Evidence map›Paper›PMID 36708450›Full record

ArticleLifetime data analysis2023

Investigating non-inferiority or equivalence in time-to-event data under non-proportional hazards.

Kathrin Möllenhoff, Achim Tresch

Abstract read
In one paragraph

Article in Lifetime data analysis, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 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

2 authors.

Kathrin MöllenhoffMathematical Institute, Heinrich Heine University, 40225, Düsseldorf, Germany. kathrin.moellenhoff@hhu.de.ORCID 0000-0001-7861-3892
Achim TreschInstitute of Medical Statistics and Computational Biology, Faculty of Medicine, University of Cologne, Cologne, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The classical approach to analyze time-to-event data, e.g. in clinical trials, is to fit Kaplan-Meier curves yielding the treatment effect as the hazard ratio between treatment groups. Afterwards, a log-rank test is commonly performed to investigate whether there is a difference in survival or, depending on additional covariates, a Cox proportional hazard model is used. However, in numerous trials these approaches fail due to the presence of non-proportional hazards, resulting in difficulties of interpreting the hazard ratio and a loss of power. When considering equivalence or non-inferiority trials, the commonly performed log-rank based tests are similarly affected by a violation of this assumption. Here we propose a parametric framework to assess equivalence or non-inferiority for survival data. We derive pointwise confidence bands for both, the hazard ratio and the difference of the survival curves. Further we propose a test procedure addressing non-inferiority and equivalence by directly comparing the survival functions at certain time points or over an entire range of time. Once the model's suitability is proven the method provides a noticeable power benefit, irrespectively of the shape of the hazard ratio. On the other hand, model selection should be carried out carefully as misspecification may cause type I error inflation in some situations. We investigate the robustness and demonstrate the advantages and disadvantages of the proposed methods by means of a simulation study. Finally, we demonstrate the validity of the methods by a clinical trial example.

Indexed as

Research DesignComputer SimulationHumansProportional Hazards ModelsSample SizeSurvival AnalysisTime FactorsEquivalenceNon-inferiorityNon-proportional hazardsSurvival analysisTime-to-event data

Identifiers

PMID36708450
PMCPMC10258187

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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.