The present CEA studies the cost-effectiveness of fluticasone vs. montelukast treatment for children with mild to moderate persistent asthma using data from the PACT clinical trial.16
The fluticasone treatment was shown to be cost-effective compared with montelukast both from point estimates and bootstrap simulations for all six cost-effectiveness measures analyzed.
To our knowledge, this report is the first to formally perform a comprehensive CEA comparing fluticasone and montelukast in mild-moderate childhood asthma based on a randomized trial conducted in the US. Previous CEA of asthma treatments compared other controller regimens.5–10, 19, 24, 28–30
Two studies,31, 32
though not formal CEA, compared the efficacy of fluticasone with montelukast relative to their costs using retrospective insurance claims data. With claims data on patients 4–17 years in 2001–2003, Stempel et al.32
found annualized asthma-related costs of $861 for fluticasone and $1616 for montelukast. Pathak et al.31
identified the annual treatment charges to be $572 (in 1999 dollars) for fluticasone and $902 for montelukast in patients 4–45 years of age. Both studies found fewer hospitalizations for those treated with fluticasone. The present study extends prior claims data based retrospective analyses of heterogeneous populations with no clinical outcome measures by showing conclusively that fluticasone was cost-effective compared with montelukast using data from a prospective clinical trial. Sensitivity analyses show that the cost-effectiveness of fluticasone over montelukast was robust in a wide range of settings, ensuring the generaliziblity of our study.
The present CEA also is the first to conduct subgroup analyses based on asthma phenotypic characteristics, and the cost-effectiveness of fluticasone over montelukast was substantiated for phenotypes indicating higher degrees of airway inflammation and hyper-responsiveness. This phenotypic subgroup analysis has similar implications as in Knuffman et al.27
in that baseline eNO levels greater than 25 ppb and PC20
value less than 2 mg/mL were more likely to show superiority of fluticasone over montelukast.
This study has several limitations. The unit cost estimates were taken from sources on pediatric asthma, but the inflators were based on the entire population rather than exclusively on children to adjust costs to the year 2003. Though this method was not ideal, sensitivity analysis showed the results were robust to a wide range of unit costs.
As to the societal cost, the monetary loss of productivity from missed school or work would vary greatly depending on the estimation method. We employed the Human Capital approach and used a published estimate, which assumed the loss to be a caregiver’s earnings. Methods to more accurately measure the societal cost of pediatric asthma are left for future study.
The PACT study found no significant difference in height growth between the two treatment groups.16
So no steroid effect on growth was considered in the cost-effectiveness analysis. The incorporation of potential steroid effect on growth into cost-effectiveness analysis was left for future research.
Rescue treatment in PACT included telephone contact with the study physician who would recommend starting oral corticosteroids if indicated by the study protocol. These contacts as well as use of oral corticosteroids occurred significantly more often in the montelukast group. It would be expected that this expeditious intervention reduced urgent care and emergent visits that would have occurred more frequently in the montelukast group than the fluticasone group if study physicians had not been available; this process could have caused an underestimation of the cost-effectiveness of fluticasone compared with montelukast.
Conclusions and Recommendations
Fluticasone is cost-effective compared with montelukast for children with mild to moderate persistent asthma. This CEA demonstrated that fluticasone dominated montelukast as it led to more ACD, a higher proportion of participants with 12% of FEV1 improvement, and fewer asthma exacerbations, yet at lower direct and societal costs.
Our study demonstrates the cost-effectiveness of low-dose fluticasone compared with montelukast in addition to the previously demonstrated clinical benefits in three clinically relevant asthma domains (asthma control days, lung function, and exacerbations) and further supports the NAEPP guidelines based on effectiveness that recommend inhaled corticosteroid monotherapy as the preferred asthma controller option for mild to moderate persistent asthma in children.