A Model for a Regional System of Care to Promote the Health and Well-Being of People with Rare Chronic Genetic Disorders.

Abstract

People with rare, inherited chronic health conditions, such as hemophilia, face added physical, social, emotional, and fiscal challenges beyond those that are common to more prevalent chronic conditions. In 1975, a partnership among clinicians, consumers, and government agencies created a nationwide regional health delivery system that increased access to clinical care, prevention, and research, thereby improving health outcomes for people with hemophilia in the United States. Today, more than 130 Comprehensive Hemophilia Diagnostic and Treatment Centers in 12 regions serve 70%–80% of the nation’s hemophilia patients. Health care leaders and advocates for other rare, expensive, chronic disorders may find that regionalization improves survival and reduces disability among affected populations. However, diverse and stable resources are needed to sustain such a model in our profit-oriented US health care arena.

Burden of illness: direct and indirect costs among persons with hemophilia A in the United States

“…The regionalized HTCs, a multi-disciplinary, team-based care delivery model, have been implemented in the US for several decades with goals to prevent orthopedic complications and to maximize the physical and psychological functioning and socioeconomic benefits for this rare disease population 18 . HTC care has been shown to be associated with lower mortality 19 and fewer bleed-related hospitalizations 20 compared with care obtained elsewhere.…”

Physical Functioning in Boys with Hemophilia in the U.S.

“…2 Care for individuals with hemophilia in the U.S. is delivered principally via a network of comprehensive hemophilia treatment centers (HTCs). More than 130 HTCs receive federal support to establish and maintain multidisciplinary teams to provide prospective, preventive, and family-centered surveillance and management of this rare chronic genetic blood coagulation disorder, 3 including data collection on outcomes related to hemophilia.…”

Public health surveillance and data collection: general principles and impact on hemophilia care

“…A network of hemophilia treatment centers (HTCs) was developed in 1975 with federal funding to provide comprehensive multidisciplinary care to people with hemophilia in the U.S. [3]. Data from the HSS showed that about 70% of all people with hemophilia were receiving some care from these centers in the early 1990s.…”