Rare diseases are not a marginal topic!
Rare diseases are not a marginal topic. There are thousands of them and they affect half a million Czechs!
Specialized centers of the VFN and the 1st Faculty of Medicine, Charles University play a key role in patient care - they provide a chance for faster diagnostics and access to the most modern treatment that was previously unavailable. The average time to diagnose rare diseases in Europe and the Czech Republic is around five years or more. There are thousands of diagnoses, and the time needed to determine them and start the correct treatment is crucial. The European Reference Networks (ERN) - virtual platforms connecting 375 hospitals and 1,606 top workplaces across 27 EU countries and Norway - help to shorten it. The VFN is a significant part of the ERN, providing top-notch care in 7 specialized centers and caring for twelve thousand patients with rare diagnoses. Last year alone, almost two thousand more patients were added.
A game of time: A coordinated system of care is needed
Especially for pediatric patients, a delay can mean a significant deterioration in their health. In addition to the treatment itself, it is therefore necessary to work on an interconnected, coordinated and accessible system of health and social care for patients and their families. European Reference Networks – virtual platforms connecting 375 hospitals and 1,606 top-notch workplaces across 27 EU countries and Norway – help to shorten this critical time. The General Teaching Hospital in Prague (VFN) is an important part of the European Reference Network (ERN) for rare diseases, providing top-notch care in 7 specialized centers and caring for twelve thousand patients with rare diagnoses, with almost two thousand more added last year alone. "We consider the inclusion of the General University Hospital in Prague among the ERN members to be an extraordinary confirmation of the long-term quality and professionalism of our workplaces. It is clear proof that the General University Hospital will stand up to a strict international comparison of the best," explains Prof. MUDr. Pavel Michálek, Ph.D., DESA, M.Sc., MBA, Deputy Director for Science, Research and Education at the General Hospital. The hospital is currently the provider with the second highest number of centers involved in the ERN in the entire Czech Republic.
Benefit for doctors: Sharing know-how and scientific progress
For specialists from the VFN, membership in the ERN means a unique opportunity to consult highly complicated cases with colleagues from all over Europe in real time. The hospital focuses on the areas of rare diseases of the endocrine, neurological, urological, ophthalmic, metabolic, immune system diseases and pulmonary hypertension. "Our hospital actively contributes its own experience to the creation of recommended practices. Membership in the ERN means new experiences, further education of doctors, supports clinical research and the opportunity to participate in international projects," calculates the benefits of Prof. MUDr. Pavel Jansa, Ph.D., head of the Center for Pulmonary Hypertension of the 2nd Internal Clinic – Department of Cardiology and Angiology of the 1st Faculty of Medicine of Charles University and the General Hospital.
Benefit for patients: Hope for accurate and rapid diagnosis
The General University Hospital is the only Czech representative of ERN RITA with European certification for three groups of immune disorders: autoimmune and autoinflammatory (hereditary immune disorders) diseases in children and adults and childhood rheumatic diseases. The center cares for about 1,400 children and 490 adults annually. The Department of Pediatrics and Inherited Metabolic Disorders of the 1st Faculty of Medicine of Charles University and the General Hospital mainly focuses on pediatric patients with a wide range of systemic inflammatory diseases, from the relatively most common juvenile idiopathic arthritis to rare, often genetically determined hereditary immune disorders. In the adult part of the center, the Department of Nephrology of the 1st Faculty of Medicine of Charles University and the General Hospital, patients with rare systemic autoimmune diseases that involve vascular damage, inflammation or narrowing of the vessel walls (vasculitis) are monitored. "We have enough excellent specialists, but patients often have a long journey from a general practitioner to a specialized team. It is necessary to improve the interconnection of disciplines and the connection to social and field services," points out Prof. MUDr. Pavla Doležalová, head of the Center for Pediatric Rheumatology and Autoinflammatory Diseases of the Department of Pediatrics and Inherited Metabolic Disorders (KPDPM).
