Current state-of-the-art and gaps in platform trials : 10 things you should know, insights from EU-PEARL
Koenig, Franz (Medical University of Vienna)
Spiertz, Cécile (Janssen Biologics BV)
Millar, Daniel (Janssen Research & Development)
Rodríguez Navarro, Sarai (Hospital Universitari Vall d'Hebron)
Machín, Núria (TEAM-IT Research S.L)
Van Dessel, Ann (Janssen Pharmaceutica NV)
Genescà Ferrer, Joan (Universitat Autònoma de Barcelona. Departament de Medicina)
Pericàs, Juan M. (Universitat Autònoma de Barcelona. Departament de Medicina)
Posch, Martin (Medical University of Vienna)

Fecha:	2023
Resumen:	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.
Ayudas:	European Commission 853966
Derechos:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Lengua:	Anglès
Documento:	Article ; recerca ; Versió publicada
Materia:	Adaptive designs ; Master protocols ; Patient-centred ; Clinical research ; Integrated research platform
Publicado en:	EClinicalMedicine, Vol. 67 (December 2023) , art. 102384, ISSN 2589-5370

DOI: 10.1016/j.eclinm.2023.102384
PMID: 38226342

12 p, 392.9 KB

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