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.
