Cost-effectiveness of CFTR Modulators
Thus far, few studies have demonstrated cost-effectiveness for CFTR modulators. This year, a retrospective study in Ireland of a national pharmacy claims database found that although there was an eight-fold increase in prescribing for all modulators, there was no reduction in other chronic therapy medication prescriptions59. Specifically, for iva, there was a reduction in patients receiving symptomatic treatments (dornase alfa and inhaled tobramycin, p<0.001), but no reduction was seen for lum/iva; however, an increase in colistimethate (p=0.01) was seen. Based on this pharmacy data alone, and no inclusion of other resource utilization such as hospitalization, cost effectiveness was not found for iva and lum/iva while tez/iva remains uncertain. A simulation model in the US, evaluated the economics of iva use over a lifetime, starting at age 6 months, compared to best supportive care alone60. Iva was found to have improved quality-adjusted life years (QALYs) over supportive care (22.92 vs 16.12) but cost approximately $6.4 million more in total lifetime costs. Scenario projections demonstrated that best supportive care was the most cost-effective treatment up to a willingness to pay $1 million, well above the commonly accepted willingness to pay threshold of $500,000 where iva’s cost effectiveness was 0%.