Evidence map›Paper›PMID 41669939›Full record

ArticleClinical pharmacology and therapeutics2026

Assessing Overall Survival Benefits in Advanced Cancers: The Role of External Comparator Cohort Studies with Real-World Data.

Francesco Pignatti, Tarec Christoffer El-Galaly, Martin Kaiser, Kimmo Porkka, Robin Doeswijk, Peter Mol, Donna R Rivera, Catherine C Lerro, Ulrich-Peter Rohr, Patrice Verpillat and 10 more

Abstract read
In one paragraph

Article in Clinical pharmacology and therapeutics, 2026. 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

20 authors.

Francesco PignattiThe European Medicines Agency, Amsterdam, The Netherlands.ORCID 0000-0001-9296-2474
Tarec Christoffer El-GalalyDepartments of Hematology, Clinical Epidemiology, and Molecular Medicine, Aarhus University Hospital and Aarhus University, Aarhus, Denmark.ORCID 0000-0002-4406-380X
Martin KaiserThe Royal Marsden NHS Foundation Trust and The Institute of Cancer Research, London, UK.ORCID 0000-0002-3677-4804
Kimmo PorkkaiCAN Digital Precision Cancer Medicine Flagship, University of Helsinki, and Helsinki University Hospital Comprehensive Cancer Center, Helsinki, Finland.ORCID 0000-0003-4112-5902
Robin DoeswijkThe European Haematology Association, Den Haag, The Netherlands.ORCID 0000-0002-5214-8889
Peter MolUniversity of Groningen, University Medical Centre Groningen, Groningen, The Netherlands.ORCID 0000-0002-4262-3180
Donna R RiveraOncology Center of Excellence at the U.S. Food and Drug Administration, Silver Spring, MD, USA.ORCID 0000-0002-4565-4556
Catherine C LerroOncology Center of Excellence at the U.S. Food and Drug Administration, Silver Spring, MD, USA.ORCID 0009-0009-1674-3822
Ulrich-Peter RohrSwiss Agency for Therapeutic Products, Bern, Switzerland.ORCID 0009-0002-3284-1739
Patrice VerpillatThe European Medicines Agency, Amsterdam, The Netherlands.ORCID 0000-0003-0010-1699
Antonios ValachisDepartment of Oncology, Faculty of Medicine and Health, Örebro University, Örebro, Sweden.ORCID 0000-0001-6059-0194
Dario TrapaniEuropean Institute of Oncology IRCCS, Milan, Italy.ORCID 0000-0003-1672-9560
Massimo Di MaioDepartment of Oncology, University of Turin, AOU Città della Salute e della Scienza di Torino, Torino, Italy.ORCID 0000-0001-8906-3785
Nicola LatinoScientific and Medical Division, European Society for Medical Oncology (ESMO), Lugano, Switzerland.ORCID 0000-0001-8225-8635
Raul CordobaFundación Jiménez Díaz University Hospital, Health Research Institute IIS-FJD, Madrid, Spain.ORCID 0000-0002-7654-8836
Nathan ChernyShaare Zedek Medical Center, Jerusalem, Israel.ORCID 0000-0003-1976-8952
Miriam KoopmanUniversity Medical Centre Utrecht, University of Utrecht, Utrecht, The Netherlands.ORCID 0000-0003-1550-1978
Diogo Martins-BrancoScientific and Medical Division, European Society for Medical Oncology (ESMO), Lugano, Switzerland.ORCID 0000-0002-7214-4818
George PentheroudakisScientific and Medical Division, European Society for Medical Oncology (ESMO), Lugano, Switzerland.ORCID 0000-0002-6632-2462
Douwe PostmusUniversity of Groningen, University Medical Centre Groningen, Groningen, The Netherlands.ORCID 0000-0002-9458-7038

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

External comparator cohort (ECC) studies with real-world data (RWD) may provide more reliable estimates of treatment differences compared to single-arm trials (SAT), yet they face limitations such as selection bias and data heterogeneity. This study assessed the perceived strength of evidence of ECC studies compared to SAT and randomized controlled studies (RCT). The study included healthcare professionals (HCP) from the European Hematology Association (EHA), the European Society for Medical Oncology (ESMO), and assessors from international regulatory agencies (RA). A conjoint analysis evaluated strength of evidence ratings for establishing an effect on OS for different hypothetical scenarios, based on different designs, RWD quality, and observed OS improvement, for a new cancer treatment for advanced disease and no effective treatments. Participants from HCP organizations rated RWD studies favorably (advantages outweigh disadvantages) more frequently (47.6%; n = 103) compared to RA participants (12.9%; n = 116). Compared to a SAT, a high-quality RWD ECC study showing a 1.5-month and 3-month OS improvement had 2.7 (95% CI: 1.9-3.8) and 14.7 (95% CI: 10.0-21.5) times higher odds of receiving a higher strength of evidence rating, respectively. The OR for RCT v. SAT was 36.4 (95% CI: 24.0-55.2) and 358.4 (95% CI: 217.3-591.3), respectively. Strength of evidence ratings were associated with maximum acceptable risk of severe or symptomatic toxicity. In conclusion, when evaluating the OS of new therapies, ECC studies with RWD, especially when based on high-quality RWD or demonstrating a larger OS benefit, were rated as more convincing than SAT without a formal control.

Indexed as

NeoplasmsResearch DesignCohort StudiesHumansRandomized Controlled Trials as TopicTreatment Outcome

Identifiers

PMID41669939
PMCPMC12997495

What Socratic holds

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LicenceCC BY-NC
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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 Socratic graph.