Cystic Fibrosis | Landscape & Forecast | Disease Landscape & Forecast

Publish date: July 2018

Login to access report

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. CFTR mutations lead to aberrant chloride transport in a variety of tissues, most notably the lungs and pancreas. As a result, CF patients generally suffer from pancreatic damage, which affects metabolism and nutrient absorption. Chronic respiratory problems, such as persistent lung infections stemming from the accumulation of thick, viscous mucus in the lungs, can result in respiratory failure—the leading cause of death in CF. Historically, therapeutic options for CF have been limited to therapies that dislodge mucus buildup, inhaled antibiotics, and pancreatic replacement therapy. With the market entry of Vertex Pharmaceuticals’ Kalydeco, Orkambi, and Symdeko, disease-modifying therapy (DMT) has become available for a subset of CF patients. Pipeline agents, including next-generation combinations, seek to further expand the reach of DMT in the CF population (e.g., triple combinations).

Table of contents

  • Disease Landscape & Forecast
    • Commercial Outlook
      • Key Findings
      • Drivers and Constraints
        • What Factors Are Driving Sales in Cystic Fibrosis?
        • What Factors Are Constraining Sales in Cystic Fibrosis?
      • Drug-Specific Trends
        • Kalydeco
        • Orkambi
        • Symdeko
        • Vertex's Triple-Therapy Combination
      • Alternative Market Scenarios
    • Forecast
    • Etiology and Pathophysiology
      • Key Findings
      • Etiology
        • Genotype-Phenotype Correlation
      • Pathophysiology
        • Respiratory Pathophysiology
        • Gastrointestinal, Hepatobiliary, and Pancreatic Pathophysiology
      • Clinical Presentation and Symptomology of Cystic Fibrosis
        • Symptoms and Complications of Cystic Fibrosis
      • Key Pathways and Drug Targets
        • CFTR Modulators for Cystic Fibrosis: Potentiators and Correctors
        • ENaC modulators
        • Key Inflammatory Pathways
    • Epidemiology Overview
      • Introduction
        • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases of Cystic Fibrosis in the United States and Europe, 2017-2027
        • Most Frequent CFTR Mutations by Country
        • Diagnosed Prevalent Cases of Cystic Fibrosis, Age Distribution, 2017
        • Drug-Treated Cases of Cystic Fibrosis
    • Current Treatment Overview
      • Key Findings
      • Diagnosis
        • Treatment Providers and Referral Patterns
      • Treatment Goals
        • Expert Insight on Key End Points Used in Clinical Trials for Cystic Fibrosis
      • Key Current Therapies
        • Overview
        • Disease-Modifying Therapies for Cystic Fibrosis
        • Mucolytics
        • Key Antibiotics Prescribed for Cystic Fibrosis
        • Pancreatic Enzyme Replacement Therapies for Cystic Fibrosis
      • Medical Practice
        • Overview
        • Treatment Start and Discontinuation with DMTs for Cystic Fibrosis
        • Treatment Guidelines
        • Drug Selection
        • Region-Specific Treatment
    • Unmet Need Overview
      • Key Findings
      • Attainment of Unmet Needs
        • Current and Future Attainment of Unmet Needs in Cystic Fibrosis
    • Emerging Therapies Overview
      • Key Findings
      • Key Emerging Therapies
        • Notable Developments Among Key Emerging Therapies for Cystic Fibrosis
        • Disease-Modifying Therapies
        • Antimicrobial Agents for Cystic Fibrosis
      • Early-Phase Pipeline Analysis
        • Notable Developments in the Early-Phase Pipeline for Cystic Fibrosis
        • Disease-Modifying Therapies
        • Mucolytics and ENaC Inhibitors
        • Antibiotics
        • Anti-Inflammatory Agents
      • Key Discontinuations and Failures in Cystic Fibrosis
      • Patient Registries
      • Orphan-Drug Designation
        • Orphan-Drug Provisions: United States
        • Orphan-Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
      • Looking for More?
    • Methodology
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Bibliography

Author(s): Claudia Dall'Osso; Fatima Garawi, MA, PhD

Claudia is a Senior Business Insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing on niche and rare indications. Before joining DRG, she held a management and strategy consultant position at Precision Medicine Group, where she worked for clients in the biopharmaceutical, medical device and diagnostic industries. Claudia completed her Master’s in Management at Harvard University; she also holds a in medical genetics from Brescia University in Italy and a BS/MS degree in medical biotechnology from University of Milano in Italy.

Fatima Garawi, MA, PhD, joined the Decision Resources Group epidemiology team in 2015 where she develops epidemiological forecasts for cardiovascular and respiratory diseases. She is the lead contact for COPD and Asthma client requests and also provides statistical and epidemiological support to internal teams and to clients on consulting-based projects. Dr. Garawi holds a MA in Statistics from Columbia University and a PhD in Epidemiology and Population Health from the London School of Hygiene and Tropical Medicine. Her PhD research focused on the social epidemiology of non-communicable diseases in the Middle East. Dr. Garawi is principal and co-author of numerous peer-reviewed articles. She previously held positions at Columbia University Medical Center in New York City, the World Bank in Washington, DC, and Goldman Sachs, NYC.