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Learning from Conect4children: A Collaborative Approach towards Standardization of Disease-Specific Paediatric Research Data

Lookup NU author(s): Dr Anando SenORCiD, Victoria HedleyORCiD, Avril PalmeriORCiD, Joanne Lee, Sonia Segovia, Jessie Trueman, Becca LearyORCiD, Professor Volker StraubORCiD



This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


The conect4children (c4c) initiative was established to facilitate the development of new drugs and other therapies for paediatric patients. It is widely recognized that there are not enough medicines tested in all relevant ages of the paediatric population. To overcome this, it is imperative that clinical data from different sources are interoperable and can be pooled for larger post-hoc studies. c4c has collaborated with the Clinical Data Interchange Standards Consortium (CDISC) to develop the cross-cutting data resources that build on existing CDISC standards, in an effort to standardize paediatric data. The natural next step was an extension to disease-specific data items. c4c brought together several existing initiatives and resources relevant to disease-specific data and to analyse their use for standardizing disease-specific data in clinical trials. Several case studies that combined disease-specific data from multiple trials have demonstrated the need for disease-specific data standardization. We identified three relevant initiatives. These include European Reference Networks, European Joint Programme on Rare Diseases, and Pistoia Alliance. Other resources reviewed were: National Cancer Institute Enterprise Vocabulary Services, CDISC standards, pharmaceutical company-specific data dictionaries, Human Phenotype Ontology, Phenopackets, Unified Registry for Inherited Metabolic Disorders, Orphacodes, Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) and Observational Medical Outcomes Partnership. The collaborative partners associated with these resources were also reviewed briefly. A plan of action focussed on collaboration was generated for standardizing disease-specific paediatric clinical trial data. A paediatric data standards multistakeholder and multi-project user group was established to guide the remaining actions– FAIRification of metadata, a Phenopackets pilot with RDCA-DAP, applying Orphacodes to case report forms of clinical trials, introducing CDISC standards into European Reference Networks, testing of the CDISC Pediatric User Guide using data from the mentioned resources and organization of further workshops and educational materials.

Publication metadata

Author(s): Sen A, Hedley V, Degraeuwe E, Hirschfeld S, Cornet R, Walls R, Owen J, Robinson PN, Neilan EG, Liener T, Nisato G, Modi N, Woodworth S, Palmeri A, Gaentzsch R, Walsh M, Berkery T, Lee J, Persijn L, Baker K, An Haack K, Segovia Simon S, Jacobsen J, Reggiardo G, Kirwin M, Trueman J, Pansieri C, Bonifazi D, Nally S, Bonifazi F, Leary R, Straub V

Publication type: Article

Publication status: Published

Journal: Data

Year: 2024

Volume: 9

Issue: 4

Online publication date: 08/04/2024

Acceptance date: 27/03/2024

Date deposited: 11/04/2024

ISSN (electronic): 2306-5729

Publisher: MDPI


DOI: 10.3390/data904005


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Funder referenceFunder name
777389IMI Joint Undertaking
European Union Horizon 2020