Evidence map›Paper›PMID 38226342›Full record

ReviewEClinicalMedicine2024

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL.

Franz Koenig, Cécile Spiertz, Daniel Millar, Sarai Rodríguez-Navarro, Núria Machín, Ann Van Dessel, Joan Genescà, Juan M Pericàs, Martin Posch, EU-PEARL Consortium

Abstract readReview
In one paragraph

Review in EClinicalMedicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed, 1 pooled it
–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

29 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. A European platform trial for major depressive disorder.World psychiatry : official journal of the World Psychiatric Association (WPA) · 2026
    Article
  3. Review
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Review
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  11. Evolution of Clinical Trial Design in ADPKD.Kidney international reports · 2026
    Review
  12. Review
  13. Review
  14. Article
  15. Promoting collaboration, harmonization and dissemination in depression research: the ECNP Depression Meta-Network.World psychiatry : official journal of the World Psychiatric Association (WPA) · 2025
    Article
  16. Article
  17. Article
  18. Article
  19. Review
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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

10 authors.

Franz KoenigMedical University of Vienna, Center for Medical Data Science, Vienna, Austria.
Cécile SpiertzJanssen Biologics BV, Leiden, the Netherlands.
Daniel MillarFormer Employee, Janssen Research & Development, LLC, Raritan, NJ, USA.
Sarai Rodríguez-NavarroVall d'Hebron Institute for Research, Barcelona, Spain.
Núria MachínTEAM-IT Research S.L., Barcelona, Spain.
Ann Van DesselJanssen Pharmaceutica NV, Beerse, Belgium.
Joan GenescàVall d'Hebron Institute for Research, Barcelona, Spain.
Juan M PericàsVall d'Hebron Institute for Research, Barcelona, Spain.
Martin PoschMedical University of Vienna, Center for Medical Data Science, Vienna, Austria.
EU-PEARL Consortium

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Platform trials bring the promise of making clinical research more efficient and more patient centric. While their use has become more widespread, including their prominent role during the COVID-19 pandemic response, broader adoption of platform trials has been limited by the lack of experience and tools to navigate the critical upfront planning required to launch such collaborative studies. The European Union-Patient-cEntric clinicAl tRial pLatform (EU-PEARL) initiative has produced new methodologies to expand the use of platform trials with an overarching infrastructure and services embedded into Integrated Research Platforms (IRPs), in collaboration with patient representatives and through consultation with U.S. Food and Drug Administration and European Medicines Agency stakeholders. In this narrative review, we discuss the outlook for platform trials in Europe, including challenges related to infrastructure, design, adaptations, data sharing and regulation. Documents derived from the EU-PEARL project, alongside a literature search including PubMed and relevant grey literature (e.g., guidance from regulatory agencies and health technology agencies) were used as sources for a multi-stage collaborative process through which the 10 more important points based on lessons drawn from the EU-PEARL project were developed and summarised as guidance for the setup of platform trials. We conclude that early involvement of critical stakeholder such as regulatory agencies or patients are critical steps in the implementation and later acceptance of platform trials. Addressing these gaps will be critical for attaining the full potential of platform trials for patients. Funding: Innovative Medicines Initiative 2 Joint Undertaking with support from the European Union's Horizon 2020 research and innovation programme and EFPIA.

Indexed as

Adaptive designsClinical researchIntegrated research platformMaster protocolsPatient-centred

Identifiers

PMID38226342
PMCPMC10788209

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.