Cost Components of Treatment Protocols of Haemophilia Patients with Inhibitors using Bypassing Agents in Iran (original) (raw)
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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.
The development of neutralizing antibodies against factor VIII and IX complicates the clinical management of patients with hemophilia, and adds significant costs to their treatment. For this reason, several formal economic analysis have been performed comparing treatment of mild and moderate bleeding episodes in these patients in an effort to determine whether one of the two available bypassing agents would be cost-effective compared with the other. Here we review and discuss the available evidence about the economic aspects of the alternatives for on-demand treatment of mild and moderate bleeding episodes for hemophilia patients with inhibitors.
Iranian journal of pharmaceutical research : IJPR, 2016
Nowadays, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrates (aPCC) are used to treat bleeding episodes in the Hemophilia patients with inhibitors. AryoSeven® is an Iranian biogeneric rFVIIa with homogeneity of efficacy and the nature to NovoSeven in a comparative trial. The current clinical trial aimed to evaluate the cost-effectiveness of FEIBA and AryoSeven® by Decision Analytic Model according to the Iranian healthcare system. An open label, multi-center, cross-over clinical trial was designed. Patients were categorized into 3 groups based on their prior tendency to one or none of the products. To determine the premium therapeutic strategy, the Incremental cost-effectiveness ratio (ICER) was calculated. Protocol F led to more treatment success in group F than the other groups (P= 0.03). Also, there was a significant statistical difference between the mean of effectiveness scores in the groups using protocol F (P = 0....
Hematology
Objective: This study aimed to compare the cost-effectiveness of these two regimens in hemophilia A patients, under-12-years-old in southern Iran. Methods: A cost-effectiveness study comparing prophylaxis versus on-demand was conducted on 34 hemophilia patients (24 and 10 patients were on the prophylaxis and on-demand regimens respectively) in 2017. The Markov model was used to estimate the economic and clinical outcomes. The costs were collected from the societal perspective, and the utility criterion was the 'quality adjusted life year (QALY)' indicator. The required data were collected using a researcher-made cost checklist, the EQ5D standard questionnaire and Hemophilia Joint Health Score. The probabilistic sensitivity analysis (PSA) was performed to determine the robustness of the results. Results: The means of costs, joint health score and QALY in the prophylaxis regimen were 478,963.1purchasingpowerparity(PPP),96.67,and11.98respectively,andintheondemandregimenwere478,963.1 purchasing power parity (PPP), 96.67, and 11.98 respectively, and in the ondemand regimen were 478,963.1purchasingpowerparity(PPP),96.67,and11.98respectively,andintheondemandregimenwere521,797.2 PPP, 93.46 and 10.99 respectively. The PSA confirmed the robustness of the model's results. The results of the scatter plots and acceptability curves showed that the prophylaxis regimen in 97% of the simulations for the thresholds below $20950 PPP was more cost-effective than on-demand regimen. Conclusion: Prophylaxis regimen showed the lower costs and higher effectiveness and utility in comparison with the on-demand regimen. It is recommended that prophylaxis should be considered as the standard care for treatment of hemophilic patients.
International Journal of Technology Assessment in Health Care, 2009
Objective: The aim of this study was to assess the incremental cost-effectiveness of on-demand versus prophylactic hemophilia therapy in Iran from a third-party payers' perspective. Methods: A retrospective chart review of twenty-five type A hemophiliacs who were treated in three hemophilia treatment centers was conducted. The patients were boys 0-9 years old receiving one of two treatments: (i) prophylaxis with concentrate at clinic; (ii) concentrate at clinic as on-demand. Fourteen boys received on-demand infusions for bleeding events, and eleven boys received infusions prophylaxis. Data were extracted from documents in the hemophilia treatment centers during a period of approximately 6 months. Results: The patients receiving prophylactic treatment had fewer bleeding events each month (mean, 0.26 versus 2.74) but used more concentrate (225.31 versus 87.20 units/kg per month). Average monthly cost per patient in the prophylaxis group was approximately 1.9 times higher than in the on-demand group. Compared with on-demand infusion, prophylaxis costs 3,201,656 Rials (€213.45) per bleeding event prevented. Conclusion: Prophylactic care markedly reduces the number of bleeding episodes, but at considerable cost.
Costs of the management of hemophilia A with inhibitors in Spain
Global & Regional Health Technology Assessment, 2021
Introduction: Emicizumab is a first-in-class monoclonal antibody, recently authorized for the treatment of hemophilia A with inhibitors. This study aims to estimate the direct and indirect costs of the management of hemophilia A with inhibitors, in adult and pediatric patients, including the prophylaxis with emicizumab. Methods: We calculated the costs of the on-demand and prophylactic treatments with bypassing agents (activated prothrombin complex concentrate and recombinant activated factor VII) and the emicizumab prophylaxis, from the societal perspective, over 1 year. The study considered direct healthcare costs (drugs, visits, tests, and hospitalizations), direct non-healthcare costs (informal caregivers), and indirect costs (productivity loss). Data were obtained from a literature review and were validated by an expert group. Costs were expressed in 2019 euros. Results: Our results showed that the annual costs of the prophylactic treatment per patient varied between €543,062.9...
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...
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 ...
Haemophilia, 2007
The first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil is currently activated prothrombin complex concentrate (aPCC), with recombinant activated factor VII (rFVIIa) used as second-line therapy or as a last resort. The aim of this study was to determine the cost and effectiveness of these treatments from the perspective of the Brazilian National Health Service. A decision analysis model was constructed to assess total direct medical costs (including drug costs, costs of outpatient or inpatient care, ambulance transportation and cost of concomitant medications) of first-line treatment with aPCC or rFVIIa. Clinical outcome and resource utilization data were obtained both retrospectively and prospectively and validated by the consensus of an expert panel of Brazilian haematologists. A total of 103 bleeds in 25 patients were included in the analysis. rFVIIa resolved bleeds more quickly (4.4 h) than aPCC (62.6 h) and was more effective (100% vs. 56.7% respectively). Mean total direct medical costs (from initiation to cessation of bleed) were estimated to be US$13 500 (aPCC) and US$7590 (rFVIIa). Extensive sensitivity analyses confirmed the costeffectiveness of rFVIIa. Compared with aPCC, rFVIIa was more effective and less expensive when used as first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil. rFVIIa should be considered a first-line treatment for the management of these patients.
Economic Burden of High-Responding Inhibitors in Patients with Hemophilia A in Taiwan
Yonsei Medical Journal, 2013
Purpose: Hemophilia A (HA) is the most common X-linked inherited bleeding disorder. In some patients with HA, particularly those with severe HA, replacement therapy results in the production of high-responding clotting factor VIII inhibitors. The economic burden of this complication is the highest reported for a chronic disease. Our aim was to investigate the direct medical expenditure burden of high-responding inhibitors in patients with HA. Materials and Methods: A retrospective study was conducted using the National Health Insurance Research Database, utilizing data covering the period of 2004-2007. Results: In total, 638 males with HA¸ including 37 patients with high-responding inhibitors were evaluated. Over 99% of the annual median medical expenditure was attributable to the cost of clotting factor concentrates (CFCs) in patients with high-responding inhibitors. The annual median expenditure related to CFCs of the total medical care and outpatient care were US$170611 and US$141982, respectively, and were 4.6-and 4.3-fold higher in these patients during the study period, respectively. In patients with high-responding inhibitors, the median hospitalization expenditure and daily hospitalization cost with or without surgical procedures were 3.0-and 2.4-fold higher, respectively, and 4.3 and 5.6-fold higher, respectively. Conclusion: Our data reveal higher medical expenditures burden for patients with HA and high-responding inhibitors in Taiwan. Future research is encouraged to evaluate the impact of this burden on patient quality of life.