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

Abstract

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.

Emicizumab state‐of‐the‐art update

“…Multiple studies have shown that bleed prevention with emicizumab is more cost-effective than bypassing agents. 34,35 The introduction of Emicizumab to healthcare systems has the potential to generate additional savings by (1) delaying FVIII inhibitor development in previously untreated patients (PUPs) with the severe disease by up to 13 years, 36 (2) offering cost saving of $2 million per patient over 20 years by reducing immune tolerance induction and breakthrough bleeds, 36 (3) eliminating the need for surgical implantation of central venous access devices, especially in paediatrics and reducing severe bleeds requiring hospitalisation. The most considerable cost saving could be associated with maintaining joint health, but still, we do not know if this will be the case.…”

Budget Impact Analysis of the use of emicizumab for bleeding prophylaxis in patients with Hemophilia A with inhibitors in the Brazilian Health System

“…39 Positive results were also reported using national insurance claims and epidemiological data from Korea, as well as information obtained from the literature and from expert surveys with hematologists in that country. 40 In the study from Korea, emicizumab was found to be dominant over comparators, with predicted prevention of bleedings, QALY gains and reduced costs in base-case and sensitivity analyses. A BIA conducted considering the public healthcare system in Malaysia, concluded that the 5-year budget impact was reasonable and possibly cost-saving.…”

Is there any place for replacement therapy of hemophilia A in children in present and future?

“…Contact activation (intrinsic pathway) Несмотря на нежелательные (НЯ) или серьезные нежелательные явления, описанные в ходе клинических испытаний [33][34][35], в реальной клинической практике, НЯ, которые привели бы к прерыванию или прекращению лечения эмицизумабом у детей зарегистрировано не было [8]. Интересно, что применение эмицизумаба при ИГА показало не только клиническую, но и экономическую эффективность в различных странах, особенно среди тех пациентов, у которых отмечалось более 10 кровотечений в год [37][38][39][40][41][42].…”