BACKGROUND: Hereditary angioedema (HAE) is a rare genetic disorder characterized by attacks of tissue swelling that can require acute clinical management. Besides on-de-mand treatment for acute attacks, current therapy includes long-term prophylactic (LTP) medications, such as lanade-lumab and berotralstat, that reduce the risk of HAE attacks.
OBJECTIVE: To compare the expected costs per patient (pp) of 2 LTP therapies (lanadelumab and berotralstat) among patients with HAE in the United States.
METHODS: A Microsoft Excel–based model was constructed to examine the annual pp treatment costs for lanadelum-ab or berotralstat from a US third-party commercial payer perspective. Model inputs included LTP costs, efficacy in-puts (reduction of breakthrough attacks from an indirect treatment comparison), and direct medical costs (acute attack treatments, emergency department [ED] visits, hos-pitalizations, adverse events) in 2023 USD. The model also included the percentage of lanadelumab patients who were maintained on every-4-week vs 2-week dosing. Percent-ages from real-world evidence were used to test the impact on the model (19.0%, 27.8%, 46.3%). The patient population comprised adults aged 18 years and older with a confirmed HAE diagnosis and a history of at least 1 attack per 4-week period. LTP treatment costs were incurred continuously throughout the 12-month period. Acute treatment costs were only incurred when breakthrough attacks occurred; costs were dependent on attack severity. One-way and probabilistic sensitivity analyses were performed to vali-date the robustness of the model’s assumptions and specific parameter estimates.
RESULTS: Total annual HAE-related pp costs were lower for lanadelumab in all 3 down-titration percentages ($634,119; $611,417; $563,877) than for berotralstat ($757,137), result-ing in an estimated annual cost savings from $123,017 to $193,260 pp with lanadelumab. Total annual LTP cost sav-ings ranged from $42,641 to −$43,141 and acute attack cost savings ranged from $150,398 to $166,158 for lanadelumab compared with berotralstat. Cost savings were driven by lower acute attack drug acquisitions, drug administration, hospitalizations, and ED visits. Total costs/annum were most sensitive to lanadelumab and berotralstat package costs, lanadelumab dosage proportion, and the attack rate ratios used to determine the number of attacks/month. However, lanadelumab remained less costly than berotral-stat in 99.9% of 1,000 simulations performed.
CONCLUSIONS: This model found lanadelumab was cost saving compared with berotralstat for LTP treatment of adults with HAE.
