Background: β-thalassemia is a prevalent disease in Italy relative to many other countries. There is a paucity of data in the literature on the direct medical care costs associated with β-thalassemia. We conducted a β-thalassemia treatment center survey and chart review to estimate the numbers of patients with transfusion-dependent (TD) or non-transfusion-dependent (NTD) β-thalassemia currently under care and the associated direct medical care costs in Italy.Methods: A preliminary list of β-thalassemia treatment centers was generated from the IMS Health audit of hospitals and treatment centers across Italy based on the use of iron chelation therapy (ICT) agents. One or more hematologists or pediatricians in each potential hospital ward were contacted, by telephone or in-person interview, and asked about the number of patients with β-thalassemia major, TD β-thalassemia intermedia, and NTD β-thalassemia intermedia managed in their ward. TD status was determined using local definitions by each physician. Subsequently, a geographically stratified sample of 60 physicians was asked to complete a web-based questionnaire on healthcare resource utilization for the four most recent patients under their care: one patient with β-thalassemia major, one with TD β-thalassemia intermedia, one with NTD β-thalassemia intermedia, and one patient at their discretion. Healthcare resource utilization focused on five categories: transfusions, ICT, laboratory work, β-thalassemia-associated complications, and inpatient hospital stay. Unit costs of each healthcare utilization were extracted from relevant literature and published government fee schedules. For prescription drug prices, IMS Health sales data were referenced (values were provided in EUR).Results: We identified 124 treatment centers, 114 (92%) of which were successfully surveyed. A total of 5,748 TD β-thalassemia patients (4,515 with β-thalassemia major plus 1,233 with TD β-thalassemia intermedia) and 1,296 NTD β-thalassemia intermedia patients were reported. Taking into consideration the sampling frame, missing data, infrequent care for NTD β-thalassemia intermedia patients, and errors (such as the fact that ICT can be used to manage conditions other than β-thalassemia), we estimated that there are approximately 6,500 TD and 6,000 NTD β-thalassemia intermedia patients in Italy. A total of 205 β-thalassemia patient records were reviewed (109 β-thalassemia major, 53 TD β-thalassemia intermedia, and 43 NTD β-thalassemia intermedia); the median ages for patients with β-thalassemia major, TD β-thalassemia intermedia, and NTD β-thalassemia intermedia were 37, 44, and 43 years, respectively. For patients with β-thalassemia major, the total annual direct cost per patient was EUR 31,883, of which ICT was the most expensive cost component at EUR 22,519, followed by transfusions (EUR 6,115), complications (EUR 1,427), in-hospital stay (EUR 1,213), and laboratory tests (EUR 392). Patients with TD β-thalassemia intermedia incurred nearly identical medical care costs at EUR 31,183, of which EUR 22,963 was attributable to ICT. Of the 162 TD patient records reviewed, 83 patients required 1-2 red blood cell (RBC) units per month, whereas 78 patients required ≥ 3 RBC units per month (1 patient had missing data). Every patient with TD β-thalassemia received ICT, and deferasirox was the most commonly administered chelation agent, prescribed to 103 of 162 patients. The annual direct cost per patient with NTD β-thalassemia intermedia was lower at EUR 13,496, largely owing to lower healthcare utilization of ICT and transfusions. Combining the costs of TD and NTD β-thalassemia treatment, we estimated that the total annual direct medical care cost was approximately EUR 287,162,500 in Italy, of which TD patients accounted for EUR 204,682,000 (71.3%).Conclusions: β-thalassemia remains a prevalent disease in Italy with about 6,500 TD and 6,000 NTD β-thalassemia patients. Management of the disease consumes significant healthcare resources, with the total annual direct medical care cost for β-thalassemia treatment estimated to be approximately EUR 287 million. DisclosuresAngelucci:Bluebird Bio: Other: Advisory board: Gene therapy in Thalassemia; Novartis Oncology: Other: Protocol Telesto: sterring committee Chair; Roche: Other: Advisory board: biosimilars; Celgene: Other: protocoll ACE 536 B-Thal 001: DMC Chair; Jazz: Other: Advisory board: AML; Novartis Oncology: Other: Advisory board: iron toxicity; Novartis Oncology Swiss: Other: Invited speakers sponsored satellite meeting during ; Celgene: Honoraria, Other: Advisory: research project . Antmen:Jazz: Other: Advisory board; Novartis: Other: Advisory board; Celgene: Honoraria, Other: Advisory for the project Burden of illness in thalassemia. Losi:QuintilesIMS: Consultancy; Celgene: Other: Commissioned to carry out the data collection survey as specified in the abstract as an employee of QuintilesIMS. Burrows:QuintilesIMS: Consultancy; Celgene: Other: Commissioned to carry out the data collection survey as specified in the abstract as an employee of QuintilesIMS. Bartiromo:Celgene: Employment, Equity Ownership. Hu:Celgene: Employment, Equity Ownership.
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Jan 1, 2024
Yesim Aydinok + 7
Open Access
