Abstract:Background
Acquired 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.
Methods
In high‐titered AHA patients with bleeding … Show more
“…The adult patient population was similar to the one we previously described with a modification to the Markov decision analytic model (Fig. ) that was developed using Microsoft Excel (Microsoft Corporation, Redmond, WA, USA) so that these hypothetical hospitalized, high‐titred (>5 Bethesda units [BU]) AHA patients with bleeding could also be treated with rpFVIII (represented by Obizur, Baxter, Westlake Village, CA, USA) in addition to aPCC (represented by FEIBA, Shire, Lexington, MA, USA) and rFVIIa (represented by NovoSeven, Novo Nordisk, Plainsboro, NJ, USA) .…”
Section: Methodsmentioning
confidence: 99%
“…) that was developed using Microsoft Excel (Microsoft Corporation, Redmond, WA, USA) so that these hypothetical hospitalized, high‐titred (>5 Bethesda units [BU]) AHA patients with bleeding could also be treated with rpFVIII (represented by Obizur, Baxter, Westlake Village, CA, USA) in addition to aPCC (represented by FEIBA, Shire, Lexington, MA, USA) and rFVIIa (represented by NovoSeven, Novo Nordisk, Plainsboro, NJ, USA) . In brief, regardless of the treatment, similar to our previous study, the model allowed the patients to be transitioned into four different health states: (1) continuous bleeding, (2) thrombosis, (3) stop bleeding and (4) death . All patients entered the model at stage (1) because they were bleeding initially, and whether they remained in state (1) or entered the other states depended on the efficacy of the treatment and the probability of an adverse event.…”
Section: Methodsmentioning
confidence: 99%
“…As mentioned in our previously published study, the cost‐effectiveness analysis (CEA) was conducted from the US payer perspective , without inflation adjustment during the 6‐day study period . Because of the disease's low incidence, the heterogeneity of published studies, and the uncertainty around the costs and effectiveness of the treatment options, given a lack of comparative trials, our primary outcomes of interest were an estimation of the joint density of cost and effectiveness differences and the quantification of uncertainty surrounding the incremental cost‐effectiveness ratios (ICER, .…”
Section: Methodsmentioning
confidence: 99%
“…Traditionally, in bleeding patients whose factor VIII inhibitors are high‐titred, bypassing medications, including recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC), both FDA‐approved for treatment of this condition, should be initiated promptly . We previously showed that aPCC is a more cost‐effective option than rFVIIa in this clinical scenario . Recently, recombinant porcine‐sequence factor VIII (rpFVIII) was also approved by the FDA for this indication after a phase 2/3 multicentre trial showing its efficacy and safety .…”
Section: Introductionmentioning
confidence: 99%
“…Furthermore, rpFVIII is relatively more expensive compared to aPCC or rFVIIa . Focusing on the population of high‐titred AHA patients, given its high morbidity and mortality and the availability of three FDA‐approved therapies currently for the treatment of bleeding in these patients, from a global healthcare policy perspective, we adapted our previously published economic model to rigorously examine the cost‐effectiveness of the new treatment, namely, rpFVIII, against traditional options of aPCC or rFVIIa, using actual institutional costs, clinical outcome data and complication data obtained from the literature.…”
Background Acquired haemophilia A (AHA), with potentially high risk of morbidity and mortality, occurs as a result of inhibitors against factor VIII. Bleeding due to AHA can be treated with activated prothrombin complex concentrate (aPCC), recombinant activated factor VII (rFVIIa) or recently, recombinant porcine-sequence factor VIII (rpFVIII). We extended our previous cost-effectiveness analysis (CEA) comparing rpFVIII against the available traditional options.Methods For high-titred, haemorrhaging AHA patients treated with either aPCC, rFVIIa or rpFVIII, over the course of 6-days, a Markov simulation was conducted to evaluate the outcomes when these patients transitioned into any of the four following health states: (1) continuous bleeding, (2) thrombosis, (3) stop bleeding and (4) death, with states (2), (3) and (4) modelled as absorbing states. All model parameters were obtained from the medical literature, except the costs of aPCC, rFVIIa and the factor VIII assay, which came from our institutional data.Results Excluding the cost of the initial treatment on day 0, the total subsequent treatment cost of rFVIIa was substantially more than the costs of aPCC and rpFVIII ($13 925 vs. $1778 vs. $6957, respectively). The average quality-adjusted life days (QALDs) gained from rpFVIII was lowest (4Á89 vs. 4Á9 for rFVIIa and 4Á91 for aPCC). Overall, aPCC dominated the other two treatments. The model was determined to be robust across the tested ranges for all input variables.
ConclusionBased on this economic model, for AHA patients with high titres who were bleeding, aPCC was the most cost-effective treatment option and may be considered for use if there is no clinical contraindication.
“…The adult patient population was similar to the one we previously described with a modification to the Markov decision analytic model (Fig. ) that was developed using Microsoft Excel (Microsoft Corporation, Redmond, WA, USA) so that these hypothetical hospitalized, high‐titred (>5 Bethesda units [BU]) AHA patients with bleeding could also be treated with rpFVIII (represented by Obizur, Baxter, Westlake Village, CA, USA) in addition to aPCC (represented by FEIBA, Shire, Lexington, MA, USA) and rFVIIa (represented by NovoSeven, Novo Nordisk, Plainsboro, NJ, USA) .…”
Section: Methodsmentioning
confidence: 99%
“…) that was developed using Microsoft Excel (Microsoft Corporation, Redmond, WA, USA) so that these hypothetical hospitalized, high‐titred (>5 Bethesda units [BU]) AHA patients with bleeding could also be treated with rpFVIII (represented by Obizur, Baxter, Westlake Village, CA, USA) in addition to aPCC (represented by FEIBA, Shire, Lexington, MA, USA) and rFVIIa (represented by NovoSeven, Novo Nordisk, Plainsboro, NJ, USA) . In brief, regardless of the treatment, similar to our previous study, the model allowed the patients to be transitioned into four different health states: (1) continuous bleeding, (2) thrombosis, (3) stop bleeding and (4) death . All patients entered the model at stage (1) because they were bleeding initially, and whether they remained in state (1) or entered the other states depended on the efficacy of the treatment and the probability of an adverse event.…”
Section: Methodsmentioning
confidence: 99%
“…As mentioned in our previously published study, the cost‐effectiveness analysis (CEA) was conducted from the US payer perspective , without inflation adjustment during the 6‐day study period . Because of the disease's low incidence, the heterogeneity of published studies, and the uncertainty around the costs and effectiveness of the treatment options, given a lack of comparative trials, our primary outcomes of interest were an estimation of the joint density of cost and effectiveness differences and the quantification of uncertainty surrounding the incremental cost‐effectiveness ratios (ICER, .…”
Section: Methodsmentioning
confidence: 99%
“…Traditionally, in bleeding patients whose factor VIII inhibitors are high‐titred, bypassing medications, including recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC), both FDA‐approved for treatment of this condition, should be initiated promptly . We previously showed that aPCC is a more cost‐effective option than rFVIIa in this clinical scenario . Recently, recombinant porcine‐sequence factor VIII (rpFVIII) was also approved by the FDA for this indication after a phase 2/3 multicentre trial showing its efficacy and safety .…”
Section: Introductionmentioning
confidence: 99%
“…Furthermore, rpFVIII is relatively more expensive compared to aPCC or rFVIIa . Focusing on the population of high‐titred AHA patients, given its high morbidity and mortality and the availability of three FDA‐approved therapies currently for the treatment of bleeding in these patients, from a global healthcare policy perspective, we adapted our previously published economic model to rigorously examine the cost‐effectiveness of the new treatment, namely, rpFVIII, against traditional options of aPCC or rFVIIa, using actual institutional costs, clinical outcome data and complication data obtained from the literature.…”
Background Acquired haemophilia A (AHA), with potentially high risk of morbidity and mortality, occurs as a result of inhibitors against factor VIII. Bleeding due to AHA can be treated with activated prothrombin complex concentrate (aPCC), recombinant activated factor VII (rFVIIa) or recently, recombinant porcine-sequence factor VIII (rpFVIII). We extended our previous cost-effectiveness analysis (CEA) comparing rpFVIII against the available traditional options.Methods For high-titred, haemorrhaging AHA patients treated with either aPCC, rFVIIa or rpFVIII, over the course of 6-days, a Markov simulation was conducted to evaluate the outcomes when these patients transitioned into any of the four following health states: (1) continuous bleeding, (2) thrombosis, (3) stop bleeding and (4) death, with states (2), (3) and (4) modelled as absorbing states. All model parameters were obtained from the medical literature, except the costs of aPCC, rFVIIa and the factor VIII assay, which came from our institutional data.Results Excluding the cost of the initial treatment on day 0, the total subsequent treatment cost of rFVIIa was substantially more than the costs of aPCC and rpFVIII ($13 925 vs. $1778 vs. $6957, respectively). The average quality-adjusted life days (QALDs) gained from rpFVIII was lowest (4Á89 vs. 4Á9 for rFVIIa and 4Á91 for aPCC). Overall, aPCC dominated the other two treatments. The model was determined to be robust across the tested ranges for all input variables.
ConclusionBased on this economic model, for AHA patients with high titres who were bleeding, aPCC was the most cost-effective treatment option and may be considered for use if there is no clinical contraindication.
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