Market Report Source

Overview

Introduction
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.

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

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.

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”

Table of Contents

TABLE OF 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 192