COST-EFFECTIVENESS OF RECOMBINANT HUMAN C1 ESTERASE INHIBITOR (RHC1-INH) VS SEBETRALSTAT FOR THE TREATMENT OF SEVERE OR VERY SEVERE HEREDITARY ANGIOEDEMA (HAE) ATTACKS
Author(s)
Raffi Tachdjian, MD, MPH1, Nihal Narsipur, PharmD, MPH2, Yang Meng, PhD3, Spencer Notinger, MSc3, Sarah Brand-Wiita, MSc3, Jonathan A. Bernstein, MD4, Amanda Harrington, PhD2;
1Division of Allergy and Clinical Immunology, David Geffen School of Medicine, University of California, Los Angeles, CA, USA, 2Pharming Healthcare, Inc., Warren, NJ, USA, 3Lumanity, Morristown, NJ, USA, 4Advanced Allergy Services, LLC, Cincinnati, OH, USA
1Division of Allergy and Clinical Immunology, David Geffen School of Medicine, University of California, Los Angeles, CA, USA, 2Pharming Healthcare, Inc., Warren, NJ, USA, 3Lumanity, Morristown, NJ, USA, 4Advanced Allergy Services, LLC, Cincinnati, OH, USA
OBJECTIVES: HAE is a rare disease characterized by recurrent and sometimes severe swelling attacks that require on-demand treatment. Previous studies have established the cost-effectiveness of rhC1-INH compared with other on-demand HAE therapies. This study analyzed the cost-effectiveness of rhC1-INH vs sebetralstat, a recently approved on-demand therapy, in the treatment of severe or very severe HAE attacks.
METHODS: A decision-tree model comparing rhC1-INH and sebetralstat was constructed from the US commercial payer perspective, focusing on one HAE attack over a 72-hour period. Key efficacy inputs—redosing rates and time from treatment administration to full resolution in severe or very severe attacks—were informed by an indirect treatment comparison using phase 2/3 and phase 3 rhC1-INH trials (NCT01188564; NCT00225147; NCT00262301 [placebo group only]) and the sebetralstat KONFIDENT trial (NCT05259917). Severity-specific health utilities were used to derive per-attack quality-adjusted life-hours (QALH). Costs and other inputs were based on available literature or reasonable assumptions. Absolute and incremental duration and cost of a single attack were calculated. These estimates were then used to derive the incremental net monetary benefit (INMB) per attack (willingness-to-pay threshold: $150,000) for rhC1-INH over sebetralstat.
RESULTS: Model results showed that the time to complete resolution of severe and very severe attacks was 6.9 hours for rhC1-INH and 18.2 hours for sebetralstat. The cost per attack was $18,408 for rhC1-INH and $25,768 for sebetralstat. rhC1-INH was cost-effectively dominant, providing 11.3 additional attack-free hours, resolving 29% more attacks with 1 dose, gaining 7.4 QALHs, and yielding $7360 in per-attack cost savings and $7487 in INMB compared with sebetralstat. Probabilistic sensitivity analysis results were similar to the deterministic results.
CONCLUSIONS: For severe or very severe HAE attacks, rhC1-INH treatment offers more attack-free time, cost savings, and QALH gains driven by faster time to complete resolution and reduced need for redosing compared with sebetralstat.
METHODS: A decision-tree model comparing rhC1-INH and sebetralstat was constructed from the US commercial payer perspective, focusing on one HAE attack over a 72-hour period. Key efficacy inputs—redosing rates and time from treatment administration to full resolution in severe or very severe attacks—were informed by an indirect treatment comparison using phase 2/3 and phase 3 rhC1-INH trials (NCT01188564; NCT00225147; NCT00262301 [placebo group only]) and the sebetralstat KONFIDENT trial (NCT05259917). Severity-specific health utilities were used to derive per-attack quality-adjusted life-hours (QALH). Costs and other inputs were based on available literature or reasonable assumptions. Absolute and incremental duration and cost of a single attack were calculated. These estimates were then used to derive the incremental net monetary benefit (INMB) per attack (willingness-to-pay threshold: $150,000) for rhC1-INH over sebetralstat.
RESULTS: Model results showed that the time to complete resolution of severe and very severe attacks was 6.9 hours for rhC1-INH and 18.2 hours for sebetralstat. The cost per attack was $18,408 for rhC1-INH and $25,768 for sebetralstat. rhC1-INH was cost-effectively dominant, providing 11.3 additional attack-free hours, resolving 29% more attacks with 1 dose, gaining 7.4 QALHs, and yielding $7360 in per-attack cost savings and $7487 in INMB compared with sebetralstat. Probabilistic sensitivity analysis results were similar to the deterministic results.
CONCLUSIONS: For severe or very severe HAE attacks, rhC1-INH treatment offers more attack-free time, cost savings, and QALH gains driven by faster time to complete resolution and reduced need for redosing compared with sebetralstat.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE169
Topic
Economic Evaluation
Disease
SDC: Rare & Orphan Diseases