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%.