Cystic Fibrosis – Unmet Need – Unmet Need – Cystic Fibrosis (US EU)
Cystic fibrosis (CF) is a genetic disease associated with more than 2,000 mutations identified in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These mutations cause aberrant chloride transport, most notably in the pancreas and lungs, leading to pancreatic damage and persistent respiratory infections due to the accumulation of thick, viscous mucus in the lungs. Treatment options include Vertex’s portfolio of disease-modifying CFTR modulators such as ivacaftor (Kalydeco), lumacaftor / ivacaftor (Orkambi), tezacaftor / ivacaftor (Symdeko / Symkevi), elexacaftor / tezacaftor / ivacaftor (Trikafta / Kaftrio), and vanzacaftor / tezacaftor / deutivacaftor (Alyftrek). Symptomatic therapies include antibiotics, mucolytics, bronchodilators, anti-inflammatory agents, and pancreatic enzyme replacement therapies. While CFTR modulator therapies aim to restore CFTR protein function, they cannot cure the disease. Additional treatments in development with novel mechanisms of action (e.g., gene therapies, RNA-based therapies), along with next-generation CFTR modulators, will expand treatment options and fuel competition in the CF space.
Questions answered
- What are the key treatment drivers and goals for CF?
- How do current therapies such as Trikafta / Kaftrio and Alyftrek perform on key treatment drivers and goals for CF?
- Which drug attributes are key influencers of pulmonologists’ prescribing decisions, which have limited impact, and which are hidden opportunities?
- Where do surveyed pulmonologists perceive the largest areas of unmet need and opportunity in the treatment of CF?
- What trade-offs across different clinical attributes and price are acceptable to U.S. and European pulmonologists for a hypothetical new drug for CF?
Product description
- Evaluate clinical and nonclinical product attributes that influence treatment decisions through physicians’ weightings and analysis of stated vs. derived importance.
- Pinpoint areas of high unmet need by assessing current drug performance against key attributes and treatment drivers.
- Analyze market scenarios for different target product profiles using the TPP Simulator.
Content highlights
Publication date: March 2026
Geography: United States, France, Germany, United Kingdom
Primary research: Survey of 60 U.S. and 33 European pulmonologists
Key drugs covered: Symdeko / Symkevi, Trikafta / Kaftrio, Alyftrek
Key analyses:
- Importance of clinical and nonclinical product attributes to physicians
- Assessment of current drug performance against treatment drivers and goals
- Physician perceptions of unmet needs in the indication and related indications
- Remaining drug development opportunities
Key feature: Target Product Profile (TPP) simulator tool allows for customizable market simulations based on conjoint analysis. Compare up to seven TPPs across multiple disease-specific attributes and price points to gauge which variables influence prescribing behavior.
