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

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.

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.

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).

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