Development of the CDISC Pediatrics User Guide: a CDISC and conect4children collaboration

John Owen; Anando Sen; Beate Aurich; Corinna Engel; Giacomo Cavallaro; Eva Degraeuwe; Dipak Kalra; Ronald Cornet; Melissa Walsh; Teresa Berkery; Avril Palmeri; Fenna Mahler; Salma Malik; Laura Persijn; Chima Amadi; Jacques Thuet; Simon Woodworth; Sinead Nally; Rebecca Leary; Richard Marshall; Volker Straub

doi:10.3389/fmed.2024.1370916

Development of the CDISC Pediatrics User Guide: a CDISC and conect4children collaboration

Front Med (Lausanne). 2024 Jun 17:11:1370916. doi: 10.3389/fmed.2024.1370916. eCollection 2024.

Authors

John Owen^#¹, Anando Sen^#², Beate Aurich³, Corinna Engel⁴, Giacomo Cavallaro⁵, Eva Degraeuwe^{6

7}, Dipak Kalra⁸, Ronald Cornet^{9

10}, Melissa Walsh¹¹, Teresa Berkery¹¹, Avril Palmeri², Fenna Mahler¹², Salma Malik¹³, Laura Persijn^{6

7

14}, Chima Amadi², Jacques Thuet¹⁵, Simon Woodworth¹¹, Sinead Nally¹⁶, Rebecca Leary², Richard Marshall^{1

17}, Volker Straub²

Affiliations

¹ CDISC Europe Foundation, Brussels, Belgium.
² John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
³ Drug Safety Consulting S.A.S.U., Paris, France.
⁴ Center for Pediatric Clinical Studies, University Children's Hospital Tübingen, Tuebingen, Germany.
⁵ NICU, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
⁶ Departement of Internal Medicine and Pediatrics, Ghent University, Ghent, Belgium.
⁷ Ghent University Hospital (UZ Gent), Ghent, Belgium.
⁸ The European Institute for Innovation through Health Data, Ghent, Belgium.
⁹ Department of Medical Informatics, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, Netherlands.
¹⁰ Amsterdam Public Health, Digital Health and Methodology, Amsterdam, Netherlands.
¹¹ INFANT Research Centre, University College Cork, Cork, Ireland.
¹² Department of Pharmacy, Division Pharmacology and Toxicology, Radboud University Medical Center, Nijmegen, Netherlands.
¹³ The European Clinical Research Infrastructure Network, Paris, France.
¹⁴ Belgian Paediatric Clinical Research Network (BPCRN), Ghent University, Ghent, Belgium.
¹⁵ Institut de Recherches Internationales Servier, Suresnes, France.
¹⁶ Novartis Pharmaceuticals, Dublin, Ireland.
¹⁷ Accurate Systems Limited, Saffron Walden, United Kingdom.

^# Contributed equally.

Abstract

Introduction: The conect4children (c4c) project aims to facilitate efficient planning and delivery of paediatric clinical trials. One objective of c4c is data standardization and reuse. Interoperability and reusability of paediatric clinical trial data is challenging due to a lack of standardization. The Clinical Data Interchange Standards Consortium (CDISC) standards that are required or recommended for regulatory submissions in several countries lack paediatric specificity with limited awareness within academic institutions. To address this, c4c and CDISC collaborated to develop the Pediatrics User Guide (PUG) consisting of cross-cutting data items that are routinely collected in paediatric clinical trials, factoring in all paediatric age ranges.

Methods and results: The development of the PUG consisted of six stages. During the scoping phase, subtopics (each containing several clinically relevant concepts) were suggested and debated for inclusion in the PUG. Ninety concepts were selected for the modelling phase. Concept maps describing the Research Topic and representation procedure were developed for the 19 concepts that had no (or partial) previous modelling in CDISC. Next, metadata and implementation examples were developed for concepts. This was followed by a CDISC internal review and a public review. For both these review stages, the feedback comments were either implemented or rejected based on budget, timelines, expert review, and scope. The PUG was published on the CDISC website on February 23, 2023.

Discussion: The PUG is a first step in bridging the lack of child specific CDISC standards, particularly within academia. Several academic and industrial partners were involved in the development of the PUG, and c4c has undertaken multiple steps to publicize the PUG within its academic partner organizations - in particular, the European Reference Networks (ERNs) that are developing registries and dictionaries in 24 disease areas. In the long term, continued use of the PUG in paediatric clinical trials will enable the pooling of data from multiple trials, which is particularly important for medical domains with small populations.

Keywords: CDISC; data dictionary; data standards; interoperability; paediatric clinical trials.

Grants and funding

The author(s) declare financial support was received for the research, authorship, and/or publication of this article. This 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. AS, AP, RL, and VS are supported by the NIHR Newcastle Biomedical Research Centre (BRC). The NIHR Newcastle Biomedical Research Centre (BRC) is a partnership between Newcastle Hospitals NHS Foundation Trust and Newcastle University, funded by the National Institute for Health and Care Research (NIHR).