The cost of simplifying treatments for cystic fibrosis: Implications of the SIMPLIFY trial.

ABSTRACT:

Background

Dornase alfa and hypertonic saline are mucoactive therapies that can improve respiratory symptoms in people with cystic fibrosis (CF). A recent randomized control trial showed that participants with well-preserved pulmonary function taking elexacaftor + tezacaftor + ivacaftor (ETI) who discontinued dornase alfa or hypertonic saline for 6 weeks had no clinically meaningful decline in lung function. This may prompt discussions with care providers regarding ongoing use of these medications.

Objective

To compare the costs of outpatient medications between people taking ETI who continued or discontinued (1) dornase alfa or (2) hypertonic saline from 2 clinical trials and project cost differences in the US CF population if these 2 medications were used only intermittent

SUBMITTER: Gold LS 

PROVIDER: S-EPMC10775778 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature