Cost of patients with hemophilia A treated with standard half-life or extended half-life FVIII in Spain

Kim, Hae Kyung; Peral, Carmen; Rubio-Rodríguez, Darío; Rubio-Terrés, Carlos

doi:10.1080/14737167.2020.1789457

Cited by 6 publications

(2 citation statements)

References 22 publications

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“…Patient-reported outcomes (humanistic burden, indirect costs, and direct non-medical costs) were available from a subset of the overall study cohort (40/75 patients), which may have reflected a selection bias specific to those willing to complete the patient questionnaire. Our findings were from 5 large European countries, whereas recent studies of EHL costs across haemophilia types and subgroups have tended to be single-center, single-country, or more tightly focused regional analyses [ 28 , 29 ].…”

Section: Discussionmentioning

confidence: 91%

Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

Burke

Asghar

O’Hara

et al. 2021

Orphanet J Rare Dis

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Background Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to better understand the clinical, humanistic, and economic burden of severe haemophilia B in Europe. Data from male adults with severe haemophilia B receiving prophylaxis were analysed from the retrospective cross-sectional CHESS II study conducted in Germany, France, Italy, Spain and the United Kingdom. Inhibitors were exclusionary. Patients and physicians completed questionnaires on bleeding, joint status, quality of life, and haemophilia-related direct and indirect costs (2019–2020). All outcomes were summarised using descriptive statistics. Results A total of 75 CHESS II patients were eligible and included; 40 patients (53%) provided self-reported outcomes. Mean age was 36.2 years. Approximately half the patients were receiving EHL versus standard half-life (SHL) prophylaxis (44% vs 56%). Most patients reported mild or moderate chronic pain (76%) and had ≥ 2 bleeding events per year (70%), with a mean annualised bleed rate of 2.4. Mean annual total haemophilia-related direct medical cost per patient was €235,723, driven by FIX costs (€232,328 overall, n = 40; €186,528 for SHL, €290,620 for EHL). Mean annual indirect costs (€8,973) were driven by early retirement or work stoppage due to haemophilia. Mean quality of life (EQ-5D) score was 0.67. Conclusions These data document a substantial, persistent real-world burden of severe haemophilia B in Europe. Unmet needs persist for these patients, their caregivers, and society.

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Section: Discussionmentioning

confidence: 91%

Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

Burke

Asghar

O’Hara

et al. 2021

Orphanet J Rare Dis

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“…By not converting from SHL to EHL rFVIII, patients in Spain could each save up to €53,078/year. 157 Finally, there are currently no financial structures for gene therapy in hemophilia A. The pharmaceutical company (Bio-Marin), which financed gene transfer research for hemophilia A, has priced Roctavian at $2.9 million for a one-time treatment.…”

Section: Affordability and Accessibility To Treatmentmentioning

confidence: 99%

Shifting Paradigms and Arising Concerns in Severe Hemophilia A Treatment

Chandran,

Tohit,

Stanslas

et al. 2024

Semin Thromb Hemost

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The management of hemophilia A has undergone a remarkable revolution, in line with technological advancement. In the recent past, the primary concern associated with Factor VIII (FVIII) concentrates was the risk of infections, which is now almost resolved by advanced blood screening and viral inactivation methods. Improving patients' compliance with prophylaxis has become a key focus, as it can lead to improved health outcomes and reduced health care costs in the long term. Recent bioengineering research is directed toward prolonging the recombinant FVIII (rFVIII) coagulant activity and synthesising higher FVIII yields. As an outcome, B-domain deleted, polyethylene glycolated, single-chain, Fc-fused rFVIII, and rFVIIIFc-von Willebrand Factor-XTEN are available for patients. Moreover, emicizumab, a bispecific antibody, is commercially available, whereas fitusiran and tissue factor pathway inhibitor are in clinical trial stages as alternative strategies for patients with inhibitors. With these advancements, noninfectious complications, such as inhibitor development, allergic reactions, and thrombosis, are emerging concerns requiring careful management. In addition, the recent approval of gene therapy is a major milestone toward a permanent cure for hemophilia A. The vast array of treatment options at our disposal today empowers patients and providers alike, to tailor therapeutic regimens to the unique needs of each individual. Despite significant progress in modern treatment options, these highly effective therapies are markedly more expensive than conventional replacement therapy, limiting their access for patients in developing countries.

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