Haemophilia A: Pharmacoeconomic Review of Prophylaxis Treatment versus On-Demand (original) (raw)
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Haemophilia, 2013
SummaryProphylaxis has been established as the treatment of choice in children with haemophilia and its continuation into the adult years has been shown to decrease morbidity throughout life. The cost of factor therapy has made the option questionable in cost‐effectiveness studies. The role of prophylaxis in pharmacokinetic dosage and tolerization against inhibitor formation were used to model the cost utility of prophylaxis vs. on‐demand (OD) therapy over a lifetime horizon in severe haemophilia A. The model was applied to a single provider national health system exemplified by the United Kingdom's National Health Service and a third party provider in the United States. The incremental cost‐effectiveness ratio (ICER) was estimated and compared to threshold values used by payer agencies to guide reimbursement decisions. A cost per quality‐adjusted life year (QALY) was also estimated for Sweden. Prophylaxis was dominant over OD treatment in the UK. The model resulted in an ICER –...
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.
Cost–utility analysis of prophylaxis versus treatment on demand in severe hemophilia A
ClinicoEconomics and Outcomes Research, 2011
Background: Individuals with severe hemophilia A have reduced blood levels of clotting factor VIII (FVIII) leading to recurrent bleeding into joints and muscles. Primary prophylaxis with clotting factor concentrates started early in childhood prevents joint bleeds, thus avoiding joint damage and improving people's quality of life. There remain significant differences in the implementation of primary prophylaxis worldwide mainly due to the cost of prophylaxis compared with treatment on demand. Objective: To evaluate the cost-effectiveness of primary prophylaxis with FVIII concentrates versus secondary prophylaxis, versus treatment on demand, and versus a "hybrid" (primary prophylaxis followed by on-demand treatment in adults) in individuals with severe hemophilia A. Methods: A Markov model was developed and run using different sources of clinical, cost, and utility data. The model was populated with a hypothetical cohort of 100 individuals with severe hemophilia A. The perspective of the Italian National Health System was used. Results: The baseline results showed that primary and secondary prophylaxis is cost-effective compared both with treatment on demand and with a hybrid strategy. The incremental costs per quality-adjusted life-year gained for individuals with hemophilia A receiving primary and secondary prophylaxis were €40,229 to €40,236 versus an on-demand strategy. However, the sensitivity analyses performed showed that the results were sensitive to the unit cost of clotting FVIII, bleeding frequency, and the discount rate. Conclusion: Although primary prophylaxis is a costly treatment, our results show that it is cost-effective compared with treatment on demand.
Cost-of-illness study of severe haemophilia A and B in five French haemophilia treatment centres
Pharmacy World & Science, 2007
Objective The aim of this study was to assess the consumption of anti-haemophilic drugs by adults and children with severe haemophilia A or B (residual activity of FVIII or FIX B2%) and to quantify the average direct medical costs. Method A retrospective multicentre costof-illness study from the perspective of French national health insurance system. The costs include only the use of clotting factors. Main outcome measure Consumption was expressed in UI/kg/year and costs in euros/kg/year. Results From January 1, 2001 to December 31, 2002, data from 81 adults and 30 children with severe haemophilia A (n = 92) or B (n = 19) and included in the ''SNH'' were collected and analysed. A coagulation factor inhibitor was present in 10 patients (9%). Four of them were high responders. Mean age and body weight were respectively 28 ± 17 years and 58 ± 24 kg. Except for one adult patient, all (99%) had outpatient treatment, 44 patients (40%) were hospitalized and treated by recombinant or/ and plasma-derived FVIII or FIX or/and rFVIIa. Overall median annual consumption of anti-haemophilic drugs per patient was estimated at 1,333 UI/kg, with a median costof-illness of 1,156 euros/kg. Patients with severe haemophilia B consumed more than patients with severe haemophilia A, though not significantly (P = 0.096), with a median of 2,167 vs. 1,100 UI/kg/year and a median cost of 1,760 vs. 917 euros/kg/year (P = 0.13). Children consumed respectively more than adults (P = 0.008), with a median of 3,204 vs. 1,106 UI/kg/year and a median cost of 2,614 vs. 913 euros/kg/year (P = 0.012). The median cost for patients with an inhibitor was 3,291 euros/kg/year, approximately threefold higher than that of patients without an inhibitor (926 euros/kg/year) (P = 0.022). Conclusion It suggests a higher consumption and cost of anti-haemophilic drugs among children when compared to adults. Haemophilia B patients did not consume significantly more than haemophilia A patients, whereas the consumption and cost for patients with or without inhibitors differed significantly.
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.
Costs of on-demand and prophylactic treatment for severe haemophilia in Norway and Sweden
Haemophilia, 2004
The expected annual cost (in the year 2000 prices) for a 30-year-old patient with average individual and treatment characteristics for on-demand EUR 51 832 (95% CI: 44 324-59 341) and for prophylaxis EUR 146 118 (95% CI: 129 965-162 271), was obtained from panel-data analysis of an 11-year retrospective panel of 156 patients with severe haemophilia in Norway and Sweden. Costs included haemophilia-related treatment costs within the health-care sector (factor concentrate, doctorsÕ visits, diagnostic procedures, hospitalisation, invasive procedures, etc.) and cost for haemophilia-related resource use in other sectors (lost production, use of special equipment, adaptation of workplace and domicile, etc). Although costs of lost production, reconstructive surgery and hospitalisation were higher for on-demand, they did not balance out the higher costs of factor-concentrate consumption in prophylaxis. The cut-off risk of premature death, where on-demand and prophylaxis would have been equally costly, was 3.7 percentage units higher for on-demand than for prophylaxis. Such a great risk difference has not been reported elsewhere to our knowledge. Estimated cost-elasticities indicated that annual costs of prophylaxis would increase by approximately the same proportion as a potential increase in the price of factor concentrate and decrease less than proportionately with a reduction in prescribed dose kg )1 . For on-demand, the annual costs would increase by approximately the same proportion as an increase in the prescribed dose kg )1 .
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.
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.