Mapping of Data-Sharing Repositories for Paediatric Clinical Research—A Rapid Review (Felisi et. al., 2024)

This resource provides a comprehensive overview of data-sharing repositories supporting paediatric clinical research and discusses the role of individual patient data (IPD) sharing in improving the efficiency, transparency and reproducibility of clinical research. The review identifies existing repositories and highlights key considerations for researchers seeking to share, access and reuse clinical trial datasets.

The resource addresses important aspects of data management beyond data collection and storage, including data accessibility, governance, interoperability and the potential for secondary use of clinical research datasets. By mapping available infrastructures, it supports researchers in developing appropriate data-sharing strategies and considering data stewardship throughout the clinical trial lifecycle.

This resource is particularly relevant for rare disease clinical trials because these studies frequently involve small patient populations, limited statistical power and geographically dispersed participants. Sharing and integrating datasets from multiple studies can help increase evidence generation capacity, support natural history analyses, facilitate identification of clinically meaningful outcomes and improve the efficiency of future trials. This is especially important in paediatric rare diseases, where recruitment challenges and limited available data are major barriers to clinical development.

For investigators planning rare disease clinical trials, the resource can support early decisions regarding data management plans, repository selection and future data reuse opportunities. It complements regulatory and ethical guidance on data governance by helping researchers understand the practical landscape of available data-sharing infrastructures.

Although focused on paediatric clinical research, the principles are broadly applicable to rare disease trials across age groups, particularly studies involving multinational collaborations, small populations, registries, natural history studies or precision medicine approaches.