UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

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

Cheng, K; Mahler, F; Lutsar, I; Nafria Escalera, B; Breitenstein, S; Vassal, G; Claverol, J; ... Cross, H; + view all (2022) Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children. Clinical and Translational Science 10.1111/cts.13459. (In press). Green open access

[thumbnail of Clinical Translational Sci - 2022 - Cheng - Clinical  methodology  and patient carer expert advice in pediatric drug.pdf]
Preview
PDF
Clinical Translational Sci - 2022 - Cheng - Clinical methodology and patient carer expert advice in pediatric drug.pdf - Published Version

Download (432kB) | Preview

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.

Type: Article
Title: Clinical, methodology, and patient/carer expert advice in pediatric drug development by conect4children
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/cts.13459
Publisher version: https://doi.org/10.1111/cts.13459
Language: English
Additional information: © 2022 The Authors. Clinical and Translational Science published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License (http://creativecommons.org/licenses/by-nc-nd/4.0/).
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy > Pharmaceutics
URI: https://discovery.ucl.ac.uk/id/eprint/10162521
Downloads since deposit
43Downloads
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item