Hemophilia Burden of Disease: A Systematic Review of the Cost-Utility Literature for Hemophilia

Thorat, Teja; Neumann, Peter J.; Chambers, James D.

doi:10.18553/jmcp.2018.24.7.632

Cited by 45 publications

(57 citation statements)

References 49 publications

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“…This is the first model in hemophilia A able to demonstrate the benefit that gene therapy may offer across a wide range of hemophilia A patients. Prophylactic treatment costs and bleed management costs vary substantially within the hemophilia A population 47 . To best capture this variability, this microsimulation incorporates patient-level heterogeneity through patient body weight, ABR (0 to 16), and valoctocogene roxaparvovec durability (3 to 50 years, average 11 years).…”

Section: Discussionmentioning

confidence: 99%

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Cook

Forbes

Adamski

et al. 2020

Journal of Medical Economics

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Aim: Hemophilia A is a genetic, chronic disorder classified by deficient or defective coagulation factor VIII (FVIII) that puts those affected at risk for spontaneous bleeding episodes, which lead to joint damage and chronic pain over time. Currently, most severe hemophilia A patients are treated with prophylactic FVIII, which requires costly and frequent infusions and lifelong adherence to medication. A gene therapy (valoctocogene roxaparvovec) is currently in development for the treatment of severe hemophilia A. This model assessed the potential cost-effectiveness of treating patients with valoctocogene roxaparvovec rather than prophylactic therapy. Materials and methods: We developed an individual-based, state-transition microsimulation model for assessing the likely cost-effectiveness of valoctocogene roxaparvovec compared to prophylactic FVIII. Men aged 30 with severe hemophilia A were modeled over a lifetime horizon, and costs were reported from the perspective of the United States health care system. Through microsimulation, patient-level heterogeneity was captured in starting weight, starting Pettersson score (PS), durability of valoctocogene roxaparvovec, and annual bleed rate (ABR). Results: The model projects that treatment with single-administration valoctocogene roxaparvovec would be cost-saving to people with hemophilia A at a price point comparable to other currently available gene therapy products due to its potential to reduce FVIII utilization, direct medical costs, lifetime bleeds, and accumulated joint damage. Limitations: The model relies upon evidence-based assumptions for clinical inputs due to limited data availability. Such uncertainty was mitigated by modeling heterogeneity across the population, specifically with regards to long-term gene therapy durability, lifetime bleed rates, and joint damage progression. Conclusion: Valoctocogene roxaparvovec was found to be cost-saving-on average by about $6.8 million per patient-and more effective than prophylactic therapy for treatment of hemophilia A. The comparative benefit of gene therapy was observed across a broad range of simulated patients that were representative of the real-world severe hemophilia A population.

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Section: Discussionmentioning

confidence: 99%

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Cook

Forbes

Adamski

et al. 2020

Journal of Medical Economics

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“…If successful, immune tolerance therapy can enable effective FVIII replacement therapy and make prophylaxis with FVIII feasible again. However, immune tolerance therapy comes at a high cost, and involves frequent prolonged infusions, and is not always effective 6,18 …”

Section: Methodsmentioning

confidence: 99%

The societal burden of haemophilia A. I – A snapshot of haemophilia A in Australia and beyond

Brown

et al. 2020

Haemophilia

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IntroductionFew studies, both in Australia and overseas, have examined the social impacts of living with haemophilia A (HA) or the economic costs associated with the disorder. The purpose of this paper is to examine the epidemiology and societal burden of people with HA (PwHA) in Australia, with a particular focus on men with this disorder.MethodsThe epidemiology and societal burden of HA in Australia, with a particular focus on men with this disorder, were assessed, using data available in the Australian and international literature and publicly available data.ResultsThe mean annual prevalence of HA is approximately 1‐2 per 10 000 males. Prophylactic treatment is used in one‐quarter (25.1%) of people with moderate HA, and 82.2% of people with severe HA. Within the latter group, 16.1% have inhibitors for Factor VIII, predisposing them to worse morbidity, mortality and quality of life when compared to the non‐inhibitor population. Joint pain and joint disease occur commonly in PwHA, with up to 70% of adults with HA experiencing joint problems. HA is associated with poor physical health, and PwHA miss school and work due to bleeding‐related events.ConclusionHA is associated with substantial economic burden; with large differences in costs reported between countries. Overall, HA imposes a significant burden of disease on PwHA, their families and the community at large.

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“…Therefore, antiviral therapy, especially in the new DAA era is an imperative in this group of patients, especially taken in account the severe burden of the healthcare system with the life-long substitution therapy for haemophilia and possible complications of untreated HCV infection. Estimated annual treatment cost for patients with severe haemophilia in Serbia is between 100,000-200,000 EUR per patient [28,29]. However, these patients in Serbia have a very poor quality of life, especially due to consequences of untreated and/or unrecognised HCV infection.…”

Section: Patients With Haemophilia and Chc Infectionmentioning

confidence: 99%

Micro-elimination of HCV as a possible therapeutic strategy: our experience and a review of literature

Bojović¹,

Simonovic-Babic²,

Mijailović

et al. 2020

J Infect Dev Ctries

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Background: Serbia has an intermediate estimated prevalence of chronic hepatitis C (CHC) infection, approximately 1.13%, with hepatitis C remaining one of the leading causes of liver-related morbidity and mortality in Serbia with impaired quality of life and overwhelming cost of treating its complications As the availability of new treatment options and resources for screening remains limited, micro-elimination of CHC becomes a top priority. Methods: Review of the available published data related to the clinical and epidemiological situation of the hepatitis C infection in Serbia, including the unpublished data from the databases of four major reference centres in Serbia (Clinical Center Serbia, Clinical Center Niš, Clinical Center Vojvodina and Clinical Center Kragujevac). Results: Currently in Serbia, micro-elimination appears to be realistic in the patients with haemophilia, who represent a small, well-defined subpopulation, under constant monitoring by the healthcare system. Other feasible targets for micro-elimination of CHC infection in Serbia are patients on hemodialysis, prisoners and people who inject drugs. Conclusions: Micro-elimination is feasible in Serbia, especially in the subpopulation of patients with haemophilia. This may represent an initial step towards achieving the WHO objective to eliminate hepatitis C infection by 2030.

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Hemophilia Burden of Disease: A Systematic Review of the Cost-Utility Literature for Hemophilia

Cited by 45 publications

References 49 publications

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

The societal burden of haemophilia A. I – A snapshot of haemophilia A in Australia and beyond

Micro-elimination of HCV as a possible therapeutic strategy: our experience and a review of literature

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