Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Jul 24:S1569-1993(25)01530-9.
doi: 10.1016/j.jcf.2025.07.005. Online ahead of print.

Treatment decision-making for using CFTR modulator therapy in patients with cystic fibrosis

Alison Carr  1 , Carlos Carneiro  2 , Michael Griffin  2 , Mark Higgins  2 , Heike Wöhling  2 , Fiona Hammond  1 , Donatello Salvatore  3 , Barry J Plant  4 , J Stuart Elborn  5
Affiliations
Free article

Treatment decision-making for using CFTR modulator therapy in patients with cystic fibrosis

Alison Carr et al. J Cyst Fibros. .
Free article

Abstract

Background: Five cystic fibrosis transmembrane conductance regulator (CFTR) modulator (CFTRm) therapies are approved for patients with cystic fibrosis (CF). Despite the established efficacy and safety of CFTRm therapy, variability exists in prescribing amongst CF physicians. Here, we describe prescriber factors influencing CFTRm use.

Methods: This observational study used clinical vignettes of patients with CF. Participants were adult pulmonologists and pediatricians specializing in respiratory medicine from Germany, Italy, UK, Ireland, and the Netherlands. Participants took part in a 3-step data collection process: (i) 60-minute individual telephone interview; (ii) decision exercise where physicians made a treatment decision about CFTRm on 10 clinical vignettes; and (iii) 30-minute web-assisted interview to provide rationale for treatment decisions. Univariate analysis, dimensionality reduction via factor analysis, and a random-effects logistic regression model were used to identify factors impacting CFTRm prescribing.

Results: Thirty-eight pulmonologists and 42 pediatricians provided 800 clinical decisions. Only 45 % of pulmonologists and 29 % of pediatricians prescribed a CFTRm for all eligible adult or pediatric vignettes. Pancreatic sufficiency decreased odds of prescribing a CFTRm (OR 0.106; 95 % CI 0.046, 0.244) whereas odds increased for patients' age >12 years (OR 3.779; 95 % CI 1.579, 9.042). Physician characteristics associated with CFTRm prescribing were being proactive/pushing boundaries (OR 1.772; 95 % CI 1.153, 2.722), having previously prescribed LUM/IVA (OR 2.732; 95 % CI 1.070, 6.974), and belief CFTRm therapies could improve adherence (OR 1.440; 95 % CI 0.946, 2.192).

Conclusions: Behavioral characteristics, attitudes toward CFTRm therapies, and experience with CFTRm therapies impact physician CFTRm prescribing more than individual disease factors.

Keywords: CFTR modulator; Clinical vignette; Cystic fibrosis; ELX/TEZ/IVA; Prescribing.

PubMed Disclaimer

Conflict of interest statement

Declaration of competing interest CC, MG, MH, and HW are employees of Vertex Pharmaceuticals and may own stock or stock options in the company. AC and FH are employees of Hammell which received support from Vertex Pharmaceuticals for this study. BJP has received both speakers’ fees and consulting fees from Vertex Pharmaceuticals, Chiesi Pharmaceutical and Viatris Pharmaceutical, outside the submitted work. DS and JSE declare no conflict of interest.

LinkOut - more resources