EClinicalMedicine (Jan 2024)

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,
  • Adrian Sánchez-Montalva,
  • Ana Belén Estevez,
  • Àlex Sánchez,
  • Anna Sanjuan,
  • Elena Sena,
  • Emma Granados,
  • Esther Arévalo de Andrés,
  • Fátima Nuñez,
  • Gara Arteaga,
  • Gabriela Perez Fuentes Ruiz,
  • Guillermo Fernández,
  • Jesus Rivera-Esteban,
  • Joan Comella,
  • Josep Antoni Ramos-Quiroga,
  • Joan Genescà,
  • Juan Espinosa,
  • Juan Manuel Pericàs,
  • Lada Murcia,
  • Lucinda Cash-Gibson,
  • Maria de Valles Silvosa,
  • María Fernanda Barroso de Sousa,
  • Olga Sánchez-Maroto Carrizo,
  • Pol Ibañez-Jiménez,
  • Salvador Augustin,
  • Santiago Perez-Hoyos,
  • Sarai Rodríguez-Navarro,
  • Sergio Muñoz-Martínez,
  • Silvia Serres,
  • Susana Kalko,
  • Amelie Michon,
  • Anton Ussi,
  • Ben Lydall,
  • Edwin van de Ketterij,
  • Ignacio Quiles,
  • Tamara Carapina,
  • Constantin Kumaus,
  • Dariga Ramazanova,
  • Elias Laurin Meyer,
  • Franz Koenig,
  • Marta Bofill Roig,
  • Martin Brunner,
  • Martin Posch,
  • Pavla Krotka,
  • Sonja Zehetmayer,
  • Charlotte Carton,
  • Eric Legius,
  • Amina Begum,
  • Carmine Pariante,
  • Courtney Worrell,
  • Giulia Lombardo,
  • Luca Sforzini,
  • Mollie Brown,
  • Nancy Gullet,
  • Nare Amasi-Hartoonian,
  • Rosalie Ferner,
  • Melisa Kose,
  • Andrea Spitaleri,
  • Arash Ghodousi,
  • Clelia Di Serio,
  • Daniela Cirillo,
  • Federica Cugnata,
  • Francesca Saluzzo,
  • Francesco Benedetti,
  • Maria Giovanna Scarale,
  • Michela Zini,
  • Paola Maria Rancoita,
  • Riccardo Alagna,
  • Sara Poletti,
  • Britt Dhaenens,
  • Johan Van Der Lei,
  • Jurriaan de Steenwinkel,
  • Maxim Moinat,
  • Rianne Oostenbrink,
  • Witte Hoogendijk,
  • Michael Hölscher,
  • Norbert Heinrich,
  • Christian Otte,
  • Cornelia Potratz,
  • Dario Zocholl,
  • Eugenia Kulakova,
  • Frank Tacke,
  • Jelena Brasanac,
  • Jonas Leubner,
  • Maja Krajewska,
  • Michaela Maria Freitag,
  • Stefan Gold,
  • Thomas Zoller,
  • Woo Ri Chae,
  • Christel Daniel,
  • Leila Kara,
  • Morgan Vaterkowski,
  • Nicolas Griffon,
  • Pierre Wolkenstein,
  • Raluca Pais,
  • Vlad Ratziu,
  • David Voets,
  • Christophe Maes,
  • Dipak Kalra,
  • Geert Thienpoint,
  • Jens Deckerck,
  • Nathan Lea,
  • Peter Singleton,
  • Kert Viele,
  • Peter Jacko,
  • Scott Berry,
  • Tom Parke,
  • Amelie Michon,
  • Burç Aydin,
  • Christine Kubiak,
  • Jacques Demotes,
  • Keiko Ueda,
  • Mihaela Matei,
  • Sergio Contrino,
  • Claas Röhl,
  • Estefania Cordero,
  • Fiona Greenhalgh,
  • Hannes Jarke,
  • Juliana Angelova,
  • Mathieu Boudes,
  • Stephan Dressler,
  • Valentina Strammiello,
  • Quentin Anstee,
  • Iñaki Gutierrez-Ibarluzea,
  • Maximilian Otte,
  • Natalie Heimbach,
  • Benjamin Hofner,
  • Cora Burgwinkel,
  • Hue Kaestel,
  • Katharina Hees,
  • Quynh Nguyen,
  • Daniel Prieto-Alhambra,
  • Eng Hooi (Cheryl) Tan,
  • Mario Raviglione,
  • Pierpaolo de Colombani,
  • Simone Villa,
  • Eduard Maron,
  • Gareth Evans,
  • Adam J. Savitz,
  • Ann Van Dessel,
  • Anna Duca,
  • Anne Kaminski,
  • Bie Wouters,
  • Brandon Porter,
  • Catherine Charron,
  • Cecile Spiertz,
  • Christopher Zizzamia,
  • Daniel Millar,
  • Danny Hasselbaink,
  • David Orr,
  • Divya Kesters,
  • Ellen Hubin,
  • Emma Davies,
  • Eva-Maria Didden,
  • Gabriela Guz,
  • Evelyn Verstraete,
  • Gary Mao,
  • George Capuano,
  • Heddie Martynowicz,
  • Heidi De Smedt,
  • Ingela Larsson,
  • Ines Bruegelmans,
  • Isabelle Coste,
  • Jesus Maria Gonzalez Moreno,
  • Julia Niewczas,
  • Jiajun Xu,
  • Karin Rombouts,
  • Katherine Woo,
  • Kathleen Wuyts,
  • Kathryn Hersh,
  • Khrista Oldenburg,
  • Lingjiao Zhang,
  • Mark Schmidt,
  • Mark Szuch,
  • Marija Todorovic,
  • Maartje Mangelaars,
  • Melissa Grewal,
  • Molli Sandor,
  • Nick Di Prospero,
  • Pamela Van Houten,
  • Pansy Minnick,
  • Polyana Bastos,
  • Robert Patrizi,
  • Salvatore Morello,
  • Severijn De Wilde,
  • Tao Sun,
  • Timothy Kline,
  • Tine de Marez,
  • Tobias Mielke,
  • Tom Reijns,
  • Vanina Popova,
  • Yanina Flossbach,
  • Yevgen Tymofyeyev,
  • Zeger De Groote,
  • Alex Sverdlov,
  • Alexandra Bobirca,
  • Annekatrin Krause,
  • Catalin Bobrica,
  • Daniela Heintz,
  • Dominic Magirr,
  • Ekkehard Glimm,
  • Fabienne Baffert,
  • Federica Castiglione,
  • Franca Caruso,
  • Francesco Patalano,
  • Frank Bretz,
  • Guenter Heimann,
  • Ian Carbarns,
  • Ignacio Rodríguez,
  • Ioana Ratescu,
  • Lisa Hampson,
  • Marcos Pedrosa,
  • Mareile Hark,
  • Peter Mesenbrink,
  • Sabina Hernandez Penna,
  • Sarah Bergues-Lang,
  • Susanne Baltes-Engler,
  • Tasneem Arsiwala,
  • Valeria Jordan Mondragon,
  • Hua Guo,
  • Jose Leite Da Costa,
  • Carl-Fredrik Burman,
  • George Kirk,
  • Anders Aaes-Jørgensen,
  • Jorgen Dirach,
  • Mette Skalshøi Kjær,
  • Alexandra Martin,
  • Diyan Hristov,
  • Florent Rousseaux,
  • Norbert Hittel,
  • Robert Dornheim,
  • Daniel Evans,
  • Nick Sykes,
  • Camille Couvert,
  • Catherine Leuven,
  • Loïc Notelet,
  • Madhavi Gidh-Jain,
  • Mathieu Jouannin,
  • Nadir Ammour,
  • Suzanne Pierre,
  • Volker Haufe,
  • Yingwen Dong,
  • Catherine Dubanchet,
  • Nathalie de Préville,
  • Tania Baltauss,
  • Zhu Jian,
  • Sara Shnider,
  • Tal Bar-El,
  • Annette Bakker,
  • Marco Nievo,
  • Uche Iloeje,
  • Almari Conradie,
  • Ece Auffarrth,
  • Leandra Lombard,
  • Majda Benhayoun,
  • Morounfolu Olugbosi,
  • Stephanie S. Seidel,
  • Berta Gumí,
  • Claudia García Guzmán,
  • Eva Molero,
  • Gisela Pairó,
  • Núria Machin,
  • Raimon Cardelús,
  • Saira Ramasastry,
  • Saskia Pelzer,
  • Andreas Kremer,
  • Erno Lindfors,
  • Chris Lynch

Journal volume & issue
Vol. 67
p. 102384

Abstract

Read online

Summary: 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.

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