Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta)

Abstract

CADTH recommends that Trikafta should be reimbursed by public drug plans for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least 1 F508del mutation in the CFTR gene if certain conditions are met. At least 1 of the following must be demonstrated after 6 months of treatment with Trikafta: an increase of at least 5% in percent predicted forced expiratory volume in 1 second (ppFEV1), a decrease in the number of pulmonary exacerbations or number of days that antibiotics are needed to be taken for pulmonary exacerbations, a decrease in CF-related hospitalizations, no decline in body mass index, or an improvement of at least 4 points in the Cystic Fibrosis Questionnaire-Revised respiratory domain scale. The price of Trikafta must be lowered to be cost-effective and affordable.