Cost-Utility Analysis of Eculizumab for the Treatment of Paroxysmal Nocturnal Hemoglobinuria From the Perspective of the Brazilian Public Health System.

Objective: To perform a first cost-utility analysis of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria from the perspective of the Brazilian Unified Health System.

Methods: A Markov decision model was developed for 35-year-old patients with symptomatic paroxysmal nocturnal hemoglobinuria. We used a cycle length of one month and a time horizon of 20 years. The effectiveness measure was the quality-adjusted life year (QALY). Data were extracted from clinical trials, historical cohorts, and Unified Health System databases. Resource use and costs were estimated from the perspective of the Unified Health System. Deterministic and probabilistic sensitivity analyses were performed.

Results: The estimated gain in effectiveness with the use of eculizumab was 1.08 QALY through the incremental cost of R$10,959,375.95. The incremental cost-effectiveness ratio was R$10,139,542.84 per QALY, being 331.92 times greater than the Brazilian gross domestic product per capita. In the deterministic sensitivity analysis, the parameters related to the utilities of health states were associated with greater impact in the model. The results of the probabilistic sensitivity analysis with 1000 simulations evidence that 100% of the simulations were not considered cost-effective with the arbitrated willingness to pay R$30,548.40 and R$91,645.20 per QALY.

Conclusions: The gain in effectiveness with the use of eculizumab was modest, associated with an unjustifiable incremental cost. Therefore, eculizumab is not a cost-effective drug compared with the current standard of care in the treatment of paroxysmal nocturnal hemoglobinuria from the Brazilian Unified Health System perspective.