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, 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
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.