Cost-effectiveness model for on-demand treatment of hereditary angioedema (HAE) attacks. (3rd September 2019)

• Record Type: Journal Article
• Authors: Tyson, Christopher; Relan, Anurag; Adams, Phillippe; Haynes, Angela; Magar, Raf
• Is Part Of: Journal of drug assessment. Volume 8(2019)Supplement 1
• Page Start: 22
• Page End: 22
• Publication Date: 2019-09-03
• Subjects: C1 inhibitor -- cost -- hereditary angioedema -- recombinant -- ruconest; Phillippe Adams. Please contact [e-mail]; Drugs -- Testing -- Periodicals; 615.1901
• DOI: 10.1080/21556660.2019.1658300
• Languages: English
• ISSNs: 2155-6660

Abstract

Abstract: Background: Hereditary angioedema (HAE) is a rare C1-inhibitor (C1-INH) deficiency disease involving recurrent painful episodes of severe swelling that should be promptly treated. Aims: To determine cost and utility estimates for on-demand treatment of HAE attacks in order to better clarify and control expenses related to disease management. Methods: A decision-tree model included four comparators (ecallantide, icatibant, plasma-derived [pd] C1-INH, and recombinant human C1-INH [rhC1-INH]) and incorporated probabilities for self-administration vs healthcare provider administration, re-dosing, and hospitalization risk. Modeled costs comprised HAE therapies and healthcare system expenses. Effectiveness considered utility during attacks (0.51), no-attack baseline (0.83), and time to attack resolution. Overall drug cost and effectiveness per attack were used to estimate cost per quality-adjusted life year (QALY). Sensitivity analyses were performed to establish cost-effectiveness ranges. A budget impact model was developed for a health plan of 1 million (M) covered lives. Results: Costs and utility per attack were, respectively, $12, 342 and 0.804 for rhC1-INH, $14, 369 and 0.749 for icatibant, $13, 993 and 0.759 for pdC1-INH, and $20, 315 and 0.786 for ecallantide. At a mean annual attack rate of 26.9, cost per QALY was $402, 769 for rhC1-INH, $475, 942 for icatibant, $462, 275 for pdC1-INH, and $666, 153 for ecallantide. Re-dose rate was identified as a primary driver of cost-effectiveness variability. Estimated annual cost to the plan was $6.64 M for rhC1-INH, $7.73 M for icatibant, $7.53 M for pdC1-INH, and $10.93 M for ecallantide. A 5000-trial probabilistic sensitivity analysis (PSA) indicated that rhC1-INH was the most cost-effective in many scenarios, while ecallantide was the least cost-effective: mean costs (effectiveness) from PSA were $12, 390 (0.786) for rhC1-INH, $14, 132 (0.738) for icatibant, $13, 050 (0.746) for pdC-1INH, and $20, 286 (0.785) for ecallantide. Conclusions: This model demonstrated that rhC1-INH was the most cost-effective and ecallantide the least cost-effective on-demand HAE treatment and, overall, cost-effectiveness was substantially impacted by re-dosing rates. For icatibant, re-dosing rates of up to 44% to treat an HAE attack have been reported, and prescribing information allows up to three doses per 24-h period to treat a single attack. Driven by higher re-dosing rates, icatibant suffers from a higher per-attack drug cost and comparatively poor effectiveness.