Introduction
The highly effective cystic fibrosis (CF) transmembrane conductance
regulator (CFTR) modulator therapy (HEMT),
elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was approved in October
2019 for patients 12 years of age and older with at least one F508del
mutation or a mutation that is responsive based on in vitro data.
Recently released phase 3 data for ELX/TEZ/IVA use in children 6 to 11
years of age showed positive safety and tolerability results, as well as
secondary endpoint improvements in ppFEV1, sweat chloride, Cystic
Fibrosis Questionnaire-Revised (CFQ-R) scores, body mass index (BMI), as
well as number of pulmonary exacerbations and
hospitalizations.1 Studies have also documented that
initiating HEMT at an earlier age has the potential to alter the
progression and prognosis of CF.2-4 Based on this
information, a supplemental New Drug Application to expand the use of
ELX/TEZ/IVA to include children 6 to 11 years was submitted in early
2021 with a targeted decision date of June 2021. Given the positive
study results and the potential time-sensitive nature of its use,
several CF centers submitted prior authorizations (PA) for ELX/TEZ/IVA
use in younger children. The aim of this project was to describe early
insurance coverage of ELX/TEZ/IVA for CF in children ages 6 to 11 years.