Pharmacoeconomic studies of bypassing agents in mild-to-moderate bleeding episodes in patients with hemophilia and inhibitors: a critical appraisal (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.
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 ...
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...
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.
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.
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...
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.
Haemophilia, 2005
sus among the panellists was refined by a second round of the process, and the median values resulting were used as inputs to a decision analysis model. Sensitivity analyses were conducted to determine threshold values for key variables. The base case model found that initial treatment with aPCC would result in a mean cost per episode of 21000,comparedwith21 000, compared with 21000,comparedwith33 400 for initial treatment with rFVIIa. Sensitivity analyses over a range of clinically plausible values for cost, dosing, and efficacy did not change the selection of aPCC as the dominant strategy.
American health & drug benefits, 2011
No evidence-based treatment guidelines are currently available for the treatment of problem bleedings in patients with hemophilia who develop clotting factor inhibitors. A treatment algorithm was developed previously to help providers optimize the approach to the treatment of this patient population. The algorithm provides the specific intervals between treatments; however, it does not specify dosing recommendations and does not offer insights into the likelihood of outcome improvements at each time interval. To develop a model to analyze the impact on patient outcomes and costs of adhering to a current treatment algorithm for the 2 available clotting therapies to treat bleeding episodes in patients with hemophilia who develop clotting factor inhibitors. A simulation model was developed using a modified Delphi method approach based on a consensus opinion of an expert panel. The model was used to analyze the impact of following the available treatment algorithm on patient outcomes an...