Pharmacoeconomic analysis of recombinant factor VIIa versus APCC in the treatment of minor-to-moderate bleeds in hemophilia patients with inhibitors (original) (raw)
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Vox Sanguinis, 2018
BackgroundAcquired haemophilia A (AHA) is an autoimmune bleeding disorder with significant morbidity and mortality. Bleeding AHA patients with high titre inhibitors can be treated with either activated prothrombin complex concentrate (aPCC) or recombinant activated factor VII (rFVIIa). Given that both replacement therapies have inherent benefits and limitations, a cost‐effectiveness analysis (CEA) was performed in this population to compare rFVIIa with aPCC.MethodsIn high‐titered AHA patients with bleeding treated with either aPCC or rFVIIa, during a 5‐day study period, a Markov model was developed such that these patients were transitioned into four different health states: (1) continuous bleeding, (2) thrombosis, (3) stop bleeding and (4) death, with states (2), (3) and (4) modelled as absorbing states. Model parameters, including probabilities, health utility index and costs, were gathered from the medical literature, except for the costs of aPCC and rFVIIa, which were obtained f...
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.
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...
Haemophilia, 2009
The clinical, humanistic and economic consequences associated with haemophilia and inhibitors are considerable. Primary treatment for mild-to-moderate bleeding disorders in such patients is recombinant factor VIIa (rFVIIa) or activated prothrombin complex concentrate (APCC). The aims of this study were to identify, review and evaluate the quality of the published literature on the relative cost-effectiveness of rFVIIa and APCC in treating haemophilia patients with inhibitors. The review concentrates on model type, design and assumptions, and results. The results of this study suggest that rFVIIa may be the cost-effective alternative to treatment with APCC. In seven out of the nine studies, rFVIIa had the lower average treatment cost. The difference in average treatment cost to resolve a bleed, between rFVIIa and APCC in these seven studies, ranged from 3000to3000 to 3000to17 000. The adapted modelling framework is similar in all the economic models reviewed, suggesting clinical acceptability of the approach used. The estimates of efficacy varied between the models, especially for APCC. The efficacy for APCC derived from retrospective studies was lower than reported in the literature. Sensitivity analysis was undertaken in the majority of the economic analyses and the results were found to be robust to realistic parameter variations. Only one of the studies was a cost-utility study, showing the lack of measuring health status within this area. This systematic review showed that models based on different sources of data produced fairly similar robust results despite differences in the estimates of efficacy, average dosage required, and unit costs. However, ideally there should be a systematic approach to identifying the relevant data.
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 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....
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, 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.
ClinicoEconomics and Outcomes Research, 2021
Background: The standard of care for patients with hemophilia A is prophylaxis with factor VIII (FVIII) therapies. Extended half-life (EHL) FVIII products offer a reduced infusion burden compared with standard FVIII treatments. However, comparative evidence between EHLs is lacking. Objective: To develop a pharmacodynamic-pharmacokinetic decision model to predict comparative bleed outcomes of adolescents and adults with hemophilia A receiving treatment with various EHL FVIII therapies, capturing differences in cumulative bleeding episodes, breakthrough bleed resolution and resource costs, as well as quality-adjusted life years (QALYs). Methods: The patient population from the pathfinder 2 Phase III clinical trial was used to understand the link between FVIII levels and annual bleeding rates (ABRs). Pharmacokinetic/pharmacodynamic modeling was subsequently applied to estimate FVIII levels for four EHL FVIII treatments (turoctocog alfa pegol [Esperoct ® ], rurioctocog alfa pegol [Adynovi ® ], efmoroctocog alfa [Elocta ® ], and damoctocog alfa pegol [Jivi ® ]) to predict comparative ABRs. FVIII consumption costs (due to prophylactic treatment and breakthrough bleed resolution) and resource costs, as well as QALYs, were subsequently estimated from a UK NHS perspective over a 70-year time horizon. Results: Turoctocog alfa pegol prophylaxis resulted in 8-19% fewer cumulative bleeding episodes versus comparators in the base case scenario. Assuming parity in annual prophylaxis costs, turoctocog alfa pegol prophylaxis reduced the cost of product and resource use to resolve a breakthrough bleed by 9-25% versus comparators. Prophylaxis with turoctocog alfa pegol was also associated with the most QALYs, representing a discounted QALY gain of 0.35-1.05 compared with the other treatments. Conclusion: Using a pharmacodynamic-pharmacokinetic decision model, turoctocog alfa pegol prophylaxis was associated with fewer cumulative bleeds, as well as lower product and resource costs related to resolving a breakthrough bleed and most QALYs versus comparators.
Value in Health, 2017
Background: Hemophilia A is a factor VIII deficiency, associated with spontaneous, recurrent bleeding episodes. This may lead to comorbidities such as arthropathy and joint replacement, which contribute to morbidity and increased health care expenditure. Recombinant factor VIII Fc fusion protein (rFVIIIFc), a prolonged half-life factor therapy, requires fewer infusions, resulting in reduced treatment burden. Objective: Use a budget impact analysis to assess the potential economic impact of introducing rFVIIIFc to a formulary from the perspective of a private payer in the United States. Methods: The budget impact model was developed to estimate the potential economic impact of adding rFVIIIFc to a private payer formulary across a 2-year time period. The eligible patient population consisted of inhibitor-free adults with severe hemophilia A, receiving recombinant-based episodic or prophylaxis treatment regimens. Patients were assumed to switch from conventional recombinant factor treatment to rFVIIIFc. Only medication costs were included in the model. Results: The introduction of rFVIIIFc is estimated to have a budget impact of 1.4% ($0.12 per member per month) across 2 years for a private payer population of 1,000,000 (estimated 19.7 individuals receiving treatment for hemophilia A). The introduction of rFVIIIFc is estimated to prevent 124 bleeds across 2 years at a cost of $1891 per bleed avoided. Conclusions: Hemophilia A is a rare disease with a low prevalence; therefore, the overall cost to society of introducing rFVIIIFc is small. Considerations for comprehensively assessing the budget impact of introducing rFVIIIFc should include episodic and prophylaxis regimens, bleed avoidance, and annual factor consumption required under alternative scenarios.