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. 2023 Mar;16(3):478-488.
doi: 10.1111/cts.13459. Epub 2022 Dec 12.

Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children

Katharine Cheng  1 , Fenna Mahler  2 , Irja Lutsar  3 , Begonya Nafria Escalera  4 , Stefanie Breitenstein  5 , Gilles Vassal  6 , Joana Claverol  4 , Nuria Noel Palacio  4 , Ron Portman  7 , Gavin Pope  8 , Martijn Bakker  9 , Tessa van der Geest  2 , Mark A Turner  10 , Saskia N de Wildt  2 ; c4c Expert Group Leads
Collaborators, Affiliations

Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children

Katharine Cheng et al. Clin Transl Sci. 2023 Mar.

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.

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Conflict of interest statement

K.C. is an employee of Johnson & Johnson, UK. S.B. is an employee of Bayer, Germany. R.P. is an employee of Novartis, USA. G.P. is an employee of Novartis, Switzerland. All other authors declared no competing interests for this work.

Figures

FIGURE 1
FIGURE 1
Roadmap for advice. HTA, Health Technology Assessment; PIP, pediatric investigation plan
FIGURE 2
FIGURE 2
Outline of c4c strategic feasibility advice process
FIGURE 3
FIGURE 3
Feedback from advice requesters and experts
FIGURE 4
FIGURE 4
Process map. c4c, conect4children
See this image and copyright information in PMC

References

    1. European Commission . State of Paediatric Medicines in the EU‐10 years of the EU Paediatric Regulation. 1–17. Accessed May 23, 2022. https://ec.europa.eu/health/system/files/2017‐11/2017_childrensmedicines...
    1. Toma M, Felisi M, Bonifazi D, et al. Paediatric medicines in Europe: the Paediatric regulation‐is it time for reform? Front Med (Lausanne). 2021;8:593281. - PMC - PubMed
    1. McCune S, Portman RJ. Innovation and opportunities in pediatric therapeutic development. Ther Innov Regul Sci. 2019;53(5):564‐566. - PMC - PubMed
    1. c4c . conect4children. Accessed May 23, 2022. https://conect4children.org/
    1. I‐ACT . Institute for advanced clinical trials for children. Accessed May 23, 2022. https://www.iactc.org/

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