Analyzing the use and impact of elexacaftor/tezacaftor/ivacaftor on total cost of care and other health care resource utilization in a commercially insured population.

Cystic fibrosis (CF) is a rare, life-threatening disease that results in severe respiratory, digestive, and metabolic problems. Elexacaftor/tezacaftor/ivacaftor is an oral drug that was approved by the US Food and Drug Administration (FDA) on October 21, 2019, after demonstrating clinical improvements compared with previous CF transmembrane conductance regulator modulators. Use of CF transmembrane conductance regulator modulators has improved CF care, but their high costs exceed commonly used cost-effectiveness thresholds. The Institute for Clinical and Economic Review issued an access and affordability alert warning that these high costs could threaten sustainable access to high-value care. There exists little real-world evidence on the uptake of elexacaftor/tezacaftor/ivacaftor and the impact on total cost of care and other health care resource utilization. This exploratory study analyzed the uptake and total cost-of-care impact of elexacaftor/tezacaftor/ivacaftor using pharmacy and medical claims data in a commercially insured patient population. To analyze the uptake of elexacaftor/tezacaftor/ivacaftor by members who qualified for treatment and to evaluate the differences in total cost of care and health care resource utilization in members who started treatment with elexacaftor/tezacaftor/ivacaftor. Uptake and per-member per-month information was obtained from Prime Therapeutics databases using cystic fibrosis transmembrane conductance regulator (CFTR) modulator claims. The total cost-of-care and resource utilization analysis used pharmacy and medical claims from Prime Therapeutics and Blue Cross NC across approximately 1.34 million commercially insured members over 20 months. Members with CF were identified by 2 or more codes (E84.xx) in any field at least 30 days apart or by a CFTR modulator claim. Only continuously enrolled members with CF with an elexacaftor/tezacaftor/ivacaftor pharmacy claim were included. The date of the first claim served as the index date. At 12 months after FDA approval, 77 (68%) Blue Cross NC members with CF were using elexacaftor/tezacaftor/ivacaftor. Of these, 33 had switched from a different CFTR modulator and 44 were naive to CFTR modulator therapy. Pharmacy and medical claims for 51 continuously enrolled members that initiated elexacaftor/tezacaftor/ivacaftor were analyzed. The average total cost of care increased by 52% ( < 0.00001). Hospitalizations decreased from an average of 7.7 (± 7.2) to 3.9 (± 5.5) ( < 0.00001). The sum and average number of infections were numerically lower, but the results did not meet statistical significance. Use of other supportive medications was numerically lower, but no statistically significant differences were observed. The uptake of elexacaftor/tezacaftor/ivacaftor was rapid, and the total cost of care increased despite reductions in hospitalizations and nonpharmacy costs. Differences in use of other CF-related medications appeared to be minimally affected. Dr Smith and Dr Borchardt have no financial conflicts of interest to report. Both authors are employed at BCBSNC at the time of writing. The project had no outside funding or sponsorship. The majority of the work and data analysis was completed as part of the requirements of the PGY1 Managed Care Pharmacy Residency program at BCBSNC during the 2020-2021 cycle year. This research does not meet the definition of human subject research as defined by the US Department of Health and Human Services at 45 C.F.R. § 46.102(f). According to definitions in section (e)(1), our research did not require either (i) information or biospecimens through intervention or interaction with any individuals or (ii) obtained, used, studied, analyzed, or generated private information or identifiable biospecimens. Therefore, institutional review board approval or a valid exemption is not required.