EQOFIX: a combined economic and quality‐of‐life study of hemophilia B treatments in France

Abstract

BACKGROUND EQOFIX is a medicoeconomic study that analyzed the health‐related quality of life (HRQoL) and costs of care of the moderate and severe forms of hemophilia B, treated on demand or by prophylaxis with either plasma‐derived Factor IX (pdFIX) or recombinant FIX (rFIX). STUDY DESIGN AND METHODS The primary objectives were evaluations of the impact of hemophilia B on HRQoL and of the costs associated with its management. The secondary objectives were evaluations of the clinical efficacy and costs of care of pdFIX and rFIX. In this observational study we included and followed for 1 year severe and moderate hemophilia B patients without inhibitor. HRQoL was evaluated through generic and disease‐specific questionnaires. Information on the health resources consumed was collected every 3 months. RESULTS The EQOFIX cohort was composed of 155 patients, including 51 children and 104 adults, with 114 having severe disease and 41 having moderate disease. The regimens were prophylactic for 61 and on demand for 94. Altogether, 78 were treated with rFIX and 77 with pdFIX. There was no difference in the QoL between the pdFIX and rFIX treatments. The extra cost of prophylaxis was €22,605 per bleeding event prevented. The consumption of FIX was 1.4‐fold higher for the patients treated with rFIX than for the patients treated with pdFIX. CONCLUSION Our findings in a cohort composed of 25% of the French population of moderate and severe hemophilia B patients show, with similar clinical and HRQoL results, that treatment with rFIX is more expensive than treatment with pdFIX.

Cost‐effectiveness of emicizumab vs bypassing agents in the prevention of bleeding episodes in haemophilia A patients with anti‐FVIII inhibitors in France

“…However, given the high cost of treatment, these savings have a limited impact on the total cost. In recent studies, the share of treatment costs in France represented 90% of the total cost of the disease 10,42 …”

“…In recent studies, the share of treatment costs in France represented 90% of the total cost of the disease. 10,42 Treatment costs (Table 4). The administered amounts of BPA to treat a bleed and the proportion of bleeds treated with a combination of aPCC and rFVIIa were lower in the emicizumab arm than in the on-demand treatment arm.…”

“…People with haemophilia A (PwHA) bruise easily have persistent bleeding after trauma or surgery, and spontaneous bleeding into joints, muscles or soft tissues 2,3 . This leads to poor quality of life and affects emotional, social and physical components of patients’ well‐being and function 4‐10 . Adequate treatment is compulsory to hinder long‐term secondary complications, such as musculoskeletal damages and psychosocial consequences 2,3 …”

A national French noninterventional study to assess the long‐term safety and efficacy of reformulated nonacog alfa

“…It should be noted that these kind of data were also observed in a recent cost‐effectiveness study EQOFIX, also conducted on patients from centers included in the Réseau FranceCoag. We can suppose that similar trends in the mean doses per injection of F IX between the two regimens are likely related to the standards of care applied in these centers . Otherwise, only 31% of patients were on once‐weekly prophylaxis with not a higher dosage than in the twice‐weekly regimen.…”

Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs

“…Unfortunately, intravenous factor replacement is cumbersome, demanding on peripheral veins, and is very costly. Importantly, factor replacement therapy does not always prevent spontaneous bleeds and may induce immune reactions to the replaced FIX8, 10, 11 and does not completely prevent arthropathy, which may even occur in patients that have never reported a joint bleed 12, 13…”