SATURN: assessing the feasibility of utilising existing registries for real-world evidence data collection to meet patients, regulatory, health technology assessment and payer requirements

This robust feasibility process highlights potential limitations and opportunities to develop and to refine the collaboration with the ROI as the SATURN programme moves forward. It also ensures that the existing datasets in the rare condition OI are being maximised to respond to the needs of patients, data customers and decision-makers. This feasibility process has allowed SATURN to build a compliant methodology that aligns with the requirements from the European Medicines Agency (EMA) and HTAs. More data variables will continue to be developed and refined along the way with more registries participating in SATURN. As a result, SATURN will become a meaningful and truly collaborative core dataset, which will also contribute to advancing understanding of OI diagnosis, treatment, and care.

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Publication Details

Journal
Orphanet Journal of Rare Diseases
Published
2024-09-12
DOI
https://doi.org/10.1186/s13023-024-03341-4
Citations
1
Primary Topic
Genomics and Rare Diseases
Type
article
Field-Weighted Citation Impact
0.55

Funders

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article

SATURN: assessing the feasibility of utilising existing registries for real-world evidence data collection to meet patients, regulatory, health technology assessment and payer requirements

Marina Mordenti, Victoria Wang, Ingunn Westerheim, Rebecca Skarberg et al.
1 citations
Orphanet Journal of Rare Diseases
Genomics and Rare Diseases
0.55
article

SATURN: assessing the feasibility of utilising existing registries for real-world evidence data collection to meet patients, regulatory, health technology assessment and payer requirements

Marina Mordenti, Victoria Wang, Ingunn Westerheim, Rebecca Skarberg, Rafael Pinedo‐Villanueva, Evert Jan Van Lente, Maria Cavaller-Bellaubi, Manila Boarini, James Clancy, Luca Sangiorgi, Mario Marchetti
article en
1 citations

Abstract

This robust feasibility process highlights potential limitations and opportunities to develop and to refine the collaboration with the ROI as the SATURN programme moves forward. It also ensures that the existing datasets in the rare condition OI are being maximised to respond to the needs of patients, data customers and decision-makers. This feasibility process has allowed SATURN to build a compliant methodology that aligns with the requirements from the European Medicines Agency (EMA) and HTAs. More data variables will continue to be developed and refined along the way with more registries participating in SATURN. As a result, SATURN will become a meaningful and truly collaborative core dataset, which will also contribute to advancing understanding of OI diagnosis, treatment, and care.

Orphanet Journal of Rare DiseasesVol. 19(1)
European Organisation for Rare Diseases (FR), IDEO (United States) (US), Nuffield Orthopaedic Centre (GB), Statistics Belgium (BE), University of Oxford (GB), Italian Medicines Agency (IT), Istituto Ortopedico Rizzoli (IT), ERN GUARD-Heart (NL), Osteogenesis Imperfecta Foundation (US)
Mereo BioPharma
Partnerships for the goals
Openalex Percentile: Top 26%
Genomics and Rare Diseases
0.55
DOIOpen Access PDFOpenAlex
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