Cystic Fibrosis | Unmet Need | US/EU | 2024
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. Treatment options include Vertex’s portfolio of disease-modifying CFTR modulator therapies, as well as a range of symptomatic therapies: antibiotics, mucolytics, bronchodilators, anti-inflammatory agents, and enzyme replacement therapies (ERTs). While CFTR modulator therapies aim to restore the function of the CFTR protein, they cannot cure the disease. Additional treatments in development with novel mechanisms of action (e.g., genetic therapies) and advanced 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 well do current therapies perform on these goals?
- Which drug attributes are key influencers, 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 prices are acceptable to U.S. and European pulmonologists for a hypothetical new CF drug?
Markets covered: United States, France, Germany, United Kingdom
Primary research: Survey of 61 U.S. and 32 European pulmonologists fielded in February 2024
Key drugs: Kalydeco (ivacaftor), Orkambi (lumacaftor / ivacaftor), Symdeko / Symkevi (tezacaftor / ivacaftor and ivacaftor), Trikafta / Kaftrio (elexacaftor / tezacaftor / ivacaftor)
Product description
Unmet Need supports clinical development decisions by identifying key attributes and assessing areas of unmet need for a specific disease or subpopulation. Based on surveys with U.S. and European physicians, this report provides insight into key treatment drivers and goals, the performance of current therapies, and the remaining commercial opportunities. One market scenario is profiled in detail by Clarivate experts, and additional customized market scenarios can be evaluated with the corresponding TPP Simulator.
Key feature
The Target Product Profile (TPP) Simulator tool allows for customizable market simulations based on conjoint analysis that depicts how physicians make decisions based on actual behavior rather than opinion. Compare up to seven TPPs across multiple disease-specific attributes and price points to gauge which variables influence prescribing behavior.
