Haemophilia prophylaxis: how can we justify the costs?

Feldman, Brian M.; Berger, Karin; Bohn, Rhonda L.; Carção, Manuel; Fischer, Kathelijn; Gringeri, A.; Hoots, Keith; Mantovani, LG; Willan, Andrew R.; Schramm, W.

doi:10.1111/j.1365-2516.2012.02790.x

Cited by 17 publications

(24 citation statements)

References 59 publications

(72 reference statements)

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“…In conclusion, based on French patients with identical clinical outcomes, QoL, and WFH joint scores, EQOFIX, the largest medicoeconomic and QoL study of hemophilia B treatment, demonstrated that rFIX treatment is more expensive than pdFIX treatment with no difference in QoL or clinical results. This finding must be considered as FIX represents more than 90% of the total cost of the pathology and as the overall health care resources are becoming more and more restrained worldwide …”

Section: Discussionmentioning

confidence: 99%

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EQOFIX: a combined economic and quality‐of‐life study of hemophilia B treatments in France

et al. 2015

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BACKGROUND EQOFIX is a medicoeconomic study that analyzed the health‐related quality of life (HRQoL) and costs of care of the moderate and severe forms of hemophilia B, treated on demand or by prophylaxis with either plasma‐derived Factor IX (pdFIX) or recombinant FIX (rFIX). STUDY DESIGN AND METHODS The primary objectives were evaluations of the impact of hemophilia B on HRQoL and of the costs associated with its management. The secondary objectives were evaluations of the clinical efficacy and costs of care of pdFIX and rFIX. In this observational study we included and followed for 1 year severe and moderate hemophilia B patients without inhibitor. HRQoL was evaluated through generic and disease‐specific questionnaires. Information on the health resources consumed was collected every 3 months. RESULTS The EQOFIX cohort was composed of 155 patients, including 51 children and 104 adults, with 114 having severe disease and 41 having moderate disease. The regimens were prophylactic for 61 and on demand for 94. Altogether, 78 were treated with rFIX and 77 with pdFIX. There was no difference in the QoL between the pdFIX and rFIX treatments. The extra cost of prophylaxis was €22,605 per bleeding event prevented. The consumption of FIX was 1.4‐fold higher for the patients treated with rFIX than for the patients treated with pdFIX. CONCLUSION Our findings in a cohort composed of 25% of the French population of moderate and severe hemophilia B patients show, with similar clinical and HRQoL results, that treatment with rFIX is more expensive than treatment with pdFIX.

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Section: Discussionmentioning

confidence: 99%

“…The availability of efficacious treatment has substantially improved the therapeutic management of hemophilia patients. However, because the disease necessitates multidisciplinary care and due to the cost of medicinal products, the treatment of persons with hemophilia illustrates the impact of biotechnology on the management of patients with orphan diseases …”

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confidence: 99%

EQOFIX: a combined economic and quality‐of‐life study of hemophilia B treatments in France

et al. 2015

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“…However, one of the main limitations of FVIII prophylaxis is the frequency of infusions, which are typically required at least three to four times per week . In addition, given the quantity of FVIII required for prophylaxis, these regimens can be costly .…”

Section: Introductionmentioning

confidence: 99%

Predicting the outcomes of using longer‐acting prophylactic factor VIII to treat people with severe hemophilia A: a hypothetical decision analysis

Miners

Krishnan

Pasi

2016

Journal of Thrombosis and Haemostasis

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Essentials No randomized trials have compared long-acting factor VIII (FVIII) with currently used products. A comparison was undertaken using a decision model to predict FVIII use and number of bleeds. In the base case, longer acting FVIII reduced factor use by 17% while resulting in similar bleeds. The value of longer acting FVIII will be largely determined by existing regimens and unit price. Click to hear Prof. Makris's presentation on new treatments in hemophilia SUMMARY: Background Recently, factor VIII (FVIII) products with longer half-lives, such as recombinant FVIII Fc fusion protein (rFVIIIFc), have become available. Use of longer-acting FVIII products will largely depend on effectiveness and cost; no direct evaluations have compared these parameters between conventional and longer-acting FVIII therapies. Objectives To present a hypothetical decision analysis, combining evidence from multiple sources to estimate bleeding frequency, resource use and cost of longer-acting prophylactic products, such as rFVIIIFc, vs. conventional recombinant FVIII (rFVIII). Patients/Methods The decision model used published pharmacokinetic parameters, bleeding frequency vs. time information below a 1-IU dL FVIII trough level, and adherence. Prophylactic treatment scenarios were modelled for a hypothetical patient with severe hemophilia A (<1 IU/dL) receiving rFVIIIFc or rFVIII. Results Infusing twice weekly with rFVIIIFc 42.7 IU kg per dose required less clotting factor than infusing every 56 h with rFVIII 33.75 IU kg per dose; annual bleeding rates were similar. Base case analysis suggested that total FVIII costs were equated when rFVIIIFc cost 1.18 times more per IU than rFVIII, assuming similar adherence. Other modelled scenarios produced similar results, although differences in FVIII consumption were particularly sensitive to assumptions regarding frequency and dose of the rFVIII and rFVIIIFc regimens. For example, decreasing rFVIII from 33.75 IU kg to 30 IU kg per dose decreased the price factor to 1.05. Conclusions Longer-acting FVIII products may reduce FVIII consumption and infusion frequency without compromising hemostatic effect; this should be considered along with other factors (e.g. adherence and underlying FVIII regimen) when evaluating a suitable price for these agents.

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“…However, it also imposes a barrier to health systems and payers, which need to record and justify drivers of treatment costs, and thereby to control costs and predict future procurement needs [66][67][68]. Accurate, real-time, patient-level cumulative data also facilitates benchmarking within a single patient over time and among patients, facilitating comparisons of outcomes between different geographies, hemophilia care centers, treatment regimens, or physician practices [69].…”

Section: Innovative Outcome-based Care and Procurement Modelmentioning

confidence: 99%

An innovative outcome-based care and procurement model of hemophilia management

Gringeri¹,

Doralt²,

Valentino

et al. 2016

Expert Review of Pharmacoeconomics & Outcomes Research

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Hemophilia is a rare bleeding disorder associated with spontaneous and post-traumatic bleeding. Each hemophilia patient requires a personalized approach to episodic or prophylactic treatment, but selfmanagement can be challenging for patients, and avoidable bleeding may occur. Patient-tailored care may provide more effective prevention of bleeding, which in turn, may decrease the likelihood of arthropathy and associated chronic pain, missed time from school or work, and progressive loss of mobility. A strategy is presented here aiming to reduce or eliminate bleeding altogether through a holistic approach based on individual patient characteristics. In an environment of budget constraints, this approach would link procurement to patient outcome, adding incentives for all stakeholders to strive for optimal care and, ultimately, a bleed-free world.ARTICLE HISTORY

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Haemophilia prophylaxis: how can we justify the costs?

Cited by 17 publications

References 59 publications

EQOFIX: a combined economic and quality‐of‐life study of hemophilia B treatments in France

EQOFIX: a combined economic and quality‐of‐life study of hemophilia B treatments in France

Predicting the outcomes of using longer‐acting prophylactic factor VIII to treat people with severe hemophilia A: a hypothetical decision analysis

An innovative outcome-based care and procurement model of hemophilia management

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