Data Strategy Consortium for the NHLBI Cure Sickle Cell Initiative

目前，美国有超过10万人患有镰状细胞病（SCD）。 每年大约有1,600名婴儿出生时患有SCD，近一半的婴儿将无法存活超过50年 鉴于目前的治疗方法。基因疗法为治愈提供了有希望的可能性，这导致了 由国家心脏、肺和血液研究所（NHLBI）发起的治愈镰状细胞（CureSC）倡议。 CureSC计划将吸引多个利益相关者和数据源，以推进 开发创新的治疗方法，改善SCD患者的生活。 RTI国际是一个非营利的全球性多学科研究机构，致力于改善 人的知识转化为实践。我们很高兴被考虑成为 CureSC倡议的数据战略联盟，将制定数据收集和管理计划， 建立数据标准，协调各种数据资源，以开发联合 数据登记和储存库。该注册表和存储库将作为患者、提供者和 研究人员，并最终确定良好特征的患者进行临床试验和其他研究。RTI 拥有丰富的专业知识和企业经验，可为患者领域的战略讨论带来 登记册、SCD、数据标准、数据源的数据协调和互操作性、共同数据要素 （CDE），数据收集和管理，数据共享和临床试验。我们还在领导 NHLBI关于Data STAGE倡议，该倡议将成为CureSC倡议的组成部分，并为数据提供信息。 战略方针。参与Data STAGE的工作人员包括在本提案中。以下各节介绍我们的 对数据战略联盟目标的理解和方法

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