Albutrepenonacog alfa (Idelvion®) for the treatment of Italian patients with hemophilia B: a budget impact model

Abstract

BACKGROUND: Enhanced pharmacokinetic profile of albutrepenonacog alfa allows to prolong the interdose period in prophylaxis, maintaining higher trough level, and to reduce dosage needed for bleeding. This improvement could lead to a better efficiency of the hemophilia B treatment.OBJECTIVES: To estimate the impact of this new drug on the Italian National Health System (NHS).METHODS: A model was developed from the NHS perspective to assess the budget impact of treating severe hemophilia B with reimbursed recombinant factor IX over 3 years in Italy. Target population was based on data from the National Registry of Congenital Coagulopathies, which collects data from 54 Hemophilia Treatment Centers. Treatment options were: albutrepenonacog alfa (Idelvion®), eftrenonacog alfa (Alprolix®) and nonacog alfa (BeneFIX®). Annual bleeding rate, dose and infusions needed to treat an episode based on clinical trials data were considered.RESULTS: Mean costs per patient were calculated for prophylaxis and bleeding treatment by age groups. Applying age-specific costs to the expected new pattern of drugs utilization, the impact on the NHS budget was € 6 million of savings cumulated in 3 years. The model results most sensitive to drug dosages. Lower drug consumption in prophylaxis and reduced bleeding rate than the alternatives reduce expenditures. Main limitations of this analysis were the assumptions that all severe patients receive prophylaxis and the lack of consideration of positive effects of hemorrhagic complications reduction (with consequent lower need of physiotherapy/prosthetic substitution).CONCLUSIONS: The introduction of Idelvion® as therapeutic option for hemophilia B is expected to decrease pharmaceutical costs and improve patient’s quality of life due to less frequent infusions.

Budget impact analysis of extended half-life recombinant factor IX (rFIXFc) in the treatment of haemophilia B

“…The overall decrease in costs in the 3-year period was estimated to be −€1,988,068 ( Fig. 2 ), considering the weekly prophylactic dosages reported in the respective SPC for rFIX and data from clinical studies and the literature to estimate the number of bleeding episodes and management ( 12 , 17 , 23 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 34 , 36 , 38 ).…”

“…b) Nonacog gamma: to estimate the amount of drug used in children <12 years of age, bleeding rates were taken from the EPAR (36), and rFIX dose was considered to be the same as administered in adults (12). c) Nonacog alfa: mean dose used for prophylaxis was applied to management of bleeding episodes, multiplied by the mean number of infusions needed to treat a bleeding episode (34).…”

“…Bleeding rates and the amount of rFIX used for each product were based on literature data ( 12 , 34 , 35 ). According to clinical practice and guidelines, bleeding episodes are treated with an additional dose of FIX products ( 1 ).…”

“…Nonacog alfa: mean dose used for prophylaxis was applied to management of bleeding episodes, multiplied by the mean number of infusions needed to treat a bleeding episode ( 34 ).…”

Expert opinion paper on the treatment of hemophilia B with albutrepenonacog alfa

“…Adult patients who require a high number of infusions or those poorly controlled with the former treatment can also especially benefit from rIX-FP, since the reduction in the number of injections can entail an even larger increase in quality of life, and it could be a cost-effective option. Actually, a recent pharmacoeconomic model assessed the budget impact of treating severe hemophilia B with reimbursed rIX-FP over 3 years in Italy, concluding that rIX-FP is expected to decrease pharmaceutical costs [50]. A recent real world study found that the cost per success (defined as patients experiencing no spontaneous bleeding) in patients switching from nonacog alfa to rIX-FP was reduced by 29% in Italy, 55% in Belgium, and 50% in the United Kingdom.…”