The Mini-Symposium on Global Child Health: Serving the Children of the World

Scutariu, Cristina; Bilardi, Davide; Ayomoh, Francis Ifeanyi; Roehr, Charles C

doi:10.1038/s41390-023-02570-w

Cited by 1 publication

(1 citation statement)

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“…While they may not directly address standardisation issues, their knowledge base is crucial for addressing disease-specific standardisation. The Paediatric European Network for Treatment of AIDS (PENTA) was established as a collaboration between paediatric HIV centres but has expanded to other infectious diseases [39]. The European Society for Paediatric Oncology (SIOPE) is a European organisation representing paediatric cancer experts.…”

Section: Pistoia Alliancementioning

confidence: 99%

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

Sen,

Hedley,

Degraeuwe

et al. 2024

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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.

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Section: Pistoia Alliancementioning

confidence: 99%