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.