Region V-East HTC Network for the Prevention of Bleeding Disorders Complications

描述(申请人提供)：本申请的目标是确保为V-East地区多学科综合诊所网络的持续发展和维护提供支持，以进行监测并实施预防血友病和其他出血性疾病并发症的计划。在之前联邦政府为工作人员和临床基础设施提供资金的基础上，疾控中心前两个五年期的资金重点是开发一个国家数据库，以收集关键数据，从而能够评估结果，包括血液传播的病毒传播、关节疾病、抑制剂形成和死亡率。这个数据库现在是世界上最大的一种罕见慢性病的临床结果数据库。第三个五年计划将侧重于扩大队列和完善收集的数据，以继续确定亚人群的特征，包括血友病A和B、患有出血性疾病的妇女、von Willebrand病和罕见的出血性疾病。已开始作出重大努力，以提高数据收集方法的复杂性，并最终完成从纸质系统向电子系统的过渡，该系统目前正在转换为与疾控中心的网络接口。这一基础设施将极大地提高我们的国家临床研究能力，以改善护理，支持基于结果的标准化，并抵御支付者通过拒绝获得治疗方案和产品来削减成本的努力。到目前为止收集的数据表明，在联邦资助的HTC接受护理的患者的死亡率和发病率有所下降。护理是可以获得的，患者有许多资源可以克服慢性病家庭面临的传统障碍，包括与其他专家的联系、基于社区的社会服务以及患者的宣传和支持。这一护理模式是根据商定的国家和区域目标进行协调的，是以全面和协作的公共卫生办法管理可能适用于其他罕见和/或代价高昂的慢性病的成功模式。区域网络基础设施和数据收集能力还可为评估预防和干预战略提供肥沃土壤，以应对影响普通民众的广泛公共卫生问题，如营养和肥胖问题以及遵从性问题。

DESCRIPTION (provided by applicant): The objective of this application is to secure support for the ongoing development and maintenance of the Region V-East network of multidisciplinary comprehensive clinics to conduct surveillance and implement programs to prevent the complications of hemophilia and other bleeding disorders. Building on previous federal funding for staff and clinical infrastructure, the first two five-year periods of CDC funding focused on the development of a national database to collect critical data that enabled outcome assessment including blood-borne viral transmission, joint disease, inhibitor formation, and mortality. This database is now the largest clinical outcomes database of a rare chronic disease in the world. This third five-year period will focus on expansion of the cohort and the refinement of data collected to continue to characterize sub-populations, including hemophilia A and B, women with bleeding disorders, von Willebrand disease, and rare bleeding disorders. Major efforts have begun to increase the sophistication of data collection methodologies and to finalize the transition from a paper to an electronic system that is currently being converted to a web-based interface with the CDC. This infrastructure will greatly increase our capacity for national clinical research to improve care, support outcomes-based standardization, and defend against efforts by payers to cut costs by denying access to therapeutic regimens and products. Data collected to date has shown decreased mortality and morbidity among patients who receive care at federally funded HTCs. Care is accessible and patients have many resources to overcome traditional barriers that face families with chronic diseases, including linkages with other specialists, community based social services and patient advocacy and support. This model of care, which is coordinated in response to agreed upon national and regional objectives, is a successful model of a comprehensive and collaborative public health approach to the management of a chronic disorder that may be applicable to other rare and/or expensive chronic diseases. The regional network infrastructure and data collection capacity may also provide fertile ground for the assessment of prevention and intervention strategies for broad public health concerns like nutrition and obesity and compliance that affect the general population.