Learning from conect4children: A Collaborative Approach towards Standardisation of Disease-Specific Paediatric Research Data

Anando Sen, Victoria Hedley, Eva Degraeuwe, Steven Hirschfeld, Ronald Cornet, Ramona Walls, John Owen, Peter N. Robinson, Edward G. Neilan, Thomas Liener, Giovanni Nisato, Neena Modi, Simon Woodworth, Avril Palmeri, Ricarda Gaentzsch, Melissa Walsh, Teresa Berkery, Joanne Lee, Laura Persijn, Kasey Baker, Kristina An Haack, Sonia Segovia Simon, Julius O. B. Jacobsen, Giorgio Reggiardo, Melissa A. Kirwin, Jessie Trueman, Claudia Pansieri, Donato Bonifazi, Sinéad Nally, Fedele Bonifazi, Rebecca Leary () and Volker Straub
Additional contact information
Anando Sen: John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne NE1 3BZ, UK
Victoria Hedley: John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne NE1 3BZ, UK
Eva Degraeuwe: Ghent University, 9000 Ghent, Belgium
Steven Hirschfeld: Uniformed Services University of the Health Sciences, Bethesda, MD 20814, USA
Ronald Cornet: Amsterdam University Medical Centers, 1105 AZ Amsterdam, The Netherlands
Ramona Walls: Critical Path Institute, Tucsan, AZ 85718, USA
John Owen: Clinical Data Interchange Standards Consortium (CDISC) Europe Foundation, Saint-Gilles, 1060 Brussels, Belgium
Peter N. Robinson: The Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USA
Edward G. Neilan: National Organization for Rare Disorders, Quincy, MA 02169, USA
Thomas Liener: Pistoia Alliance, Wakefield, MA 01880, USA
Giovanni Nisato: Pistoia Alliance, Wakefield, MA 01880, USA
Neena Modi: Imperial College London, London SW7 2AZ, UK
Simon Woodworth: INFANT Research Centre, University College Cork, Cork T12 YE02, Ireland
Avril Palmeri: John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne NE1 3BZ, UK
Ricarda Gaentzsch: IQVIA Italy S.r.l., 20124 Milan, Italy
Melissa Walsh: INFANT Research Centre, University College Cork, Cork T12 YE02, Ireland
Teresa Berkery: INFANT Research Centre, University College Cork, Cork T12 YE02, Ireland
Joanne Lee: John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne NE1 3BZ, UK
Laura Persijn: Ghent University, 9000 Ghent, Belgium
Kasey Baker: National Organization for Rare Disorders, Quincy, MA 02169, USA
Kristina An Haack: Sanofi Genzyme, 75017 Paris, France
Sonia Segovia Simon: John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne NE1 3BZ, UK
Julius O. B. Jacobsen: Queen Mary University of London, London E1 4NS, UK
Giorgio Reggiardo: Consorzio per Valutazioni Biologiche e Farmacologiche, 70122 Bari, Italy
Melissa A. Kirwin: Clinical Data Interchange Standards Consortium (CDISC), Austin, TX 78701, USA
Jessie Trueman: John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne NE1 3BZ, UK
Claudia Pansieri: Consorzio per Valutazioni Biologiche e Farmacologiche, 70122 Bari, Italy
Donato Bonifazi: Consorzio per Valutazioni Biologiche e Farmacologiche, 70122 Bari, Italy
Sinéad Nally: Novartis Pharmaceuticals, Dublin D04 NN12, Ireland
Fedele Bonifazi: Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus, 70124 Bari, Italy
Rebecca Leary: John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne NE1 3BZ, UK
Volker Straub: John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne NE1 3BZ, UK

Data, 2024, vol. 9, issue 4, 1-18

Abstract: The conect4children (c4c) initiative was established to facilitate the development of new drugs and other therapies for paediatric patients. It is widely recognised that there are not enough medicines tested for 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 cross-cutting data resources that build on existing CDISC standards in an effort to standardise 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 analysed their use for standardising 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 standardisation. 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 standardising 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 organisation of further workshops and educational materials.

Keywords: conect4children; paediatric clinical trials; data standards; FAIR principles; interoperability; CDISC (search for similar items in EconPapers)
JEL-codes: C8 C80 C81 C82 C83 (search for similar items in EconPapers)
Date: 2024
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