Data Strategy Consortium for the NHLBI Cure Sickle Cell Initiative

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More than 100,000 individuals in the United States are currently living with sickle cell disease (SCD). Approximately 1,600 infants are born each year with SCD, and nearly half will not survive past their fifth decade given the availability of current treatments. Gene therapies offer promising possibilities for a cure, which has led to the initiation of the Cure Sickle Cell (CureSC) Initiative by the National Heart, Lung, and Blood Institute (NHLBI). The CureSC Initiative will engage multiple stakeholders and data sources with the goal of advancing the development of innovative treatments and improving the lives of individuals with SCD. RTI International is a nonprofit global and multidisciplinary research institute dedicated to improving the human condition by turning knowledge into practice. We are pleased to be considered for membership to the CureSC Initiative’s Data Strategy Consortium, which will develop a data collection and management plan to establish data standards and harmonization across various data resources toward the development of a federated data registry and repository. The registry and repository will serve as a resource for patients, providers, and researchers, and ultimately the identification of well-characterized patients for clinical trials and other studies. RTI has extensive personnel expertise and corporate experience to bring to strategic discussions in the areas of patient registries, SCD, data standards, data harmonization and interoperability of data sources, common data elements (CDEs), data collection and management, data sharing, and clinical trials. We are also providing leadership to NHLBI on the Data STAGE Initiative, which will be an integral part of the CureSC Initiative and inform the data strategy approach. Staff involved in Data STAGE are included in this proposal. The following sections describe our understanding and approach to the objectives of the Data Strategy Consortium