Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children. Issue 3 (12th December 2022)

• Record Type: Journal Article
• Authors: Cheng, Katharine; Mahler, Fenna; Lutsar, Irja; Nafria Escalera, Begonya; Breitenstein, Stefanie; Vassal, Gilles; Claverol, Joana; Noel Palacio, Nuria; Portman, Ron; Pope, Gavin; Bakker, Martijn; van der Geest, Tessa; Turner, Mark A.; de Wildt, Saskia N.
• Other Names: Lanzerath Dirk investigator.; Tuleu Catherine investigator.; Schwab Matthias investigator.; Smits Anne investigator.; Treluyer Jean‐Marc investigator.; Moretti Francesco investigator.; Wong Ian investigator.; Roes Kit investigator.; Moreno Carmen investigator.; Allegaert Karel investigator.; Helbing Wim investigator.; Naumburg Estelle investigator.; de Hoog Matthijs investigator.; Metsvaht Tuuli investigator.; Danne Thomas investigator.; Scarpa Maurizio investigator.; Ruperto Nicola investigator.; Zaoutis Theoklis investigator.; Grigg Jonathan investigator.; Croft Nick investigator.; Schaeffer Franz investigator.; Zuddas Alessandro investigator.; Straub Volker investigator.; Bont Louis investigator.; Cross Helen investigator.
• Is Part Of: Clinical and translational science. Volume 16:Issue 3(2023)
• Page Start: 478
• Page End: 488
• Publication Date: 2022-12-12
• Subjects: Medicine, Experimental -- Periodicals; Medical innovations -- Periodicals; 616.027
• DOI: 10.1111/cts.13459
• Languages: English
• ISSNs: 1752-8054

Abstract

Abstract: Many medicines are used "off‐label" in children outside the terms of the license. Feasible pediatric clinical trials are a challenge to design. Conect4children (c4c) is an Innovative Medicines Initiative project to set up a pan‐European pediatric clinical trial network aiming to facilitate the development of new medicines for children. To optimize pediatric trial development by promoting innovative trial design, c4c set up a European multidisciplinary advice service, including the voice of young patients and families, tailored to industry and academia. A network of experts was established to provide multidisciplinary advice to trial sponsors. Experts were selected to join clinical and innovative methodology expert groups. A patient and public involvement (PPI) database, to include the expert opinion of patients and parents/carers was formed. A stepwise process was developed: (1) sponsors contact c4c, (2) scoping interview takes place, (3) ad hoc advice group formed, (5) advice meeting held, and (6) advice report provided. Feedback on the process was collected. Twenty‐four clinical and innovative methodology expert groups (>400 experts) and a PPI database of 135 registrants were established. As of September 30, 2022, 36 advice requests were received, with 25 requests completed. Clinical and methodology experts and PPI representatives participated in several advice requests. Sponsors appreciated the advice quality and the multidisciplinary experts from different countries, including experts not known before. Experts and PPI participants were generally satisfied with the process. The c4c project has shown successful proof of concept for a service that presents a new framework to plan innovative and feasible pediatric trials.