

Article in JCO clinical cancer informatics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
5.4field-weighted citation impact, top 3% 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

15 citing papers in PubMed, 16 citations in OpenAlex.

  1. Article
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  9. Real-World Data and Causal Machine Learning to Enhance Drug Development.Therapeutic innovation & regulatory science · 2025
    Review
  10. Review
  11. Article
  12. Review
  13. Article
  14. Article
  15. Clinical Research Informatics: a Decade-in-Review.Yearbook of medical informatics · 2024
    Review
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 at 4 institutions in 1 country.

Sandi A KweeThe Queen's Medical Center, Honolulu, HI.ORCID 0000-0002-4624-8787
Linda L WongUniversity of Hawai`i Cancer Center, Clinical and Translational Sciences Program, University of Hawaii at Manoa, Honolulu, HI.
Christina LudemaIndiana University School of Public Health, Bloomington, IN.ORCID 0000-0003-0779-810X
Chris K DengUniversity of Hawai`i Cancer Center, Clinical and Translational Sciences Program, University of Hawaii at Manoa, Honolulu, HI.
Deborah TairaThe Daniel K. Inouye College of Pharmacy, University of Hawaii at Hilo, Hilo, HI.
Todd SetoThe Queen's Medical Center, Honolulu, HI.ORCID 0000-0001-9872-4290
Douglas LandsittelIndiana University School of Public Health, Bloomington, IN.
University of Hawaiʻi at Mānoa · USIndiana University Bloomington · USQueen's Medical Center · USUniversity of Hawaii at Hilo · US

Funding

University of Hawaii Cancer Center CCSGP30CA071789 · NCI · UNIVERSITY OF HAWAII AT MANOA · PI GERTRAUD MASKARINEC · 1996 to 2026
$56.2M
Expanding National Capacity in PCOR through Training (ENACT)R25HS023185 · AHRQ · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI LANDSITTEL, DOUGLAS P · 2014 to 2018
$2.1M
AHRQ HHS R25 HS023185NCI NIH HHS P30 CA071789
6 · The paper itself

Abstract

purposeTo apply target trial emulation to explore the potential impact of eligibility criteria on the primary outcome of a randomized controlled trial.

methodsSimulations of a real-world explanatory trial of transarterial radioembolization for advanced unresectable hepatocellular carcinoma with portal vein invasion were performed to examine the effects of cohort specification on survival outcomes and patient sample size. Simulations comprised 24 different permutations of the trial varied on three disease nonspecific eligibility parameters. Treatment and control arms for these emulated trials were drawn from the National Cancer Database and matched by treatment propensity. Target trial emulation served as the causal framework for this analysis, allowing the architecture of a true controlled experiment to address forms of bias routinely encountered in comparative effectiveness studies involving real-world observational data.

resultsTwenty-four propensity score-matched cohorts comprising a wider clinical spectrum of patients than specified by the original target trial were successfully generated using the National Cancer Database. The arms for each of the emulated trials demonstrated exchangeability across all eligibility criteria and other clinical covariates. Significant treatment benefits were associated with only a narrow range of eligibility criteria, indicating that the original target trial was well specified.

conclusionThe impact of patient selection on treatment outcomes can be studied using target trial emulation. This analytical framework can furthermore serve to leverage existing real-world data to inform the task of cohort specification for a randomized controlled trial, facilitating a more data-driven approach for this important step in clinical trial design.

Indexed as

NeoplasmsRandomized Controlled Trials as TopicResearch DesignBiasHumansSample Size

Identifiers

PMID36608311
PMCPMC10166475
OpenAlexW4313639121

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.