OBJECTIVES: Chronic lung infection with Pseudomonas aeruginosa occurs in approximately 50% of patients with cystic fibrosis (CF) which compromises lung function, and significantly contributes to the increased healthcare costs. Inhaled tobramycin, used to manage P. aeruginosa infection in CF patients. However, guidelines suggest dornase alfa as the only mucolytic agent that showed improvements in lung function and a reduction in infection in patients with CF. Hence, this analysis aimed to evaluate the cost-effectiveness of Inhaled tobramycin versus nebulized dornase alfa for the management of patients with CF from the perspective of the Iranian National Health Service (NHS).

METHODS: A patient-level simulation model was developed over a 10-year time horizon. A Markov structure was used to consider transitions between health states, defined principally by levels of percent predicted of FEV1. Model parameters were informed by patient-level data from randomized controlled trials together with the best available evidence from the literature. Resource use and costs associated with drug acquisition and the management of exacerbations were drawn from reference sources and expert opinion. Both costs and benefits were discounted annually at 5%. All costs were presented in 2018 US dollars. Extensive deterministic and probabilistic sensitivity analyses were performed.

RESULTS: Dornase alfa is expected to produce higher quality-adjusted life-years (QALYs) than inhaled tobramycin (5.618 vs. 5.388). In addition, dornase alfa is expected to be associated with marginally higher cost than tobramycin ($118,617 vs. $118,002). in consequence, the ICER for dornase alfa versus tobramycin was found to be approximately $2,673 per QALY gained. Sensitivity analyses indicated that results were sensitive to drug acquisition cost. Probabilistic sensitivity analyses showed dornase alfa to be cost-effective treatment in 78.5% of 1000 simulations at the threshold of %5,400 per QALY.

CONCLUSIONS: dornase alfa can be considered a cost-effective bronchodilator compared with inhaled tubramycin in the management of patients with CF for the NHS.