Stakeholder Opinions: Cystic Fibrosis

Summary

Approximately 50,000 cystic fibrosis patients worldwide still await a cure of their disease (WHO, 2004). Gene therapy experienced its first failure in 2005 therefore focusing attention on new strategies such as ion channel modulation. In the meantime, the administration of current drugs is improving, which should advance general patient-compliance.

Highlights

Although initially controversial, neonatal screening recommended by the Cystic Fibrosis Foundation and CDC is likely to become more widespread especially due to recent evidence that early diagnosis and intervention extends a better outcome.Extended antibacterial use due to increasing life-expectancy brings colonization with drug resistant P. aeruginosa, S. aureus and B. cepacia. Beyond the continued concerns with these prevalent pathogens, others such as non-tuberculosis mycobacteria and Stenotrophomonas maltophilia are increasingly in the minds of treating experts.After the first failure of gene therapy, the realization that treatment leading to cure is an unachievable goal drives limited research and development towards disease modification and symptomatic treatments. Datamonitor identified 15 ongoing cystic fibrosis related projects most of which were in Phase II clinical trials.

Scope

• Discusses disease background in detail, providing insight into cystic fibrosis epidemiology, etiology and symptoms
• Examines current diagnosis and treatment trends, providing physicians' opinions with regard to mucolytics, antibiotics, and anti-inflammatory drugs
• Defines the main unmet needs regarding the lack of cure, method of drug administration, and the need for new antibiotics
• Examines the cystic fibrosis pipeline by type of treatment, including a short discussion of each pipeline product

Reasons to Purchase

• Gain insight into the disease background and issues in the current diagnosis and treatment of cystic fibrosis through key opinion leader comments
• Review the unmet needs and the clinical and commercial factors driving new product decisions
• Identify the opportunities and threats presented by the cystic fibrosis pipeline and predict future trends in the market

Contents

• Chapter 1 Executive Summary
  • Scope
  • Datamonitor insight into the disease market
• Chapter 2 Disease Background
  • Cystic fibrosis has a low prevalence, mainly concentrated in Caucasians
  • Cystic fibrosis affects mainly Caucasians
  • The cystic fibrosis population is aging
  • Gender does not play a significant role in the occurrence of cystic fibrosis
  • Mortality is decreasing depending on age-group and gene mutation
  • Etiology of cystic fibrosis
  • The chances of a child born with CF is 25% if both parents are carriers of the mutated gene
  • Most common mutation inhibits chloride ion movement
  • Cystic fibrosis classifications can be based on symptoms, clinical history, chest X-rays, and/or genotypes
  • Cystic fibrosis symptoms affect several organs but have most significant impact on the lungs
  • Bacterial pathogens play a key role in infection and inflammation of cystic fibrosis lungs
  • Thickening of mucus in pancreas and intestines leads to maldigestion, malabsorption, and obstructions
  • Cystic fibrosis patients suffer from reproductive tract symptoms
  • Skeletal symptoms are caused by nutritional problems and adverse effects of steroids
  • Liver symptoms due to obstruction may require liver transplant
  • Common co-morbidities include diabetes, bone disease and nasal polyps
• Chapter 3 Diagnosis
  • Most cystic fibrosis sufferers are diagnosed by age three
  • Diagnosis of cystic fibrosis in adulthood is increasing
  • Cystic fibrosis patients diagnosed as adults enjoy improved prognosis
  • Most important diagnostic test remains 40-year-old sweat test
  • Antenatal and neonatal screening is not always standard
  • Other tests only confirm cystic fibrosis diagnosis
  • Diagnostic cystic fibrosis guidelines do not provide sufficient detail
  • Diagnostic complications include late, mis- and under-diagnosis
  • Current diagnostic tests offer opportunity for improvement
  • Factors influencing changes in diagnosis rates
• Chapter 4 Treatment Options
  • Mucolytics battle only symptoms
  • DNase
  • N-acetylcysteine (NAC)
  • Hypertonic saline
  • Antibiotics are mainstay treatment but can't entirely clear infections
  • Difference between antibiotic therapy in CF patients and non-CF patients lies in higher doses and longer treatment
  • Antibiotics used to treat infections in cystic fibrosis
  • Prescription of anti-inflammatories is contradictory to evidence
  • Corticosteroids
  • Non-steroidal anti-inflammatories drugs (NSAID)
  • Macrolides
  • Additional therapies with possible anti-inflammatory effects
  • Nutritional supplements focus on enzymes and vitamins
  • Physiotherapy is most important non-pharmacological therapy
  • Oxygen therapy and surgery are last resorts
  • Most physicians rely on center-specific treatment guidelines
  • Patient-compliance is key barrier to many treatment options
• Chapter 5 Future Trends
  • Ion channel therapy is the only promise in the pipeline
  • Gene therapy is in the far future
  • P. aeruginosa vaccines might provide valuable adjunct therapy
• Chapter 6 References
• Appendix1 Opinion Leader And Stakeholder Transcripts
  • UK opinion leader
  • UK opinion leader
  • US opinion leader
  • German opinion leader