Presentation (CTI)

OBJECTIVES: Hereditary angioedema (HAE) is an ultra-rare, autosomal dominant genetic disorder characterized by painful, disfiguring, and debilitating attacks, with potential fatality. It significantly impacts patients’ quality of life, often associated with anxiety and depression. Long-term prophylaxis with lanadelumab offers a new therapeutic option, more effective compared to C1 esterase inhibitors-IV (C1-INH IV).To evaluate the cost-effectiveness of Lanadelumab in the prophylaxis of recurrent HAE attacks in patients aged 12 years and older from the perspective of Brazilian supplementary health
METHODS: The study used a Markov model to compare prophylaxis with Lanadelumab to existing treatments, C1-INH IV, considering an appropriate time horizon based on life expectancy and median age of HAE diagnosis, and 5% discount rate for costs and outcomes. The costs of drug acquisition, administration, and adverse events were analyzed, along with effectiveness measured in quality-adjusted life years (QALYs) and the number of attacks prevented. Univariate and probabilistic analyses were used to estimate the most influential variables of the model and characterize the uncertainty around the results
RESULTS: The cost-effectiveness analysis showed that Lanadelumab was dominant compared to C1-INH IV, providing a gain of 2.72 QALYs and preventing 380 attacks. The total cost of Lanadelumab was R$ 30,395,126.36 million, while C1-INH IV was R$ 59,067,244.92, resulting in a saving of R$ 28,672,118.55. The variables that had the greatest impact on the results were: the cost of prophylactic treatment with C1-INH IV, the average duration of attacks, and the cost of prophylactic treatment with Lanadelumab
CONCLUSIONS: Lanadelumab proved to be dominant, providing a more effective and cost-saving option for the prophylaxis of HAE attacks compared to C1-INH IV