Early insurance coverage of elexacaftor/tezacaftor/ivacaftor for cystic fibrosis in children 6 to 11 years of age

Abstract

Introduction: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is a highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy (HEMT) originally approved in 2019 for use in patients 12 years of age and older with at least one F508del mutation or a mutation in the CFTR gene that is responsive based on in vitro data. This report describes coverage of ELX/TEZ/IVA for CF in children 6 to 11 years of age prior to the recent age expansion by the Food and Drug Administration (FDA). Methods: An email was sent to pharmacists and pharmacy technicians through the CF Foundation LISTSERV and all responses regarding ELX/TEZ/IVA use in children 6 to 11 years of age were collected. Results: A total of 65 children from 15 CF care centers were included in the study. A total of 55 children had early coverage of ELX/TEZ/IVA for an overall approval rate of 84.6%. The median time to approval was 15 days. Lung function and weight outcomes were also positive. Conclusions: Early insurance coverage of ELX/TEZ/IVA for CF in children 6 to 11 years was achieved regardless of insurance type and should be considered an option for patients in need of HEMT therapy.