Economic Burden of High-Responding Inhibitors in Patients with Hemophilia A in Taiwan (original) (raw)
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Blood, 2003
Inhibitors in patients with hemophilia are a rare complication of a rare disease causing pain and disability in patients and impairment to the quality of their lives. Recent advances in treatment have brought improvements, but they have done so by absorbing larger amounts of financial resources. This study involved 52 Italian patients with hemophilia with high-responding inhibitors who were longitudinally observed for 18 months to evaluate concomitantly cost of care and quality of life. Overall, 0.6 bleeding episodes per patient per month were recorded. This frequency of events was lower than that reported in other cohorts of patients with hemophilia who were not taking inhibitors. The average monthly cost of care was, in euros, €18 000 (US $18 000) per patient, mainly because of treatment products. Recombinant activated factor VII, mostly used for orthopedic surgery, represented 50% of the expenses. Quality of life, measured through validated questionnaires, was similar to that of ...
Cost of care of haemophilia with inhibitors
Haemophilia, 2010
Summary. In Western countries, the treatment of patients with inhibitors is presently the most challenging and serious issue in haemophilia management, direct costs of clotting factor concentrates accounting for >98% of the highest economic burden absorbed for the healthcare of patients in this setting. Being designed to address questions of resource allocation and effectiveness, decision models are the golden standard to reliably assess the overall economic implications of haemophilia with inhibitors in terms of mortality, bleeding-related morbidity, and severity of arthropathy. However, presently, most data analyses stem from retrospective short-term evaluations, that only allow for the analysis of direct health costs. In the setting of chronic diseases, the cost-utility analysis, that takes into account the beneficial effects of a given treatment/healthcare intervention in terms of health-related quality of life, is likely to be the most appropriate approach. To calculate net benefits, the quality adjusted life year, that significantly reflects such health gain, has to be compared with specific economic impacts. Differences in data sources, in medical practice and/or in healthcare systems and costs, imply that most current pharmacoeconomic analyses are confined to a narrow healthcare payer perspective. Long-term/lifetime prospective or observational studies, devoted to a careful definition of when to start a treatment; of regimens (dose and type of product) to employ, and of inhibitor population (children/adults, low-responding/high responding inhibitors) to study, are thus urgently needed to allow for newer insights, based on reliable data sources into resource allocation, effectiveness and cost-utility analysis in the treatment of haemophiliacs with inhibitors.
Journal of Medical Economics, 2005
The effectiveness data were derived from a retrospective analysis, expert opinion and published studies. Link between effectiveness and cost data The cost analysis was carried out retrospectively on the same sample as that used for the effectiveness analysis. Study sample The study sample comprised 105 bleeding episodes in 24 patients. Bleedings were treated with rFVIIa in 28 events, with PCC in 25, with aPCC in 9, and with high-dose Factor VIII in 43. Sixteen of the 24 patients were adults aged over 17 years. The mean weight was 49 kg. No further information was given.
Iranian Journal of Pharmaceutical Sciences, 2016
Bypassing agents are the most commonly used medicines for the treatment of hemophilia patients with inhibitors. The aim of this study is to identify the cost components of management of bleeding vents in hemophilia patients with inhibitors in Iran. This study is a cross-sectional study using a bottom-up approach to determine the cost components of treatment of hemophilia patients with inhibitors via ascertaining of all direct medical and non-medical costs.The evaluating of cost components of 20 patients with 40 episodes of bleeding showed that the price of medicines used is responsible for more than 96% and 97% of costs in treatment of hemophilia patients using FEIBA® and AryoSeven, respectively. The results of this study showed that cost of treating one bleeding episode in hemophilia A patients with high antibody titer using FEIBA is 376 USD compared to 857.3 USD for AryoSeven®.Despite the small number of hemophilia patients with inhibitor in Iran, due to high cost of treating thes...
Health economics of treating haemophilia A with inhibitors
Haemophilia, 2005
Haemophilia is a rare, inherited blood disorder in which blood clotting is impaired such that patients suffer from excessive internal and external bleeding. At present there is no cure for haemophilia A and patients require expensive, lifelong treatment involving clotting factor replacement therapy. Treatment costs are perceived to be higher for patients who have developed inhibitory antibodies to factor VIII, the standard therapy for haemophilia A. However, initial cost analyses suggest that clotting factor therapy with alternative haemostatic agents, such as recombinant activated factor VII or activated prothrombin complex concentrate, is no more expensive for the majority of haemophilia A patients with inhibitors than for those without inhibitors. With the availability of effective alternative haemostatic agents, orthopaedic surgery for haemophilia A patients with inhibitors is now a clinical option, and initial cost analyses suggest this may be a cost-effective treatment strategy for patients with inhibitors whose quality of life (QoL) is severely impaired by joint arthropathy. In an era of finite healthcare resourcing it is important to determine whether new treatments justify higher unit costs compared with standard therapies and whether such higher costs are justified from an individual perspective in terms of improved QoL, and from a societal perspective in terms of improved productivity and reduced overall healthcare costs. This paper examines current data on the health economics of treating haemophilia A patients with inhibitors, focusing on the overall costs of clotting factor replacement therapy and the cost consequences of joint replacement.
Inhibitors in patients with haemophilia are rare complication causing pain, disability and impairment. There is a general perception that it has a determinant impact on costs of care in these patients. The costs of factor consumption in 6 Iranian hemophiliacs with high responding inhibitors as the case group and 6 hemophiliacs without inhibitors as the control during 12 months were studied in a clinical centre in south of Iran. The data concerning clinical and demographic characteristics of patients were retrieved from their medical records. Results indicated that all patients suffered from severe haemophilia. The mean weight of inhibitors and non-inhibitors groups was 67.33 (±18.67) and 66 (±15.23) respectively. Overall 1.79 (±0.75) and 1.95 (±1.66) bleeding per patient monthly were recorded for inhibitors and non-inhibitors groups, respectively. The median annual costs of care were three time higher for inhibitors than non-inhibitors patients (US$ 67987.875 vs. US$ 21415.145). in conclusion: Based on the finding from the current study, presence of inhibitor affects the cost of factor replacement therapy in treating of haemophilia A in short-term.
Haemophilia, 2010
This study compared secondary prophylaxis treatment with on-demand treatment for severe haemophilia A in Taiwan. Fifty patients from one medical centre were evaluated over a 5-year period. Differences in annual bleed rates and factor VIII (FVIII) utilization were assessed between patients receiving secondary prophylaxis and patients receiving FVIII concentrates on-demand. Results were then used as inputs in a pharmacoeconomic model to predict outcomes of future haemophilia therapy strategies in Taiwan. The median annual number of total bleeding episodes was significantly lower in the 13 (26%) patients who received secondary prophylaxis than in the 37 patients who received FVIII on-demand (7.76 vs. 31.91, P < 0.0001). The between-group difference in median annual factor VIII utilization was statistically significant (1824.41 IU kg)1 for the prophylaxis group and 1324.81 IU kg)1 for the on-demand group, P < 0.01). It was estimated that approximately $2 million (USD) per year would be added to the cost of treatment by having all severe haemophilia A patients in Taiwan receive secondary prophylaxis instead of on-demand therapy while 12 566 bleeding will be prevented. It is recommended that National Health Insurance officials utilize these data to evaluate the benefits of enhanced treatment strategies and before making substantial policy changes to haemophilia care in Taiwan.
Pharmacoeconomic Analysis of Hemophilia Care in Romania
2020
Hemophilia, a congenital X linked disease, has the serious burden of bleeding, requiring life-long replacement with coagulation factors (CF). In the present day, there is a continuously improving treatment for this condition. Objective: Our observational, cross-sectional study aims at finding out whether a prophylactic replacement with CF is affordable from the point of view of its cost-effectiveness in our country. Material and methods: A cohort of 122 persons with Hemophilia were included in this patient-reported outcome survey, and they answered a questionnaire consisting of 56 items, focused on 4 domains—socio-demographic, medical, quality of health/life and cost/cost-effectiveness. Results and discussion: The markers for quality of health/life were correlated with the direct and indirect costs of care, comparing subgroup 1 of patients with prophylactic vs. subgroup 2 with on-demand replacement. Based on the incremental quality adjusted life years and the incremental costs, we c...
Journal of managed care & specialty pharmacy, 2016
Hemophilia patients use factor-clotting concentrates (factor VIII for hemophilia A and factor IX for hemophilia B) for improved blood clotting. These products are used to prevent or stop bleeding episodes. However, some hemophilia patients develop inhibitors (i.e., the patient's immune system develops antibodies against these factor concentrates). Hence, these patients do not respond well to the factor concentrates. A majority of hemophilia patients with inhibitors are managed on-demand with the following bypassing agents: recombinant factor VIIa (rFVIIa) and activated prothrombin complex concentrate (aPCC). The recently published U.S. registries Dosing Observational Study in Hemophilia (DOSE) and Hemostasis and Thrombosis Research Society (HTRS) reported higher rFVIIa on-demand use for bleed management than previously described. To estimate aPCC and rFVIIa prophylaxis costs relative to rFVIIa on-demand treatment cost based on rFVIIa doses reported in U.S. registries. A literatu...