Cost‐effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States

Potnis, Kunal C; Viswanathan, Girish; Bona, Robert; Ito, Satoko; Kempton, Christine L.; Pandya, Ankur; Krumholz, Harlan M.; Goshua, George

doi:10.1002/ajh.27014

American J Hematol

2023

DOI: 10.1002/ajh.27014

|View full text |Cite

Cost‐effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States

Kunal C Potnis

Girish Viswanathan

Robert Bona

et al.

Abstract: Prophylactic emicizumab is cost‐ineffective in adults with moderate or mild hemophilia A without inhibitors at current pricing. The price of prophylactic emicizumab would need to decrease by >35% to become cost‐effective in this patient population.

Help me understand this report

Search citation statements

Order By: Relevance

Paper Sections

Select...

Citation Types

Supporting

Mentioning

Contrasting

Year Published

2024

Publication Types

Select...

Article2

Relationship

Self Cite0

Independent2

Authors

Journals

Cited by 2 publications

References 8 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

Evaluating the benefits of emicizumab prophylaxis for haemophilia A with inhibitors: A cost‐effectiveness and budget impact analysis in Thailand's upper‐middle income setting

Kengkla,

Wilairat,

Natesirinilkul

et al. 2024

Haemophilia

View full text Add to dashboard Cite

BackgroundIn Thailand, an upper‐middle‐income country, managing haemophilia A (HA) with inhibitors poses significant challenges, often necessitating bypassing agents (BPAs) for bleeding control. This study evaluates the cost‐effectiveness and budget impact of emicizumab, a novel prophylactic agent, as an alternative to both episodic and prophylactic BPA treatments from a societal perspective.MethodsA Markov model was employed to estimate the lifetime societal costs and outcomes of emicizumab prophylaxis for HA patients with inhibitors. Treatment efficacy, cost, and epidemiological data were obtained through a comprehensive literature review and incorporated into the model. A 5‐year budget impact analysis complemented the cost‐utility analysis, with a 3% annual discount rate applied to future costs and outcomes.ResultsIn the base‐case scenario, emicizumab prophylaxis in HA patients aged 2 years and above demonstrated superior cost‐effectiveness, yielding 18.1 quality‐adjusted life years (QALYs) per patient over a lifetime and resulting in cost savings of 138 million Thai Baht (THB) compared to BPA prophylaxis. Compared to episodic BPA treatment, emicizumab yielded 30.5 QALYs and saved 25 million THB per patient. The 5‐year budget impact was projected at 1775 million THB.ConclusionsEmicizumab offers a cost‐saving approach for HA treatment with inhibitors in Thailand, promising significant health benefits and budgetary savings. This supports its potential inclusion in Thailand's National List of Essential Medicines to enhance haemophilia care access.Highlights Managing haemophilia A (HA) with inhibitors in Thailand, an upper‐middle‐income country, faces challenges due to limited access to effective treatments or newer drugs for bleeding management. Emicizumab prophylaxis found to as a cost‐effective and viable alternative to traditional treatments, effectively preventing bleeding in Thai HA patients over 2 years old with inhibitors. Demonstrating improved clinical outcomes and reduced costs, emicizumab prophylaxis outperforms episodic BPA treatments, positioning it as a superior treatment option for HA patients with inhibitors in Thailand.

show abstract

Evaluating the benefits of emicizumab prophylaxis for haemophilia A with inhibitors: A cost‐effectiveness and budget impact analysis in Thailand's upper‐middle income setting

Kengkla,

Wilairat,

Natesirinilkul

et al. 2024

Haemophilia

View full text Add to dashboard Cite

show abstract

Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials

Grabowska,

Grzelak,

Zhao

et al. 2024

CPPS

View full text Add to dashboard Cite

Hemophilia is a plasma bleeding disorder characterized by a deficiency of certain blood clotting factors. The most common forms of this disease, i.e., type A and type B, affect approximately 400,000 people worldwide. Without appropriate treatment ensuring the proper coagulation cascade, this disease may lead to serious disability. Minimizing patient discomfort is possible via replacement therapy, consisting of the substitution of a missing coagulation factor via intravenous administration. Frequent medication and the risk related to factor inhibitors are significant disadvantages, necessitating the improvement of current therapies or the development of novel ones. This review examines the humanized bispecific antibody Emicizumab which ensures hemostasis by mimicking the action of the coagulation factor VIII, a deficiency of which causes type A hemophilia. The paper outlines the topic and then summarizes available clinical trials on Emicizumab in type A hemophilia. Several interventional clinical trials have found Emicizumab to be effective in decreasing bleeding episodes and raising patient satisfaction among various hemophilia A populations. Current Emicizumab-related trials are forecast to be completed between 2024 and 2030, and in addition to congenital hemophilia A, the trials cover acquired hemophilia A and patients playing sports. Providing a more comprehensive understanding of Emicizumab may revolutionize the management of hemophilia type A and improve quality of life. Conclusively, Emicizumab is a gentler therapy owing to subcutaneous delivery and fewer injections, which reduces injection-site reactions and makes therapy less burdensome, ultimately decreasing hospital visits and indirect costs.

show abstract

scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.

Contact Info

customersupport@researchsolutions.com

10624 S. Eastern Ave., Ste. A-614

Henderson, NV 89052, USA

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

Blog Terms and Conditions API Terms Privacy Policy Contact Cookie Preferences Do Not Sell or Share My Personal Information

Made with 💙 for researchers

Part of the Research Solutions Family.

Cost‐effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States

Abstract: Prophylactic emicizumab is cost‐ineffective in adults with moderate or mild hemophilia A without inhibitors at current pricing. The price of prophylactic emicizumab would need to decrease by >35% to become cost‐effective in this patient population.

Cited by 2 publications

References 8 publications

Evaluating the benefits of emicizumab prophylaxis for haemophilia A with inhibitors: A cost‐effectiveness and budget impact analysis in Thailand's upper‐middle income setting

Evaluating the benefits of emicizumab prophylaxis for haemophilia A with inhibitors: A cost‐effectiveness and budget impact analysis in Thailand's upper‐middle income setting

Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials

Contact Info

Product

Resources

About