Cost-Minimisation Analysis of Recombinant Factor VIII FC Fusion Protein Versus Emicizumab for Haemophilia a Prophylaxis in Europe
Author(s)
Hakimi Z1, Nazir J1, Pochopień M2, Aballéa S3, Skrzeczek A2
1Sobi AB, Stockholm, Sweden, 2Creativ-Ceutical, Krakow, Poland, 3Creativ-Ceutical, Rotterdam, Netherlands
OBJECTIVES : Indirect treatment comparisons have shown that recombinant factor VIII-Fc fusion protein (rFVIIIFc) is at least as effective as emicizumab for haemophilia A (HA) prophylaxis. This cost‑minimisation model evaluates rFVIIIFc versus emicizumab for HA prophylaxis over 5 years in 100 people, from a healthcare payer perspective, in the UK, France, Germany, Italy and Spain. METHODS : Doses used in the model were based on the A-LONG study for rFVIIIFc and the summary of product characteristics for emicizumab. Bodyweight was based on data from the A‑LONG (adults/adolescents, >12 years) and Kids A-LONG (children, ≤12 years) studies. Other model inputs included costs associated with: drug acquisition; emicizumab wastage according to bodyweight (manufacturer’s recommendations); increased emicizumab dose in hypo‑responders; wasted on‑demand factor VIII (FVIII) due to expired shelf life; and additional FVIII for treatment of bleeds for both groups. RESULTS : In the UK, total 5-year costs for adolescents/adults were €150.0 million for rFVIIIFc and €272.9 million for emicizumab, a total incremental saving with rFVIIIFc of €122.9 million. In children, total 5‑year costs were €51.0 million and €115.8 million, respectively, a total incremental saving with rFVIIIFc of €64.8 million. Most of the excess cost with emicizumab reflects its higher acquisition cost and emicizumab wastage; the latter can be substantial, corresponding to up to 13% of used drug in adults/adolescents and >50% in children. At maximal wastage (bodyweight set at 81 kg and 41 kg for adult/adolescents and children, respectively), cost of wasted drug increased by 87% in adolescents/adults and 101% in children compared with the base‑case analysis. In other European countries, cost savings associated with rFVIIIFc use ranged from €119.1–159.2 million in adolescents/adults and €63.8–76.1 million in children. CONCLUSIONS : The cost of HA prophylaxis in Europe would be substantially higher if patients were treated with emicizumab compared with rFVIIIFc.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PSY7
Topic
Clinical Outcomes, Economic Evaluation
Topic Subcategory
Comparative Effectiveness or Efficacy, Cost-comparison, Effectiveness, Utility, Benefit Analysis
Disease
Drugs