OBJECTIVES: The sustainability of the healthcare systems is complex due to aging population and innovative drugs that lengthen life expectancy by turning deadly diseases into chronic ones. The complexity is even higher talking about chronic and rare diseases. The aim of this analysis was to show how value based healthcare (VBHC) could enhance quality of care while containing costs in providing optimal care for hemophilic patients.

METHODS: The analysis was conducted in three Hemophilia Centers (HC) in Italy. The most relevant steps in the clinical management of hemophilic patients were identified. A survey was conducted for the analysis of the organizational models adopted by each hospital while interviews and direct observation of clinical activities allowed to describe the patient pathways. In parallel, an activity-based cost analysis was carried out. Patients were classified in patient profiles representing homogeneous categories in terms of resources needed. Clinical and economical KPIs were set.

RESULTS: Eight patients’ profiles were defined according to patients’ age, disease severity and comorbidities. For each profile, costs, not considering drugs, were very different. For example, an adult patient with severe disease and arthropaty, inhibitor positive and co-morbidity cost between 2,984€ and 3,869€ per year. Conversely for an adult patient with mild hemophilia and without co-morbidities or arthropathy the average annual cost was, in all the three centers, around 200€.

CONCLUSIONS: The study outlined the importance of designing clinical pathways, at hospital level, around patient profiles, contextualizing national and international guidelines. An appropriate management of hemophilia should call for a different set of activities (e.g. number of visits or diagnostics) for these types of patients with a relevant economic impact. Finally, the role of HC should be embedded within the regional network on the basis of the characteristics of each center.