A cost-effectiveness analysis of the prophylaxis versus on-demand regimens in severe hemophilia A patients under 12 years old in southern Iran

Zahedi, Zohreh; Karimi, Mehran; Keshavarz, Khosro; Haghpanah, Sezaneh; Ravangard, Ramin

doi:10.1080/16078454.2021.1885123

Cited by 6 publications

(1 citation statement)

References 32 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…However, this factor, in addition to the costs with overall treatment, imposes a significant direct costs to the SUS. To reduce this economic burden in the early days of life and potentially for adults with the condition, a prophylaxis regimen is advantageous due to the higher effectiveness and utility when compared with an on-demand regimen (Zahedi et al, 2021).…”

Section: Discussionmentioning

confidence: 99%

Analysis of epidemiological and economic data of hemophilia A patients in Brazil

Sarmento¹,

Picoli²,

Renni³

et al. 2022

JBES

View full text Add to dashboard Cite

Objective: To describe the annual medical direct costs per hemophilia A (HA) patient in the Brazilian public healthcare system (SUS) and to understand and describe the patients’ hospital journey, demographical characteristics, and the procedures in the. Methods: This retrospective analysis of DataSUS databases. Data from individuals with registries of HA treatment were gathered between January 1st, 2018, and June 30th, 2021. Besides the D66 ICD-10th code (HA), were also considered the occurrence of some procedures like factor VIII dosage and by-pass therapy dispensation or administration as inclusion criteria. Exclusion criteria were occurrence of factor IX dispensation and female patients were excluded, among others. A record linkage using sociodemographic characteristics was conducted to identify unique patients. Results: Were identified 2,298 individuals underwent ambulatory and 1,018 underwent hospital treatments. The results show that most patients are from the Southeast region of the country, white and middle-aged individuals. The median cost of HA treatment per patient-year was BRL 90.36 for ambulatory care and BRL 1,015.31 for hospital care procedures. The costs were significantly higher for more severe patients and for those between 12 and 18 years old (BRL 1,974.75 and BRL 1,049.09, respectively). Conclusion: The evidence demonstrated encourages the implementation of policies aiming to improve the quality of care provided to patients with HA. Providing referral centers for hemophiliac patients is primordial for the success of the treatment and can result in efficiency.

show abstract

Section: Discussionmentioning

confidence: 99%

Analysis of epidemiological and economic data of hemophilia A patients in Brazil

Sarmento¹,

Picoli²,

Renni³

et al. 2022

JBES

View full text Add to dashboard Cite

show abstract

Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors

Pochopien,

Tytuła,

Toumi

et al. 2024

Adv Ther

View full text Add to dashboard Cite

Introduction Recombinant factor IX (rFIX) and recombinant FIX Fc fusion protein (rFIXFc) are standard half-life and extended half-life FIX replacement therapies, respectively, and represent established treatment options indicated for adults and children with haemophilia B. These FIX replacement therapies can be administered as prophylaxis (to prevent bleeding) or ‘on-demand’ (to stop bleeding). This analysis aimed to estimate the cost-effectiveness of once-weekly prophylaxis with rFIXFc versus on-demand treatment with rFIX in patients with haemophilia B without inhibitors in the Italian healthcare setting. Methods A Markov model was developed to assess a hypothetical cohort of adolescent or adult male patients (≥ 12 years) with haemophilia B (FIX level of ≤ 2 IU/dL) without inhibitors. Model inputs were derived from the pivotal phase 3 clinical studies for rFIXFc and rFIX, published literature and assumptions when published data were unavailable. The model employed a lifelong time horizon with 6-monthly transitions between health states, and it estimated total costs, total quality-adjusted life years (QALYs), number of bleeds, number of surgeries and incremental cost-effectiveness ratio. Results rFIXFc prophylaxis was associated with lower total costs per patient (€5,308,625 versus €6,564,510) and greater total QALYs per patient (15.936 versus 11.943) compared with rFIX on-demand; rFIXFc prophylaxis was therefore the dominant treatment strategy. The model also demonstrated that rFIXFc prophylaxis was associated with fewer incremental bleeds (− 682.29) and surgeries (− 0.39) compared with rFIX on-demand. Conclusions rFIXFc prophylaxis provides improved health outcomes and lower costs, and represents a cost-effective treatment option compared with rFIX on-demand for adolescent and adult male patients with haemophilia B. This comparative assessment of cost-effectiveness should help to inform both clinicians and healthcare policy makers when making treatment decisions for patients with haemophilia B. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-024-02841-w.

show abstract

Evaluating the Effectiveness of Prophylactic Strategies for Hemophilia A Management: A Real-World, Longitudinal Observational Study

Chiou,

Lin,

Weng

et al. 2024

Drugs - Real World Outcomes

View full text Add to dashboard Cite

Background Hemophilia A (HA) treatment strategies aim to manage bleeding episodes and improve patients' quality of life. This study investigates the effectiveness of a preventative approach using intermediate-dose prophylaxis with standard half-life FVIII products in reducing bleeding rates and enhancing the quality of life for patients with severe HA. Methods A 4-year prospective longitudinal study followed 35 patients with severe HA (without FVIII inhibitors) who transitioned from a reactive treatment approach to intermediate-dose prophylaxis in Taiwan from 2014 until 2018. The study tracked annual bleeding rates (ABR) and annual joint bleeding rates (AjBR) alongside associated costs and patient-reported quality-of-life measures. Results Prophylaxis significantly reduced both ABR and AjBR compared with the previous treatment. After one year, ABR and AjBR decreased by 76.9% and 72.5%, respectively, with further reductions to 91.0% and 90.8% after 4 years ( p < 0.001). While the average annual cost of factor VIII concentrate increased by 41.0% in the first year, the incremental cost-effectiveness ratio demonstrated ongoing benefits from ABR avoidance over the 4 years. Additionally, patients reported significant improvements in quality-of-life measures following the switch to prophylaxis ( p = 0.036). Conclusion Intermediate-dose prophylaxis effectively reduced bleeding rates and improved quality of life in patients with severe HA. Despite initial cost increases, the intervention became cost effective over time. This study provides valuable data for healthcare policymakers, highlighting the long-term benefits of prophylaxis as a preventative approach for managing bleeding and improving overall well-being in patients with severe HA.

show abstract

A cost-effectiveness analysis of the prophylaxis versus on-demand regimens in severe hemophilia A patients under 12 years old in southern Iran

Cited by 6 publications

References 32 publications

Analysis of epidemiological and economic data of hemophilia A patients in Brazil

Analysis of epidemiological and economic data of hemophilia A patients in Brazil

Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors

Evaluating the Effectiveness of Prophylactic Strategies for Hemophilia A Management: A Real-World, Longitudinal Observational Study

Contact Info

Product

Resources

About