Partnership of I-ACT for children (US) and European pediatric clinical trial networks to facilitate pediatric clinical trials

Eva Degraeuwe; Collin Hovinga; Annelies De Maré; Ricardo M Fernandes; Callie Heaton; Lieve Nuytinck; Laura Persijn; Ann Raes; Johan Vande Walle; Mark A Turner

doi:10.3389/fped.2024.1388170

Partnership of I-ACT for children (US) and European pediatric clinical trial networks to facilitate pediatric clinical trials

Front Pediatr. 2024 Nov 7:12:1388170. doi: 10.3389/fped.2024.1388170. eCollection 2024.

Authors

Eva Degraeuwe¹, Collin Hovinga², Annelies De Maré³, Ricardo M Fernandes^{4

5}, Callie Heaton⁶, Lieve Nuytinck¹, Laura Persijn^{1

3}, Ann Raes^{1

3}, Johan Vande Walle^{1

3}, Mark A Turner^{4

7}

Affiliations

¹ Department of Internal Medicine and Pediatrics, Ghent University, Ghent, Belgium.
² Critical Path Institute, Tucson, AZ, United States.
³ The Department of Pediatrics, University Hospital of Ghent, ERKNET Centre, Ghent, Belgium.
⁴ conect4children Stichting, Utrecht, Netherlands.
⁵ Laboratory of Clinical Pharmacology and Therapeutics, Faculty of Medicine, University of Lisbon, Lisbon, Portugal.
⁶ Institute for Advanced Clinical Trials for Children, Rockville, MD, United States.
⁷ Institute of Life Course and Medical Sciences, University of Liverpool, Liverpool, United Kingdom.

Abstract

Background/aims: Due to a lack of standard pediatric prescribing information, medicines are often used in a dosage form or for an indication that has not been investigated in children. Pediatric clinical trial research networks aim to facilitate the timely availability of innovative drugs for children by developing standardized trial facilitation and conduct processes. This paper aims to assess the (pre)feasibility duration and characteristics of a US-sponsored clinical trial, in collaboration with I-ACT for Children, for distribution across European sites via European clinical research facilitation networks.

Method: A transatlantic partnership between the Belgian Pediatric Clinical Research Network (BPCRN,) and I-ACT for Children conducted feasibilities in Europe for industry-sponsored early-stage pharmacological clinical trials between 2019 and 2022. The collaboration recorded time to event for key elements of feasibility, influences on successful feasibility, and benefits of collaboration.

Results: Trials were conducted across 17 European countries with 202 participating hospital sites. The initial phase, the pre-feasibility questionnaire had a 70% response rate from 142 sites, and sites took a median 38 days (IQR 20 days) to complete the questionnaire for five trials. All responses underwent a quality control, addressing inaccuracies in site capabilities and recruitment. The first trial's CDA and feasibility questionnaire were completed in roughly 2 months for 7 countries. Time to completion was affected by precontracted sites, limited scope of studies, changes in timelines, COVID-related disruptions, and a learning curve for collaboration.

Conclusion: Collaboration between European collaborative national networks and US-network I-ACT for Children has supported site identification of global pediatric clinical trials. This illustrates one method for the importance of early engagement with sponsors and implementation of effective communication systems.

Keywords: drug development; global; metrics; networks; pediatric.

Grants and funding

The author(s) declare that financial support was received for the research, authorship, and/or publication of this article. I-ACT for Children is supported through a grant (U18 FD006297-05) funded by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS). The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government. The c4c project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 777389. The Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA. The Collaborative Research Networks involved are part of the consortium, yet activities of trial facilitation as a subcontractor for I-ACT for children is not included within the scope of the grant. Therefore, BPCRN was appointed as liaison between I-ACT for Children and the Collaborative Research Network for the above activities without grant support.