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.