Evidence map›Paper›PMID 37689623›Full record

ArticleBMC medical research methodology2023

Using public clinical trial reports to probe non-experimental causal inference methods.

Ethan Steinberg, Nikolaos Ignatiadis, Steve Yadlowsky, Yizhe Xu, Nigam Shah

Abstract read
In one paragraph

Article in BMC medical research methodology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

5 authors.

Ethan SteinbergCenter for Biomedical Informatics Research, Stanford University, Stanford, US. ethanid@stanford.edu.
Nikolaos IgnatiadisDepartment of Statistics, University of Chicago, Chicago, US.
Steve YadlowskyGoogle Research, Google, Cambridge, US.
Yizhe XuCenter for Biomedical Informatics Research, Stanford University, Stanford, US.
Nigam ShahCenter for Biomedical Informatics Research, Stanford University, Stanford, US.

Funding

Methods for generalized ontology terms enrichment analysisR01LM011369 · NLM · STANFORD UNIVERSITY · PI SHAH, NIGAM H · 2013 to 2020
$5.0M
NLM NIH HHS R01 LM011369
6 · The paper itself

Abstract

backgroundNon-experimental studies (also known as observational studies) are valuable for estimating the effects of various medical interventions, but are notoriously difficult to evaluate because the methods used in non-experimental studies require untestable assumptions. This lack of intrinsic verifiability makes it difficult both to compare different non-experimental study methods and to trust the results of any particular non-experimental study.

methodsWe introduce TrialProbe, a data resource and statistical framework for the evaluation of non-experimental methods. We first collect a dataset of pseudo "ground truths" about the relative effects of drugs by using empirical Bayesian techniques to analyze adverse events recorded in public clinical trial reports. We then develop a framework for evaluating non-experimental methods against that ground truth by measuring concordance between the non-experimental effect estimates and the estimates derived from clinical trials. As a demonstration of our approach, we also perform an example methods evaluation between propensity score matching, inverse propensity score weighting, and an unadjusted approach on a large national insurance claims dataset.

resultsFrom the 33,701 clinical trial records in our version of the ClinicalTrials.gov dataset, we are able to extract 12,967 unique drug/drug adverse event comparisons to form a ground truth set. During our corresponding methods evaluation, we are able to use that reference set to demonstrate that both propensity score matching and inverse propensity score weighting can produce estimates that have high concordance with clinical trial results and substantially outperform an unadjusted baseline.

conclusionsWe find that TrialProbe is an effective approach for probing non-experimental study methods, being able to generate large ground truth sets that are able to distinguish how well non-experimental methods perform in real world observational data.

Indexed as

Research DesignBayes TheoremCausalityHumansPropensity ScoreCausal inferenceClinical trialsMeta-analysisMethod evaluation

Identifiers

PMID37689623
PMCPMC10492298

What OpenQuestion holds

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LicenceCC BY
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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.