RESUMO
BACKGROUND: Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. METHODS: Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. RESULTS: The mean age of participants was 41.50 years (SD 16.01, range 18-81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. CONCLUSIONS: This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies.
Assuntos
Talassemia beta , Humanos , Feminino , Masculino , Talassemia beta/terapia , Terapia por Quelação , Transfusão de Sangue/métodos , Análise Custo-Benefício , Análise de Custo-EfetividadeRESUMO
BACKGROUND: Von Willebrand disease (VWD) is one of the most common inherited bleeding disorders, imposing a substantial health impact and financial burden. The Cost of von Willebrand disease in Europe: A Socioeconomic Study (CVESS) characterises the socio-economic cost of VWD across Germany, Spain, Italy, France, and the UK. METHODS: A retrospective, cross-sectional design captured 12 months of patient disease management, collected from August-December 2018, for 974 patients. This enabled estimation of direct medical, direct non-medical and indirect costs, utilising prevalence estimates to extrapolate to population level. RESULTS: Total annual direct medical cost (including/excluding von Willebrand factor [VWF]) across all countries was the highest cost (2â 845â 510â 345/444â 446â 023), followed by indirect costs (367â 330â 271) and direct non-medical costs (60â 223â 234). Differences were seen between countries: the UK had the highest direct medical costs excluding VWF (159â 791â 064), Italy the highest direct-non medical (26â 564â 496), and Germany the highest indirect cost burden (197â 036â 052). Total direct medical costs per adult patient increased across VWD types with Type 1 having the lowest cost (23â 287) and Type 3 having the highest cost (133â 518). CONCLUSION: A substantial financial burden arises from the prevalence of VWD for the European healthcare systems considered.
Assuntos
Doenças de von Willebrand , Adulto , Estudos Transversais , Europa (Continente)/epidemiologia , Humanos , Estudos Retrospectivos , Doenças de von Willebrand/epidemiologia , Fator de von WillebrandRESUMO
BACKGROUND & AIMS: Non-alcoholic steatohepatitis (NASH) leads to cirrhosis and is associated with a substantial socioeconomic burden, which, coupled with rising prevalence, is a growing public health challenge. However, there are few real-world data available describing the impact of NASH. METHODS: The Global Assessment of the Impact of NASH (GAIN) study is a prevalence-based burden of illness study across Europe (France, Germany, Italy, Spain, and the UK) and the USA. Physicians provided demographic, clinical, and economic patient information via an online survey. In total, 3,754 patients found to have NASH on liver biopsy were stratified by fibrosis score and by biomarkers as either early or advanced fibrosis. Per-patient costs were estimated using national unit price data and extrapolated to the population level to calculate the economic burden. Of the patients, 767 (20%) provided information on indirect costs and health-related quality of life using the EuroQOL 5-D (EQ-5D; n = 749) and Chronic Liver Disease Questionnaire - Non-Alcoholic Fatty Liver Disease (CLDQ-NAFLD) (n = 723). RESULTS: Mean EQ-5D and CLDQ-NAFLD index scores were 0.75 and 4.9, respectively. For 2018, the mean total annual per patient cost of NASH was 2,763, 4,917, and 5,509 for direct medical, direct non-medical, and indirect costs, respectively. National per-patient cost was highest in the USA and lowest in France. Costs increased with fibrosis and decompensation, driven by hospitalisation and comorbidities. Indirect costs were driven by work loss. CONCLUSIONS: The GAIN study provides real-world data on the direct medical, direct non-medical, and indirect costs associated with NASH, including patient-reported outcomes in Europe and the USA, showing a substantial burden on health services and individuals. LAY SUMMARY: There has been little research into the socioeconomic burden associated with non-alcoholic steatohepatitis (NASH). The GAIN study provides real-world data on the direct medical, direct non-medical, and indirect costs associated with NASH, including patient-reported outcomes in five European countries (UK, France, Germany, Spain, and Italy) and the USA. Mean total annual per patient cost of NASH was estimated at 2,763, 4,917, and 5,509 for the direct medical, direct non-medical, and indirect cost categories, respectively.