Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors

Cortesi, P.A.; Castaman, Giancarlo; Trifirò, Gianluca; Creazzola, Simona Serao; Improta, Giovanni; Mazzaglia, Giampiero; Molinari, Angelo Claudio; Mantovani, Lorenzo Giovanni

doi:10.1055/s-0039-3401822

Cited by 53 publications

(41 citation statements)

References 50 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…26 Notably, emicizumab prophylaxis during ITI was well tolerated; 27 nonetheless, co-administration of FVIII and emicizumab may pose a substantial financial burden on healthcare systems. 28 Prospective studies will be necessary to compare treatment outcomes of a combined ITI and emicizumab regimen with a standard ITI or emicizumab prophylaxis-only regimen and to investigate whether emicizumab prophylaxis modifies the immunologic response to FVIII. 27 Breakthrough bleeds occurred in 50% of the patients in our group, which is slightly higher than rates reported in previous studies.…”

Section: Discussionmentioning

confidence: 99%

Emicizumab treatment and monitoring in a paediatric cohort: real‐world data

et al. 2020

View full text Add to dashboard Cite

Real-world data on emicizumab use and monitoring in paediatric severe haemophilia A (HA) patients are scarce. We therefore sought to evaluate safety, efficacy, and laboratory monitoring of emicizumab prophylaxis in a cohort of 40 children with severe HA, including 22 non-inhibitor patients and nine infants younger than one year. Bleeding, trauma, adverse events, and surgeries were documented during a median follow-up of 45 weeks. Emicizumab levels, activated partial thromboplastin time (aPTT) values, and thrombin generation were measured before and during therapy. Twenty patients experienced zero bleeds. All bleeding was trauma-related, and bleeding risk was positively correlated with the length of emicizumab prophylaxis. Sixteen surgical interventions were performed in 12 patients, with no thrombotic complications or thrombotic microangiopathy. Prolonged aPTT values normalised after emicizumab initiation, correlating with an increase in emicizumab plasma levels. Elevation in the thrombin generation was observed following emicizumab prophylaxis, with lower values recorded in younger infants. Emicizumab prophylaxis was safe and well tolerated. As all bleedings were trauma-related, laboratory monitoring could not predict bleeding risk. Our results do not support routine thrombin generation monitoring in children treated by emicizumab, yet further studies are warranted in the context of surgical procedures.

show abstract

Section: Discussionmentioning

confidence: 99%

Emicizumab treatment and monitoring in a paediatric cohort: real‐world data

et al. 2020

View full text Add to dashboard Cite

show abstract

“…105 A recent cost-effectiveness analysis, comprising a Markov model and a budget impact model, further substantiates the findings of previous studies. 106…”

Section: Economic Considerationsmentioning

confidence: 99%

Bridging the Missing Link with Emicizumab: A Bispecific Antibody for Treatment of Hemophilia A

et al. 2020

View full text Add to dashboard Cite

Hemophilia A, characterized by absent or ineffective coagulation factor VIII (FVIII), is a serious bleeding disorder that entails severe and potentially life-threatening bleeding events. Current standard therapy still involves replacement of FVIII, but is often complicated by the occurrence of neutralizing alloantibodies (inhibitors). Management of patients with inhibitors is challenging and necessitates immune tolerance induction for inhibitor eradication and the use of bypassing agents (activated prothrombin complex concentrates or recombinant activated factor VII), which are expensive and not always effective. Emicizumab is the first humanized bispecific monoclonal therapeutic antibody designed to replace the hemostatic function of activated FVIII by bridging activated factor IX and factor X (FX) to activate FX and allow the coagulation cascade to continue. In the majority of hemophilic patients with and without inhibitors, emicizumab reduced the annualized bleeding rate to almost zero in several clinical trials and demonstrated a good safety profile. However, the concurrent use of emicizumab and activated prothrombin complex concentrate imposes a high risk of thrombotic microangiopathy and thromboembolic events on patients and should be avoided. Yet, the management of breakthrough bleeds and surgery remains challenging with only limited evidence-based recommendations being available. This review summarizes published clinical trials and preliminary reports of emicizumab and discusses the clinical implications of emicizumab in treatment of hemophilia A.

show abstract

“…Several studies have reported cost‐effectiveness of emicizumab prophylaxis, and emicizumab was considered a cost‐saving option in all previous studies; however, these previous studies differed regarding methods and aspects such as choice of comparator, demographic characteristics of the study cohorts and time horizon. Cortesi et al assessed cost‐effectiveness of emicizumab prophylaxis in HAPI over throughout their lifetime from the Italian perspective; however, in contrast to our study, they compared emicizumab prophylaxis with BPA prophylaxis and the starting age of their cohort was 4 years, which may explain differences in results 35 . Patel et al assessed cost‐effectiveness of emicizumab prophylaxis compared with factor VIII prophylaxis in severe haemophilia A patients without inhibitors by estimating the economic impact over 20 years, beginning at age 1 36 .…”

Section: Discussionmentioning

confidence: 59%

Cost‐utility analysis of emicizumab prophylaxis in haemophilia A patients with factor VIII inhibitors in Korea

et al. 2020

View full text Add to dashboard Cite

Aims Haemophilia A patients with factor VIII inhibitors (HAPI) experience frequent spontaneous bleeding, approximately once a week, and require expensive bypassing agent (BPA) treatments to control bleeding over their lifetime. According to the HAVEN 1 trial, weekly emicizumab (Hemlibra®) prophylaxis injection reduces annualized bleeding rates (ABR) by 87% compared with BPA on‐demand treatment (BPA‐OD) administered at the time of bleeding. Our study aimed to assess the cost‐effectiveness of emicizumab prophylaxis in HAPI in Korea. Methods Using a lifetime Markov model with health states of ‘alive with bleeds’ and ‘dead’, we simulated the experience of HAPI receiving emicizumab prophylaxis (treatment arm) or BPA‐OD (control arm) and estimated expected clinical and economic outcomes under each treatment arm. Model parameters included comparative effectiveness, clinical and epidemiologic characteristics of Korean HAPI, costs of drug treatment and medical events and utility for ‘alive with bleeds’ state under each treatment. We utilized local data, including National Health Insurance claims data, national statistics, literature and expert surveys with haematologists. Results Base‐case analysis results showed that compared with BPA‐OD, lifetime emicizumab prophylaxis prevented 807 bleedings, extended 3.04 quality‐adjusted life‐years and reduced costs by 2.6 million US dollars. Thus, emicizumab prophylaxis is a dominant treatment option with better effectiveness and lower costs than BPA‐OD. A series of one‐way sensitivity analyses consistently showed dominant results, confirming that lifetime emicizumab prophylaxis is a cost‐saving intervention for HAPI. Conclusion Emicizumab prophylaxis is an excellent treatment choice reducing ABR, improving quality of life and reducing costs.

show abstract

Cost-Effectiveness and Budget Impact of Emicizumab Prophylaxis in Haemophilia A Patients with Inhibitors

Cited by 53 publications

References 50 publications

Emicizumab treatment and monitoring in a paediatric cohort: real‐world data

Emicizumab treatment and monitoring in a paediatric cohort: real‐world data

Bridging the Missing Link with Emicizumab: A Bispecific Antibody for Treatment of Hemophilia A

Cost‐utility analysis of emicizumab prophylaxis in haemophilia A patients with factor VIII inhibitors in Korea

Contact Info

Product

Resources

About