Abstract Rationale Clinical outcomes for people with cystic fibrosis (pwCF) have improved over time in part due to improvements in pharmacotherapy, including cystic fibrosis transmembrane conductance receptor (CFTR) modulator therapies and non-modulator treatments. Data regarding changes in prescription fills and retail spending for non-modulator treatments, after starting CFTR modulators, is limited. We quantified changes in fills and retail spending for four non-modulator treatments in the era of modulator therapy among patients insured by Medicaid. We hypothesized that with increasing modulator use, there would be decreased prescription fills and retail spending for non-modulator treatments. Methods We performed a serial cross-sectional assessment of US Medicaid prescription data using the Centers for Medicare and Medicaid Services (CMS) drug spending dashboard which provides volume and retail spending data from 2012 to 2023 by patients enrolled in Medicaid. Prescription fills and retail spending by year were obtained for four non-modulator treatments most specific to pwCF (inhaled dornase alfa, inhaled aztreonam, inhaled tobramycin, and pancreatic enzyme replacement therapy PERT), and modulator therapies (ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor ETI). We used descriptive statistics to analyze prescription fills and retail spending over time. Results From 2012 to 2023, prescription fills decreased by 20% for inhaled aztreonam, 14% for inhaled dornase alfa, 30% for inhaled tobramycin, and 19% for all inhaled therapies. Fills for PERT increased by 68%. Through 2023, prescription fills increased by 15% for ivacaftor, 31% for lumacaftor/ivacaftor, and 2606% for ETI; tezacaftor/ivacaftor decreased by 90%. From 2012 to 2023, retail spending increased by 72% for inhaled aztreonam and 46% for inhaled dornase alfa; inhaled tobramycin decreased by 55%, and all inhaled therapies increased by 8%. Retail spending for PERT increased by 378%. Through 2023, retail spending increased by 21% for ivacaftor, 41% for lumacaftor/ivacaftor, 2755% for ETI, and 1311% for any CFTR modulator; tezacaftor/ivacaftor decreased by 89%. Image 1 illustrates the retail spending for inhaled therapies and any CFTR modulator by year. Conclusion After introduction of modulator therapies, prescription fills for all inhaled therapies decreased by 19%. However, retail spending for all inhaled therapies increased by 8%. One limitation of this study is that the CMS data includes all patients enrolled in Medicaid, and not only pwCF. Additionally, retail spending does not account for confidential manufacturer rebates for brand name medications. Future investigation is needed to explore the impact of manufacturer rebates on costs, as well as trends in other non-modulator treatments such as inhaled hypertonic saline. This abstract is funded by: None
Moo-Young et al. (Fri,) studied this question.