Results
A total of 65 patients from 15 different CF care centers were included in the analysis. The average age of the patients was 10 (1.5) years and ranged from 6-11.8 years. Most patients were male (52.8%) and homozygous F508del genotype (56.9%). (Table 1) Thirty-six patients (55.4%) were on a different CFTR modulator prior to ELX/TEZ/IVA, while 29 patients (44.6%) were modulator naive. Patient insurance was evenly split with 32 patients (49.2%) having commercial coverage and the same number having state-funded insurance plans, such as Medicaid.
A total for 55 children were able to obtain early access to ELX/TEZ/IVA. The overall approval rate was 84.6%. The median time until insurance approval was 15 days. Approval occurred on the initial prior authorization (PA) for 19 (29.2%), 22 (33.8%) on first appeal, 10 (15.4%) on the second appeal, and 2 (3%) on the third appeal. The most common types of appeals utilized were letter of medical necessity (LMN) and external review (ER). For first appeal, there were 39 LMN, 3 peer to peer reviews (P2P), and 2 additional information requested. On second appeal, there were 6 P2P, 2 LMN, 6 ER, 1 additional information, 2 LMN/ER combination, and 1 family LMN. Those that required third appeals were 2 ER.
There was some variation in the dose of ELX/TEZ/IVA prescribed for these patients. The majority, 30 (54.6%), received full dose, 12 (21.8%) received half dose, and 8 (15%) had a dose titration. Drug interactions accounted for 3 of the dose adjustments. The average duration of ELX/TEZ/IVA at data collection was 0.5 (0.4) years. After the first quarter of ELX/TEZ/IVA, patients had an average weight gain of 2.5 kg and mean change in ppFEV1 of 13.3%. After the second quarter of therapy, average weight gain was 4.5 kg from baseline and mean change in ppFEV1 was 16.4% from baseline.