Real-world outcomes and direct care cost before and after elexacaftor/tezacaftor/ivacaftor initiation in commercially insured members with cystic fibrosis

by Selleckchem | Sep 02 2023

BACKGROUND: Cystic fibrosis (CF) is a chronic, progressive genetic disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene resulting in a dysfunctional CFTR protein. Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is a triple combination oral drug therapy with an annual cost greater than $300,000 and available to nearly 90% of the CF population based on age and genotype. Limited real-world direct medical cost offset data are available for ELX/TEZ/IVA among commercially insured individuals.

OBJECTIVE: To describe and compare total cost of care and health care resource utilization (HRU) 180 days before and 180 days after first ELX/TEZ/IVA drug claim among CFTR modulator treatment-naive, commercially insured members.

METHODS: This study was a retrospective analysis of integrated pharmacy and medical claims data from 17.9 million commercially insured members. A 180-day prestudy and 180-day poststudy design was used to compare outcomes prior to and following ELX/TEZ/IVA initiation. Study inclusion was limited to members with first ELX/TEZ/IVA claim (index date) between October 21, 2019, and December 31, 2021, continuously enrolled 180 days before and 180 days after index date, and no CFTR-modulator drug claim 180 days prior to index date. Total paid amounts from medical and pharmacy claims after network discounts (defined as total cost of care), HRU, and pulmonary exacerbation events were summarized using descriptive statistics and compared using Wilcoxon signed rank test.

RESULTS: 494 members newly initiating ELX/TEZ/IVA met inclusion criteria. Prestudy to poststudy mean member total cost of care increased from $58,180 to $198,815 (difference: $140,635; P < 0.001). Mean member medical benefit costs decreased from $28,764 to $12,484 (difference: -$16,280; P < 0.001), whereas mean member pharmacy benefit costs increased from $29,416 to $186,331 (difference: $156,915; P < 0.001). Mean member inpatient hospitalizations (62% absolute reduction; P < 0.001), emergency department visits (43% absolute reduction; P < 0.01), and pulmonary exacerbation events (44% absolute reduction; P < 0.001) were significantly lower in the postperiod compared with the preperiod.

CONCLUSIONS: Among members with CF newly initiating CFTR modulator with ELX/TEZ/IVA, mean member total cost of care increased 3-fold despite significant and meaningful reductions in pulmonary exacerbation events, HRU, and medical benefit spend. Pharmacy benefit spend outpaced medical benefit spend at a rate of $9.64 to $1 in the 180 days following ELX/TEZ/IVA initiation. Real-world data should be used to objectively measure the clinical and economic benefits of costly medications, such as CFTR modulators, to align price with value.

Comments:

The provided background is a summary of a study that examined the real-world medical cost offset and healthcare resource utilization (HRU) of the triple combination oral drug therapy, elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA), in commercially insured individuals with cystic fibrosis (CF). The study aimed to compare the total cost of care and HRU 180 days before and after the initiation of ELX/TEZ/IVA treatment in CF patients who had not previously received CFTR-modulator drugs.

The study analyzed integrated pharmacy and medical claims data from 17.9 million commercially insured members. The inclusion criteria for the study required individuals to have their first ELX/TEZ/IVA claim between October 21, 2019, and December 31, 2021, continuous enrollment for 180 days before and after the index date, and no CFTR-modulator drug claims in the 180 days prior to the index date.

The results of the study showed that among the 494 CF patients who met the inclusion criteria, the mean member total cost of care increased significantly from $58,180 to $198,815 after initiating ELX/TEZ/IVA treatment (a difference of $140,635, P < 0.001). The mean member medical benefit costs decreased from $28,764 to $12,484 (a difference of -$16,280, P < 0.001), while the mean member pharmacy benefit costs increased from $29,416 to $186,331 (a difference of $156,915, P < 0.001).

Additionally, the study found that there was a significant reduction in healthcare resource utilization after ELX/TEZ/IVA initiation. The mean member inpatient hospitalizations decreased by 62% (P < 0.001), emergency department visits decreased by 43% (P < 0.01), and pulmonary exacerbation events decreased by 44% (P < 0.001) during the poststudy period compared to the prestudy period.

In conclusion, the study observed a threefold increase in the mean member total cost of care following the initiation of ELX/TEZ/IVA treatment for CF. This increase was accompanied by significant reductions in HRU and medical benefit spend. The study highlights the need to objectively measure the clinical and economic benefits of costly medications like CFTR modulators, using real-world data, in order to align the price of these medications with their value.