Artikel
Patient Preferences and Cost effectiveness of Haemophilia Treatment: A Cross-National Assessment
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Autoren
Veröffentlicht: | 14. September 2004 |
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Gliederung
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Objective
To derive patient preferences for and determine incremental cost effectiveness of on-demand vs. prophylactic haemophilia therapy in Sweden, United Kingdom, The Netherlands, and Germany from the Third Party Payers' perspective.
Methods
Patients with severe haemophilia A and B, without inhibitors and at least 14 years of age, were enrolled in this subanalysis of original data collected by the European Haemophilia Economic Study Group. Patient preferences were measured as utilities. For derivation of utilities associated with on-demand and prophylactic therapy, a preference-based algorithm was used to transform the 36-items of the SF-36 into a single SF-6D utility measure. A decision-tree model was developed to compare clinical outcomes, quality-adjusted life years, and direct medical costs of on-demand versus prophylactic treatment. Using multiple regression analysis, utilities were estimated and combined with economic data to perform an incremental cost effectiveness analysis.
Results
The analysis included 506 patients. Patients receiving prophylactic treatment had higher mean utilities than patients on on-demand therapy (>30 yrs: 0.68 vs. 0.66, p=0.15; ≤30 yrs: 0.76 vs. 0.73, p=0.02). Stratification by HIV-infection showed that the utility gain with prophylactic treatment was higher in HIV-negative patients (prophylaxis: 0.73, on demand: 0.68; p<0.0001) compared to HIV-positive patients (prophylaxis: 0.69; on demand: 0.68; p=0.67). With prophylactic treatment, the incremental cost per avoided bleeding ranged from EUR 6,650 for patients ≤30 yrs in Germany to EUR 14,138 for patients >30 yrs in Sweden. The incremental effectiveness ratios in Germany were €1.2 million per QALY gained for patients ≤30yrs/HIV-positive and €2.2 million for patients ≤30yrs/HIV-negative. In the age group >30yrs/HIV-positive the on-demand treatment strategy was dominant, whereas in the >30yrs/HIV-negative group the incremental cost-utility ratio was €4.7 million per QALY.
Conclusion
Prophylactic treatment appears to have a high cost-effectiveness ratio when only a 1-year time horizon is considered. Further research should focus on the long-term consequences of these strategies.