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Research output: Contribution to Journal/Magazine › Review article › peer-review
Research output: Contribution to Journal/Magazine › Review article › peer-review
}
TY - JOUR
T1 - Current state-of-the-art and gaps in platform trials
T2 - 10 things you should know, insights from EU-PEARL
AU - EU-PEARL Consortium
AU - Koenig, Franz
AU - Spiertz, Cécile
AU - Millar, Daniel
AU - Rodríguez-Navarro, Sarai
AU - Machín, Núria
AU - Van Dessel, Ann
AU - Genescà, Joan
AU - Pericàs, Juan M.
AU - Posch, Martin
AU - Sánchez-Montalva, Adrian
AU - Estevez, Ana Belén
AU - Sánchez, Àlex
AU - Sanjuan, Anna
AU - Sena, Elena
AU - Granados, Emma
AU - Arévalo de Andrés, Esther
AU - Nuñez, Fátima
AU - Arteaga, Gara
AU - Fuentes Ruiz, Gabriela Perez
AU - Fernández, Guillermo
AU - Rivera-Esteban, Jesus
AU - Comella, Joan
AU - Ramos-Quiroga, Josep Antoni
AU - Espinosa, Juan
AU - Pericàs, Juan Manuel
AU - Murcia, Lada
AU - Cash-Gibson, Lucinda
AU - de Valles Silvosa, Maria
AU - Barroso de Sousa, María Fernanda
AU - Sánchez-Maroto Carrizo, Olga
AU - Ibañez-Jiménez, Pol
AU - Augustin, Salvador
AU - Perez-Hoyos, Santiago
AU - Muñoz-Martínez, Sergio
AU - Serres, Silvia
AU - Kalko, Susana
AU - Michon, Amelie
AU - Ussi, Anton
AU - Lydall, Ben
AU - van de Ketterij, Edwin
AU - Quiles, Ignacio
AU - Carapina, Tamara
AU - Kumaus, Constantin
AU - Ramazanova, Dariga
AU - Meyer, Elias Laurin
AU - Roig, Marta Bofill
AU - Jacko, Peter
AU - Magirr, Dominic
AU - Hampson, Lisa
AU - Evans, Daniel
PY - 2024/1/31
Y1 - 2024/1/31
N2 - 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.
AB - 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.
KW - Adaptive designs
KW - Clinical research
KW - Integrated research platform
KW - Master protocols
KW - Patient-centred
U2 - 10.1016/j.eclinm.2023.102384
DO - 10.1016/j.eclinm.2023.102384
M3 - Review article
AN - SCOPUS:85180956599
VL - 67
JO - eClinicalMedicine
JF - eClinicalMedicine
SN - 2589-5370
M1 - 102384
ER -