Our 2nd paper about Leigh syndrome patient registry is PUBLISHED! Our paper, “Interoperability of Leigh Syndrome Patient Registry Data with Regulatory Submission Standards”, is published in the Journal of the Society for Clinical Data Management (SCDM). You can read the paper here: https://www.jscdm.org/article/id/244/
The Clinical Data Interchange Standards Consortium (CDISC) is dedicated to helping to improve clinical research by driving meaningful and efficient research through data standardization. CDISC standards are mandatory for submissions to the FDA and PMDA.
Establishment of interoperability of registry data with regulatory submission standards like CDISC is essential to accelerate the development of therapies. The project described in our paper has been undertaken to establish such interoperability for Leigh syndrome patient registry data.
To our knowledge, this is the first such project for any mitochondrial disease patient registry, and we hope our experience will serve as a model for other mitochondrial disease and rare disease patient registries.
Additionally, we are proud to see that our work on the registry is very well aligned with the 2 recent #FDA guidances on patient registries and data standards: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/real-world-data-assessing-registries-support-regulatory-decision-making-drug-and-biological-products?fbclid=IwAR11BRCICJz8yocxhzHalgnYiA1R2HOSU5BXOXu6P5UURJOIYE19Pj2AF5w and
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/data-standards-drug-and-biological-product-submissions-containing-real-world-data
This project is a result of our partnership with Sumptuous Data Sciences . Sumptuous Data Sciences provides biostatistics and data operations services in healthcare sector with vision to work towards bridging the gap between healthcare and clinical data analysis and management. We are extremely grateful to Parag Shiralkar and Pallavi Bakare and their team for their partnership on this project and for their support of the Cure Mito Foundation and Leigh syndrome community.
We also thank all patient families who enrolled into the registry so far and all the doctors who share the registry with their patients.
