Evidence map›Paper›PMID 38233102›Full record

ArticlePharmaceutical statistics

Transporting randomized trial results to estimate counterfactual survival functions in target populations.

Zhiqiang Cao, Youngjoo Cho, Fan Li

Open access · greenAbstract read
In one paragraph

Article in Pharmaceutical statistics. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
2.1field-weighted citation impact, top 15% 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

2 citing papers in PubMed, 3 citations in OpenAlex.

  1. Article
  2. 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 3 institutions in 3 countries.

Zhiqiang CaoDepartment of Mathematics, College of Big Data and Internet, Shenzhen Technology University, Shenzhen, People's Republic of China.
Youngjoo ChoDepartment of Applied Statistics, Konkuk University, Seoul, Republic of Korea.
Fan LiDepartment of Biostatistics, Yale University School of Public Health, New Haven, Connecticut, USA.ORCID 0000-0001-6183-1893
Konkuk University · KRShenzhen Technology University · CNYale University · US

Funding

Yale Clinical and Translational Science Award (U Component)UL1TR001863 · NCATS · YALE UNIVERSITY · PI John H. Krystal, LUCILA OHNO-MACHADO · 2016 to 2026
$102.9M
NCATS NIH HHS UL1 TR001863
6 · The paper itself

Abstract

When the distributions of treatment effect modifiers differ between a randomized trial and an external target population, the sample average treatment effect in the trial may be substantially different from the target population average treatment, and accurate estimation of the latter requires adjusting for the differential distribution of effect modifiers. Despite the increasingly rich literature on transportability, little attention has been devoted to methods for transporting trial results to estimate counterfactual survival functions in target populations, when the primary outcome is time to event and subject to right censoring. In this article, we study inverse probability weighting and doubly robust estimators to estimate counterfactual survival functions and the target average survival treatment effect in the target population, and provide their respective approximate variance estimators. We focus on a common scenario where the target population information is observed only through a complex survey, and elucidate how the survey weights can be incorporated into each estimator we considered. Simulation studies are conducted to examine the finite-sample performances of the proposed estimators in terms of bias, efficiency and coverage, under both correct and incorrect model specifications. Finally, we apply the proposed method to assess transportability of the results in the Action to Control Cardiovascular Risk in Diabetes-Blood Pressure (ACCORD-BP) trial to all adults with Diabetes in the United States.

Indexed as

Randomized Controlled Trials as TopicBiasCardiovascular DiseasesComputer SimulationData Interpretation, StatisticalHumansModels, StatisticalResearch DesignSurvival Analysisinverse odds of sampling weightinginverse probability of censoring weightingsurvey samplesurvival analysistransportability

Identifiers

PMID38233102
PMCPMC13005602
OpenAlexW4390989062

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.