Budget Impact Analysis of Prolonged Half-Life Recombinant FVIII Therapy for Hemophilia in the United States

McMullen, Suzanne; Buckley, Brieana; Hall, Eric; Kendter, J; Johnston, Karissa

doi:10.1016/j.jval.2016.09.2396

Cited by 15 publications

(12 citation statements)

References 19 publications

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“…Although initial estimates of outcomes of extended half-life factor products suggest that they are efficacious and safe [ 20 , 23 , 24 , 34 ], more information is needed on their impact on quality of life. Since caregivers are greatly impacted by hemophilia, it would be worthwhile to know if such treatments also have a beneficial effect on caregivers of people with hemophilia.…”

Section: Introductionmentioning

confidence: 99%

The impact of extended half-life versus conventional factor product on hemophilia caregiver burden

Schwartz

Powell

Su³

et al. 2018

Qual Life Res

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IntroductionExtended half-life factor products have reduced annualized bleeding rates in hemophilia patients. The impact of extended half-life versus conventional factor products on hemophilia caregiver burden has not been investigated. This study aimed to evaluate caregiver burden in extended half-life versus conventional factor products for hemophilia A and B.MethodsThis cross-sectional web-based study of caregivers of people with hemophilia A or B was recruited from a panel research company and by word of mouth. Participants completed the Hemophilia Caregiver Impact measure, the PedsQL Family Impact Module (PedsQL), and the Work Productivity and Activity Impairment Questionnaire (WPAI). We also collected demographic, insurance coverage, and medical information related to the hemophilia patient(s). Burden differences were assessed using linear regression and matched cohort analyses.ResultsThe sample (n = 448) included 49 people who were caring for people on extended half-life factor products. Worse caregiver burden was associated with more infusions per week and more bleeds in the past 6 months. Regression analyses suggested that caring for someone who is on a extended half-life factor product is associated with lower emotional impact (β = − 0.11, p < 0.05, Adjusted R2 = 0.06), and shows a trend association with lower practical impact (β = − 0.09, p < 0.10, Adjusted R2 = 0.05). The matched cohort analysis also revealed that people on extended half-life factor product had lower Emotional Impact and Practical Impact scores (t = − 2.95 and − 2.94, respectively, p < 0.05 in both cases). No differences were detected on the PedsQL or the WPAI.ConclusionThe reduced required frequency of factor product infusions of extended half-life factor products appears to reduce the emotional distress and practical burden of caregiving. Future work should evaluate the longitudinal impact.

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Section: Introductionmentioning

confidence: 99%

The impact of extended half-life versus conventional factor product on hemophilia caregiver burden

Schwartz

Powell

Su³

et al. 2018

Qual Life Res

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“…Moreover, rFVIIIFc allows a significant reduction in the number of administrations, with decrease median annual bleeding rate [5, 7, 10, 36], thus impacting on the general condition of HA patients, QoL and costs.…”

Section: Discussionmentioning

confidence: 99%

Budget impact analysis of the use of extended half-life recombinant factor VIII (efmoroctocog alfa) for the treatment of congenital haemophilia a: the Italian National Health System perspective

Lorenzoni

Triulzi

Turchetti

2018

BMC Health Serv Res

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BackgroundCongenital haemophilia A (HA) is a rare, inherited, life-long bleeding disorder characterised by prolonged or spontaneous bleeding due to the lack of clotting factor VIII (FVIII) in the body. Treatment for HA involves FVIII replacement therapy and poses great economic burden to National Health Systems and to society. Availability of novel products as extended half-life clotting factor products might change treatment approches and their economic evaluation is essential for an informed treatment choice. Accordingly the objective of the present work is to analyse the economic impact of using efmoroctocog alfa (recombinant factor VIII-Fc fusion protein, rFVIIIFc) for the treatment of children and adults with severe congenital haemophilia A (HA).MethodsA budget impact analysis was performed to estimate the economic impact of the introduction of rFVIIIFc in the market-mix of products for the treatment of HA. The analysis condidered a 3-year time horizon and the Italian National Health System (INHS) perspective. The model estimated drug costs associated with the treatment of HA in the current scenario - representing the marketplace forecast for the time period of interest assuming that rFVIIFc is not introduced - and a new scenario, assuming that rFVIIIFc is available in the market. The size of the target population was calculated using epidemiological national data. Univariate one-way sensitivity analyses and scenario analyses were performed.ResultsOverall 3-year costs of treating the HA population in the current scenario were 555,277,691 Euro for the INHS. With the introduction of rFVIIIFc, the costs were reduced to 541,897,466 Euro suggesting potential savings to the INHS of 13,380,255 Euro. Results were consistent at variation of most of the model’s parameters; only in case of lower dosage of conventional products and higher dosage of rFVIIIFc, costs for the INHS increased, in both cases, of about 20 million Euro.ConclusionsThe use of rFVIIIFc for the treatment of HA has been recently approved by the Italian Medicines Agency (AIFA) and this is the first study estimating the financial impact of this new therapeutic alternative in the Italian context. The analysis suggests that rFVIIIFc use does not result in higher expenditure for the INHS.

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“…Prophylaxis is considered as the gold standard for countries in which it is economically affordable [41,85,86], and it is associated with a better health-related quality of life (HRQoL) as compared to episodic treatment [87]. Furthermore, it leads to a decrease in bleeding-related hospitalization, shortens length of hospital stay, and thus reduces resource utilization [85,88].…”

Section: Current Evidence On the Impact Of Quality Of Life And Healthmentioning

confidence: 99%

“…In an analysis of the potential financial consequences of introducing rFVIIIFc to a private payer system in the United States, rFVIIIFc was anticipated to have a budget impact of 1.4% across 2 years for a private payer population of 1,000,000 through reducing the ABR by approximately 3.1 bleeds per individual patient with hemophilia A [85]. The total population budget was predicted to decrease for episodic treatment with the introduction of rFVIIIFc based on the lower factor consumption data observed in the ASPIRE trial [63,85].…”

Section: Current Evidence On the Impact Of Quality Of Life And Healthmentioning

confidence: 99%

Extended Half-Life (EHL) Coagulation Factors: A New Era in the Management of Haemophilia Patients

Balkan

Kavaklı

2019

Tjh

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Despite effective factor replacement and various treatment schedules, there remain several challenges and unmet needs in the prophylactic treatment of hemophilia limiting its adoption and thereby posing an increased risk of spontaneous bleeding. In this regard, extended half-life (EHL) recombinant factor VIII (rFVIII) and factor IX (rFIX) products promise optimal prophylaxis by decreasing the dose frequency, increasing the compliance, and improving the quality of life without compromising safety and efficacy. EHL products might lead to higher trough levels without increasing infusion frequency, or could facilitate the ability to maintain trough levels while reducing infusion frequency. This paper aims to provide a comprehensive review of the rationale for developing EHL coagulation factors and their utility in the management of hemophilia, with special emphasis on optimal techniques for half-life extension and criteria for defining EHL coagulation factors, as well as indications, efficacy, and safety issues of the currently available EHL-rFVIII and EHL-rFIX products. Potential impacts of these factors on quality of life, health economics, and immune tolerance treatment will also be discussed alongside the challenges in pharmacokinetic-driven prophylaxis and difficulties in monitoring the EHL products with laboratory assays.

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Budget Impact Analysis of Prolonged Half-Life Recombinant FVIII Therapy for Hemophilia in the United States

Cited by 15 publications

References 19 publications

The impact of extended half-life versus conventional factor product on hemophilia caregiver burden

The impact of extended half-life versus conventional factor product on hemophilia caregiver burden

Budget impact analysis of the use of extended half-life recombinant factor VIII (efmoroctocog alfa) for the treatment of congenital haemophilia a: the Italian National Health System perspective

Extended Half-Life (EHL) Coagulation Factors: A New Era in the Management of Haemophilia Patients

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