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

描述（由申请人提供）：本申请的目的是确保对东部V区多学科综合诊所网络的持续开发和维护的支持，以进行监测和实施预防血友病和其他出血性疾病并发症的计划。在之前为工作人员和临床基础设施提供的联邦资金的基础上，疾病预防控制中心前两个五年期的资金重点是建立一个国家数据库，以收集关键数据，从而能够进行结果评估，包括血源性病毒传播、关节疾病、抑制物形成和死亡率。该数据库目前是世界上最大的罕见慢性病临床结果数据库。第三个五年期将侧重于扩展队列和完善收集的数据，以继续表征亚群，包括血友病A和B、出血性疾病女性、血管性血友病和罕见出血性疾病。已开始作出重大努力，以提高数据收集方法的复杂性，并完成从纸面系统向电子系统的过渡，目前正在将电子系统转换为与疾病预防控制中心的网络接口。这一基础设施将大大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。迄今为止收集的数据显示，在联邦资助的卫生技术中心接受治疗的患者死亡率和发病率下降。护理是可以获得的，病人有许多资源来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病人的宣传和支持。这一护理模式是根据商定的国家和区域目标进行协调的，是一个成功的模式，采用综合和协作的公共卫生办法来管理慢性疾病，可能适用于其他罕见和/或昂贵的慢性疾病。区域网络基础设施和数据收集能力也可为评估营养和肥胖症等影响一般民众的广泛公共卫生问题的预防和干预战略以及遵守情况提供肥沃的土壤。

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.