When it comes to rare blood diseases, the introduction of Emicizumab, a bispecific monoclonal antibody, has been a significant milestone in the treatment of haemophilia A. The drug, produced by Roche, mimics activated factor VIII (FVIII) and is administered via subcutaneous injection, offering a more convenient alternative to intravenous treatment options mostly used. However, the cost-effectiveness of Emicizumab in non-severe haemophilia A patients remains uncertain. This article aims to shed light on this issue.
The HAVEN 6 trial demonstrated that patients with non-severe haemophilia A on prophylactic Emicizumab experienced a significant decrease in treated bleeding monthly probability. However, Emicizumab’s high cost, roughly $47,000 per month in the United States, raises questions about its cost-effectiveness.
To evaluate the cost-effectiveness of Emicizumab compared to standard recombinant FVIII, a recent study developed a Markov model. The model evaluated the quality-adjusted life expectancy and costs for these patients, using a lifetime time horizon with a 1-month Markov cycle. The annual discount rate for cost and health outcomes is 3%, and the perspective is from the United States payer. The willingness-to-pay level (WTP) is set at $150,000 per quality-adjusted life year (QALY).
The analysis revealed that Emicizumab prophylaxis resulted in 62% fewer treated bleed events over patients’ lifetimes than recombinant FVIII prophylaxis. However, the cost of Emicizumab prophylaxis accrued to $17,400,000, resulting in an incremental cost-effectiveness ratio (ICER) of 14.5 million USD/QALY, making it cost-ineffective compared to standard of care. The two secondary outcome threshold analyses showed that (1) a 100% reduction in chronic arthropathy prevalence (i.e., 0% chronic arthropathy) decreased the ICER to $1 160 000/QALY and (2) 1 mg of emicizumab must decrease by 35% to $64.60 to be cost-effective at $150 000/QALY.
Studies conducted in countries like Canada and reports from the Institute for Clinical and Economic Review suggest that Emicizumab prophylaxis can be cost-saving for patients with severe haemophilia A. However, this cost-saving benefit does not extend to patients with moderate or mild haemophilia A due to the lower prevalence of bleeding events and other related complications in this population. This discrepancy highlights the need for a more nuanced approach to pricing that takes into account the severity of the disease.
These findings underscore the need for careful consideration of indication-based pricing of novel therapies like Emicizumab. With the goal of value-based healthcare transformation in mind in the United States, indication-based pricing could improve patient outcomes at costs commensurate with clinical improvement.
In conclusion, the introduction of Emicizumab has undoubtedly shaken up the treatment landscape for haemophilia A, offering a more convenient and effective treatment option. However, the high cost of this novel therapy raises significant concerns about its cost-effectiveness, particularly for patients with non-severe haemophilia A. While Emicizumab prophylaxis has demonstrated significant clinical benefits in reducing bleeding events, its cost-effectiveness compared to standard recombinant FVIII remains unfavourable.
Furthermore, the discrepancy in cost-effectiveness across different countries and patient populations underscores the need for a more nuanced approach to drug pricing, which considers the severity of the disease and the specific patient population.
As the healthcare landscape continues to evolve towards a value-based model, it is crucial for stakeholders to balance the need for therapeutic innovation with cost-effectiveness. We must maintain a balance that makes new treatments like Emicizumab both affordable and accessible. This approach shouldn’t compromise the sustainability of healthcare systems. Future research should explore strategies to maximise the cost-effectiveness of these new therapies. One possible strategy is indication-based pricing. This way, we can ensure that we financially sustain the benefits of therapeutic innovation.
🚀 Discover how the AI-driven LabelComp tool is transforming drug safety surveillance! By automating the identification of adverse events in drug labelling, LabelComp enhances accuracy and efficiency, supporting regulatory decision-making and public health. 🌐💊
#SyenzaNews #AIinHealthcare #DrugSafety #PharmaInnovation #RegulatoryScience
🌟 School-based health centres (SBHCs) are improving healthcare for underserved youth across the US! These centres provide vital services, from preventive care to chronic disease management, right where students need them most – in schools. 📚🏥
SBHCs improve academic performance, reduce absenteeism, and enhance overall student well-being. Let’s support these essential centres and ensure every child has access to quality healthcare. 🌟
#SyenzaNews #SBHC #ChronicDiseaseManagement #HealthEquity #PreventiveCare
🌟 Exciting developments in Abu Dhabi! The Department of Health has introduced new ABA guidelines for Autism Spectrum Disorder, aiming to improve care for People of Determination. This initiative focuses on standardising care, enhancing accessibility, and fostering collaboration between healthcare and education professionals.
Learn more about how these guidelines can make a difference in the lives of individuals with ASD.
#SyenzaNews #HealthcareInnovation #AutismCare #InclusiveHealth #ABAGuidelines #AbuDhabiHealth
CORRESPONDENCE ADDRESS
WRITE US