Evidence mapPaperPMID 41047965Full record

ReviewJournal of comparative effectiveness research2025

Estimating per-protocol effects in external comparator analyses using real-world data.

Alind Gupta, Evie Merinopoulou, Stephen J Duffield, Manuel Gomes, Seamus Kent, Nicolas Scheuer, Gerardo Machnicki, Thibaut Sanglier

Abstract readReview
In one paragraph

Review in Journal of comparative effectiveness research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

8 authors.

Alind GuptaFifty1 AI LabsVancouver, British Columbia. V6H 3Y4 Canada.ORCID 0000-0003-1139-9769
Evie MerinopoulouCytel Inc., London, UK.
Stephen J DuffieldNational Institute for Health & Care Excellence, Manchester, UK.
Manuel GomesUniversity College London, Primary Care & Population Health, UK.ORCID 0000-0002-1428-1232
Seamus KentErasmus School of Health Policy & Management, Erasmus University Rotterdam, Netherlands.ORCID 0000-0001-7298-3163
Nicolas ScheuerRoche Products Ltd, Welwyn Garden City, UK.
Gerardo MachnickiF. Hoffmann-La Roche Ltd., Basel, Switzerland.ORCID 0000-0002-4696-605X
Thibaut SanglierF. Hoffmann-La Roche Ltd., Basel, Switzerland.ORCID 0000-0003-0740-2048

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Analysis of single arm trials complemented with external comparator arms (ECAs) may be used to support evidence of effectiveness of novel therapies in oncology research when a randomized trial is unavailable or unfeasible. However, the intention-to-treat effect, which is a common target of estimation in ECA studies, is difficult to interpret when there are differences in adherence between the trial and ECA. This paper describes an approach to estimation of per-protocol effects in ECA studies using the target trial emulation framework for study design and analysis based on the results from an exploratory case study (TBASEL). We highlight challenges, potential solutions and future opportunities from the perspectives of protocol specification, data suitability and analysis, to help guide future implementations of per-protocol effects in ECAs.

Indexed as

Intention to Treat AnalysisResearch DesignHumansRandomized Controlled Trials as Topicadherencecomparative effectivenessexternal controlsper-protocol effectstarget trial emulation

Identifiers

PMID41047965
PMCPMC12580923

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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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 Socratic graph.