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

作者：Franz Koenig, Cécile Spiertz, Daniel Millar, Sarai Rodríguez, Núria Machín, Ann Van Dessel, Joan Genescà, Juan M. Pericàs, Martin Posch, Adrián Sánchez‐Montalvá, Ana Belén Estevez, Álex Sánchez‐Pla, A. McNulty Sanjuan, Elena Sena, Emma Granados, Esther Arévalo de Andrés, Fátima Nuñez, Gara Arteaga, Gabriela Perez Fuentes Ruiz, Guillermo Fernández, Jesús Rivera‐Esteban, Joan Comella, Josep Antoni Ramos‐Quiroga, Joan Genescà, Juan Felipe Espinosa‐Cristia, Juan M. Pericàs, Lada Murcia, Lucinda Cash‐Gibson, Maria de Valles Silvosa, María Fernanda Barroso de Sousa, Olga Sánchez-Maroto Carrizo, Pol Ibañez-Jiménez, Salvador Augustín, Santiago Pérez‐Hoyos, Sarai Rodríguez, Sergio Muñoz, S. Bezner Serres, Susana G. Kalko, Amelie Michon, Antón Ussi, Ben Lydall, Edwin van de Ketterij, Ignacio Quiles, Tamara Carapina, Constantin Kumaus, Д. М. Рамазанова, Elias Laurin Meyer, Franz Koenig, Marta Bofill Roig, Martin Brunner, Martin Posch, Pavla Krotka, Sonja Zehetmayer, Charlotte Carton, Eric Legius, Amina Begum, Carmine M. Pariante, Courtney Worrell, Giulia Lombardo, Luca Sforzini, Mollie Brown, Nancy Gullet, Nare Amasi-Hartoonian, Rosalie E. Ferner, Melisa Kose, Andrea Spitaleri, Arash Ghodousi, Clelia Di Serio, Daniela María Cirillo, Federica Cugnata, Francesca Saluzzo, Francesco Benedetti, Maria Giovanna Scarale, Michela Zini, Paola M. V. Rancoita, Riccardo Alagna, Sara Poletti, Britt A. E. Dhaenens, Johan van der Lei, Jurriaan de Steenwinkel, Maxim Moinat, Rianne Oostenbrink, Witte J.G. 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 M. Gold, Thomas Zöller, Woo Ri Chae, Christel Daniel, Leila Kara, Morgan Vaterkowski, Nicolas Griffon, P. 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‐Mainard, Keiko Ueda, Mihaela Matei, Sergio Contrino, Claas Röhl, E. Cordero, Fiona Greenhalgh, Hannes Jarke, Juliana Angelova, Mathieu Boudes, Stephan Dressler, Valentina Strammiello, Quentin M. Anstee, Iñaki Gutiérrez‐Ibarluzea, Maximilian Otte, Natalie Heimbach, Benjamin Hofner, Cora Burgwinkel, Hue Kaestel, Katharina Hees, Quynh Nguyen, Daniel Prieto‐Alhambra, Eng Hooi Tan, Mario Raviǵlione, Pierpaolo de Colombani, Simone Villa, Eduard Maron, D. Gareth Evans, Adam Savitz, Ann Van Dessel, Anna Duca, Anne Kaminski, Bie Wouters, Brandon Porter, Catherine E. Charron, Cécile Spiertz, Christopher Zizzamia, Daniel Millar, Danny M. Hasselbaink, David Orr, Divya Kesters, E.A. 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 E. Schmidt, Mark Szuch, Marija Todorović, 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, В. В. Попова, 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 López Rodríguez, Ioana Ratescu, Lisa V. Hampson, Marcos Pedrosa, Mareile Hark, Peter Mesenbrink, Sabina Hernandez Penna, Sarah Bergues-Lang, Susanne Baltes-Engler, Tasneem Arsiwala, Valeria Jordán Mondragón, 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 D. 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 J. Shnider, Tal Bar-El, Annette Bakker, Marco Nievo, Uche Iloeje, Almari Conradie, Ece Auffarrth, Leandra Lombard, Majda Benhayoun, Morounfolu Olugbosi, Stephanie Seidel, Berta Gumí, Claudia García Guzmán, Eva Molero, Gisela Pairó, Núria Machín, Raimon Cardelús, Saira Ramasastry, Saskia Pelzer, Andreas Kremer, Erno Lindfors, C. Lynch · 发表于：EClinicalMedicine · 年份：2023 · DOI：10.1016/j.eclinm.2023.102384 · 被引用次数：45 · 研究领域：Statistical Methods in Clinical Trials、Meta-analysis and systematic reviews、Health Systems, Economic Evaluations, Quality of Life

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 ar...