Cystic Fibrosis – Unmet Need – Detailed, Expanded Analysis (US/EU)
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)
Table of contents
- Cystic Fibrosis - Unmet Need - Detailed, Expanded Analysis (US/EU)
- Executive summary
- Unmet Need - Cystic Fibrosis - Executive Summary - July 2023
- Introduction
- Overview
- Methodology
- Rationale for treatment drivers and goals selection
- Rationale for drug selection
- Products for cystic fibrosis and rationale for drug selection
- Treatment drivers and goals
- Key findings: attribute importance
- Relative importance of efficacy, safety and tolerability, convenience of administration, and nonclinical attributes to surveyed pulmonologists' / pediatric pulmonologists' prescribing decisions in cystic fibrosis
- Importance of efficacy attributes to prescribing decisions in cystic fibrosis: United States
- Importance of efficacy attributes to prescribing decisions in cystic fibrosis: Europe
- Importance of safety and tolerability attributes to prescribing decisions in cystic fibrosis: United States
- Importance of safety and tolerability attributes to prescribing decisions in cystic fibrosis: Europe
- Importance of convenience of administration attributes to prescribing decisions in cystic fibrosis: United States
- Importance of convenience of administration attributes to prescribing decisions in cystic firbosis: Europe
- Importance of nonclinical factors to prescribing decisions in cystic fibrosis: United States
- Importance of nonclinical factors to prescribing decisions in cystic fibrosis: Europe
- Key findings: stated vs. derived importance
- Stated vs. derived importance of key efficacy, safety and tolerability, convenience of administration, and nonclinical attributes to prescribing decisions in cystic fibrosis: United States
- Stated vs. derived importance of key efficacy, safety and tolerability, convenience of administration, and nonclinical attributes to prescribing decisions in cystic fibrosis: Europe
- Product performance against treatment drivers and goals
- Key findings
- Overall performance of key therapies for cystic fibrosis: United States
- Overall performance of key therapies for cystic fibrosis: Europe
- Mean overall performance of key therapies for cystic fibrosis: United States and Europe
- Relative performance of key therapies for cystic fibrosis across select efficacy attributes: United States
- Relative performance of key therapies for cystic fibrosis across select efficacy attributes: Europe
- Relative performance of key therapies for cystic fibrosis across select safety and tolerability attributes: United States
- Relative performance of key therapies for cystic fibrosis across select safety and tolerability attributes: Europe
- Relative performance of key therapies for cystic fibrosis across select convenience of administration attributes: United States
- Relative performance of key therapies for cystic fibrosis across select convenience of administration attributes: Europe
- Relative performance of key therapies for cystic fibrosis across select nonclinical attributes: United States
- Relative performance of key therapies for cystic fibrosis across select nonclinical attributes: Europe
- Assessment of unmet need
- Key findings: unmet need in cystic fibrosis
- Surveyed pulmonologistsu2019 satisfaction with the performance of key therapies for cystic fibrosis on efficacy, safety and tolerability, convenience of administration, and nonclinical factors: United States
- Surveyed pulmonologistsu2019 satisfaction with the performance of key therapies for cystic fibrosis on efficacy, safety and tolerability, convenience of administration, and nonclinical factors: Europe
- Surveyed pulmonologists' ascribed level of unmet need across key efficacy attributes in cystic fibrosis: United States
- Surveyed pulmonologists' ascribed level of unmet need across key efficacy attributes in cystic fibrosis: Europe
- Surveyed pulmonologists' ascribed level of unmet need across key safety and tolerability attributes in cystic fibrosis: United States
- Surveyed pulmonologists' ascribed level of unmet need across key safety and tolerability attributes in cystic fibrosis: Europe
- Surveyed pulmonologists' ascribed level of unmet need across key convenience of administration attributes in cystic fibrosis: United States
- Surveyed pulmonologists' ascribed level of unmet need across key convenience of administration attributes in cystic fibrosis: Europe
- Surveyed pulmonologists' ascribed level of unmet need across key nonclinical factors in cystic fibrosis: United States
- Surveyed pulmonologists' ascribed level of unmet need across key nonclinical factors in cystic fibrosis: Europe
- Key findings: unmet need in cystic fibrosis and related indications
- Surveyed pulmonologists' ascribed level of unmet need in cystic fibrosis and related indications: United States
- Surveyed pulmonologists' ascribed level of unmet need in cystic fibrosis and related indications: Europe
- Opportunity analysis
- Areas of opportunity in the cystic fibrosis market and emerging therapy insights
- Opportunity: therapies that are more effective in enhancing lung function, thereby improving quality of life and overall survival
- Areas of opportunity in the cystic fibrosis market and emerging therapy insights
- Target product profiles
- Assessing drug development opportunities
- Target product profile methodology
- Attributes and attribute levels
- Attributes of key current therapies for cystic fibrosis
- Attribute importance and part-worth utilities
- Cystic fibrosis target product profile: attribute importance
- Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1)
- Absolute change in baseline in sweat chloride (SwCl)
- % reduction from baseline in the number of acute pulmonary exacerbations (PEx)
- Absolute change from baseline in CF Questionnaire-revised respiratory domain score
- % of patients experiencing headaches
- % of patients experiencing respiratory issues (e.g., upper respiratory tract infection)
- Price per year
- Conjoint analysis-based simulation of a market scenario
- Cystic fibrosis market simulation: share of preference of target product profiles included in the market scenario
- Cystic fibrosis market simulation: likelihood to prescribe of target product profiles included in the market scenario
- Cystic fibrosis market simulation: target product profiles included in the market scenario
- Appendix
- Key abbreviations
- Bibliography
- Executive summary
Shivangi Anand, M.Pharm., Associate Healthcare Research & Data Analyst, Cardiovascular, Metabolic, Renal, and Hematologic Diseases. Before joining Clarivate, Ms. Anand worked for a global market research and consulting firm, where she was involved in numerous drug-based and service-based projects and worked closely with clients, gaining experience in both secondary market research and competitive intelligence. She holds both a bachelor’s and a master’s degree in pharmaceutics from Chitkara University in Punjab, India.