Although current research suggests that for most people with CF, mental health remains stable or might improve after starting CFTR modulator treatment, some individuals experienced new or worsening symptoms, including anxiety, depression, thoughts of self-harm, and sleep problems. As a result, updated prescribing labels (the FDA-approved package insert that accompanies prescriptions) for modulators recommend that clinicians monitor patients for new or worsening mental health symptoms and, in consultation with them, decide on the best course of action, which may include a pause in treatment if necessary.

The Cystic Fibrosis Foundation strongly recommends that people with CF talk with their care team before making any changes to their medications, as abruptly stopping a modulator can lead to a significant decline in lung function or other negative health effects. People with CF can reference the Navigating Mental Health and CFTR Modulators guide for suggestions on how to discuss this topic with their care teams.

The Cystic Fibrosis Foundation supports care and research focused on mental health in CF. In 2016, the Foundation established the Mental Health Advisory Committee to promote mental well-being and reduce the mental health burden for people with CF and their families through screening, evidence-based treatments, mental health services, and research-informed care. The Foundation also provides financial support to CF care centers to coordinate mental health screening and services.

To guide research priorities, the Foundation formed the Prioritizing Research in Mental Health (PRIME) Working Group, a multidisciplinary group of researchers, clinicians, and individuals and families affected by CF. In addition, the Foundation is supporting research to better understand the relationship between CFTR modulators and mental health, including the RETRIAL study, the TIDES 2.0 study, and basic (laboratory) research examining how modulators affect the central nervous system.