Cystic Fibrosis | Unmet Need | US/EU | 2023

Cystic fibrosis (CF) is a genetic disease caused by any one of the more than 2,000 mutations identified in the cystic fibrosis transmembrane conductance regulator (CFTR) gene; such mutations lead to aberrant chloride transport, most notably in the lungs and pancreas, leading to pancreatic damage and persistent respiratory infections from the accumulation of thick, viscous mucus in the lungs. Prescription treatment options include Vertex’s franchise of disease-modifying therapies (DMTs), as well as a range of symptomatic therapies: antibiotics, mucolytics, bronchodilators, anti-inflammatory agents, and enzyme replacement therapies (ERTs). This content provides quantitative insight into U.S. and European pulmonologists’ perceptions of key treatment drivers and goals in the management of CF, the existing level of unmet need in this indication, and remaining commercial opportunities in the space.

QUESTIONS ANSWERED

• What are the treatment drivers and goals for CF? How well do current therapies perform on these goals?
• Which drug attributes are key influences, which have limited impact, and which are hidden opportunities?
• How do current therapies such as Trikafta / Kaftrio and Symdeko / Symkevi perform on key treatment drivers and goals for CF?
• Where do surveyed pulmonologists perceive the largest gaps 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 CF drug?

PRODUCT DESCRIPTION

Provides quantitative insight into U.S. and European physician perceptions of key treatment drivers and goals and the current level of unmet need for a specific disease. Commercial opportunities are analyzed, and the extent to which emerging therapies may capitalize on these opportunities is evaluated.

Markets covered: United States, France, Germany, United Kingdom

Primary research: Survey of 62 U.S. and 30 European pulmonologists fielded in April 2023

Key drugs: Kalydeco (ivacaftor), Orkambi (lumacaftor / ivacaftor), Symdeko / Symkevi (tezacaftor / ivacaftor and ivacaftor), Trikafta / Kaftrio (elexacaftor / tezacaftor / ivacaftor)