Cost-utility analysis of life-long prophylaxis with recombinant factor VIIIFc vs recombinant factor VIII for the management of severe hemophilia A in Sweden

Henry, Nathaniel; Jovanović, Jelena; Schlueter, Max; Kritikou, P.; Wilson, Koo; Myrén, Karl-Johan

doi:10.1080/13696998.2017.1405816

Cited by 26 publications

(34 citation statements)

References 32 publications

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“…Indeed, Henry et al 38 BPAs represent a budget increase. However, our study shows that this increase in more than compensated by saving realized for patients under prophylaxis, as the overall results is dominance.…”

Section: Discussionmentioning

confidence: 99%

Cost‐effectiveness of emicizumab vs bypassing agents in the prevention of bleeding episodes in haemophilia A patients with anti‐FVIII inhibitors in France

Polack

Trossaërt²,

Cousin

et al. 2020

Haemophilia

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Introduction The development of an anti‐FVIII inhibitor is the most serious complication of haemophilia A occurring in up to 30% of severe haemophilic patients. The current management of haemophilia A with inhibitor uses bypassing agents (BPA) and represents a significant therapeutic burden together with a limited adherence to prophylactic treatment. Emicizumab is the first monoclonal antibody developed in haemophilia A approved for the prevention of bleeding episodes in patients with anti‐FVIII inhibitor. Aim The purpose of this study is to evaluate the incremental cost‐effectiveness ratio (ICER) of emicizumab versus BPAs. Methods A Markov model was developed over a five‐year time horizon to estimate the comparative costs and benefits of the different therapeutic approaches in this rare disease. Model inputs were clinical, including annual bleeding rate and quality of life, and economical including mainly costs of prophylaxis, bleeds and adverse events. Results Emicizumab treatment is dominant, ie lest costly and more effective, in the base‐case analysis saving 234 191 € for a gain of 0.88 QALY. This is confirmed by both the deterministic and probabilistic sensitivity analyses. The main limit of the study remains the absence of long‐term clinical data allowing to relate treatment consumption to clinical benefit, especially in the progression of haemophilic arthropathy. Conclusion Our results show that emicizumab is a cost‐effective treatment allowing to consider an easy to implement prophylactic treatment for haemophilia A patients with anti‐FVIII inhibitors.

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Section: Discussionmentioning

confidence: 99%

Cost‐effectiveness of emicizumab vs bypassing agents in the prevention of bleeding episodes in haemophilia A patients with anti‐FVIII inhibitors in France

Polack

Trossaërt²,

Cousin

et al. 2020

Haemophilia

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“…Because of their relatively short half‐life (approximately 12 hours), standard FVIII formulations require administration twice or three times daily to maintain haemostatic FVIII levels in the postoperative period. An extended half‐life FVIII may offer less frequent dosing and the possibility of earlier hospital discharge and attendant cost savings …”

Section: Introductionmentioning

confidence: 99%

Perioperative haemostasis with full‐length, PEGylated, recombinant factor VIII with extended half‐life (rurioctocog alfa pegol) in patients with haemophilia A: Final results of a multicentre, single‐arm phase III trial

et al. 2019

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IntroductionRurioctocog alfa pegol (BAX 855, TAK‐660) is a PEGylated, full‐length, recombinant factor VIII (rFVIII) with extended half‐life developed from unmodified rFVIII (antihaemophilic factor [recombinant]).AimTo determine the perioperative haemostatic efficacy and safety of rurioctocog alfa pegol in male previously treated patients (PTPs) with severe haemophilia A.MethodsThis multicentre, single‐arm, phase III study included PTPs who were to undergo major or minor elective or minor emergency surgical, dental or other invasive procedures. Rurioctocog alfa pegol dose and frequency were individualized based on patients’ pharmacokinetic profiles for major surgeries and by rurioctocog alfa pegol incremental recovery for minor surgeries. Haemostatic efficacy was assessed using the Global Haemostatic Efficacy Assessment score.ResultsTwenty‐one patients aged 16‐61 years underwent 21 major and five minor surgeries. For all 24 evaluable surgeries, overall haemostatic efficacy was rated as excellent and blood loss comparable to that expected in non‐haemophilic patients. No blood transfusions were required intraoperatively but were administered postoperatively for four surgeries in three patients. Five injury‐related postoperative bleeding episodes occurred in five patients, of which two required additional rurioctocog alfa pegol treatment. Two non‐serious adverse events of mild severity (increased ALT level and headache) were considered possibly related to rurioctocog alfa pegol. There were no deaths or treatment‐related serious adverse events. No patients developed inhibitory antibodies to FVIII or persistent IgG‐ or IgM‐binding antibodies to FVIII, PEG‐FVIII or PEG.ConclusionRurioctocog alfa pegol was well tolerated and effective for perioperative use in patients with haemophilia A and showed no signs of immunogenicity.

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“…national tenders, assay availability or cost [83,107]. Cost can be a substantial issue; however, longacting products have been associated with reduced consumption and may lead to long-term health savings, although it is important to note that direct treatment costs could increase [108]. This may be the case if patients are switching to longacting products to achieve higher trough levels, so retain similar doses and dosing regimens to their previous product.…”

Section: Ongoing Assessmentmentioning

confidence: 99%

Switching patients in the age of long-acting recombinant products?

Escobar¹,

Santagostino

Mancuso

et al. 2019

Expert Review of Hematology

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Introduction: Prophylaxis with factor replacement therapy is the gold standard for the treatment of hemophilia, but this often requires frequent infusions. A number of long-acting factor products have been developed to reduce the burden on patients. Areas covered: This is an overview of information presented at two symposia held at the World Federation of Hemophilia and International Society on Thrombosis and Haemostasis-Scientific and Standardization Committee annual meetings. The pharmacokinetic, safety and efficacy data for longacting recombinant products are reviewed, with a focus on recombinant factor IX albumin fusion protein (rIX-FP) and rVIII-SingleChain. This overview also provides a guide for managing a patient's switch to long-acting products. Expert opinion: Long-acting products may allow patients to maintain or decrease bleeding rates whilst increasing their dosing interval, which may in turn reduce the burden on patients and caregivers. When switching patients to long-acting products health-care professionals should provide balanced and thorough education to the patient, whilst supporting their emotional well-being. Regimens should address patients' needs and goals but should also be guided by clinical phenotype and pharmacokinetic assessment. Follow-up should assess safety concerns, bleeding rates, joint health and the impact of the regimen on patients' lifestyle.

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Cost-utility analysis of life-long prophylaxis with recombinant factor VIIIFc vs recombinant factor VIII for the management of severe hemophilia A in Sweden

Abstract: The results suggest that rFVIIIFc may be a cost-effective option for hemophilia A prophylaxis, generating greater quality of life and reduced costs for the Swedish payer compared to more frequently administered rFVIII alternatives.

Cited by 26 publications

References 32 publications

Cost‐effectiveness of emicizumab vs bypassing agents in the prevention of bleeding episodes in haemophilia A patients with anti‐FVIII inhibitors in France

Cost‐effectiveness of emicizumab vs bypassing agents in the prevention of bleeding episodes in haemophilia A patients with anti‐FVIII inhibitors in France

Perioperative haemostasis with full‐length, PEGylated, recombinant factor VIII with extended half‐life (rurioctocog alfa pegol) in patients with haemophilia A: Final results of a multicentre, single‐arm phase III trial

Switching patients in the age of long-acting recombinant products?

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