Abstract

Objectives

to evaluate the cost-utility of ataluren plus standard of care (SoC) for treating patients with nonsense mutation Duchenne muscular dystrophy from the Brazilian public health system perspective.

Methods

A semi-Markov model, comprising 5 states, was developed to estimate the incremental cost per quality-adjusted life-years (QALYs) of ataluren plus SoC compared with SoC alone. A 48-year time horizon was used in the base-case analysis, considering annual cycles. Costs were expressed as international dollars (Int$) applying the purchasing power parity conversion rate (Brazilian purchasing power parity of 2.44 BRL/Int$ in 2023). The transition probabilities and utility data were obtained from previous studies, and costs were based on data from a microcosting study and the Brazilian Drug Market Regulation Chamber. Both costs and QALYs were discounted using a rate of 5%. Parameter uncertainty was assessed through deterministic and probabilistic sensitivity analyses.

Results

Ataluren plus SoC leads to incremental 0.503 QALYs and incremental costs of Int$13 298 877.45, resulting in an incremental cost-utility ratio of Int$26 415 910.08/QALY. According to the deterministic sensitivity analysis, utilities related to the state’s health progression of the model were the parameter that most significantly influenced the incremental cost-utility ratio. In the probabilistic sensitivity analysis, all estimates were above the current willingness-to-pay threshold of Int$49 180.33 per QALY for rare diseases.

Conclusions

Ataluren plus SoC for treating nonsense mutation Duchenne muscular dystrophy patients was not a cost-effective intervention compared with SoC alone from the perspective of the Brazilian public health system.