Alpha 1 Antitrypsin Deficiency: Key Trends and Statistics Just Published.

NEW YORK -- Reportlinker.com announces that a new market research report related to the Healthcare industry is available in its catalogue.

Stakeholder Opinions: Alpha 1 Antitrypsin Deficiency 

To order this report:

www.reportlinker.com/p075995/2008/02/Stakeholder-Opinions-Alpha-1- Antitrypsin-Deficiency.html

Despite being one of the most common hereditary diseases among Caucasians, low physician awareness and a lack of disease-modifying drugs have led to massive underdiagnosis of the condition. the resulting perceived small number of patients and restricted market is a major obstacle for the development of new treatments.

Understand the epidemiology of alpha-1 antitrypsin deficiency and assess the untapped patient potential. Evaluate current strategies for the diagnosis and treatment of this disease.Assess the potential for new drugs and their potential for additional usesGain an understanding of some of the principal challenges in the treatment of alpha 1 antitrypsin deficiency as defined by key opinion leaders.

Prevalence estimates have identified 200,000 heterozygous patients in the US and Europe making alpha-1 antitrypsin deficiency one of the most common hereditary disorders in the Western world. However, physician resistance has led to the diagnosis of only a small proportion of the estimated population.

Enzyme replacement augmentation therapy is available but lacks randomized, controlled efficacy data. Although widely used in the US, augmentation therapy is unavailable in the UK and Denmark. Patients who receive standard COPD treatment in the UK have comparable mortality rates to US patients who receive augmentation therapy.

Inhaled alpha-1 antitrypsin therapy holds promise for the prevention of disease progression but a lack of efficacy data and drug delivery issues are hindering development. Roche has a gamma retinoid agonist, R-667, in Phase II trials and the drug is the only potential therapy that offers lung regeneration through the promotion of alveolar growth.

Get an overview of alpha-1 antitrypsin deficient phenotypes and their prevalence in the US and Western European marketsAssess the market opportunity, where established therapies and other developmental compounds fail to address key unmet clinical needsEnhance your commercial positioning through an improved understanding of the alpha-1 antitrypsin deficiency market dynamics

ABOUT DATAMONITOR HEALTHCARE 2

About the Respiratory & Infectious Disease (RID) analysis team 2

CHAPTER 1 EXECUTIVE SUMMARY 3

Scope of the analysis 3

Datamonitor insight into the disease market 4

Related reports 4

CHAPTER 2 DISEASE BACKGROUND 6

Alpha-1 antitrypsin deficiency is a hereditary disease found mainly in Caucasians 6

Etiology of alpha-1 antitrypsin deficiency 6

Genetics of Alpha-1 antitrypsin deficiency 8

Epidemiology 10

Emphysema 12

Liver disease 14

Unmet needs 17

Risk factors 17

Smoking 18

Environmental tobacco smoke exposure 19

Occupational exposure 19

Bacterial infections 20

Body mass index 20

CHAPTER 3 DIAGNOSIS 21

Diagnosis of alpha-1 antitrypsin deficiency 21

Lung function 21

Imaging 21

Serum alpha-1 antitrypsin levels 22

Biochemical markers 22

Phenotyping 22

Diagnostic guidelines 22

Genetic screening 23

Increase in age at diagnosis and delay in diagnosis of alpha-1 antitrypsin deficiency 25

Problems with physician knowledge 27

Difficulty in conducting clinical trials in COPD 27

Declining smoking rates 28

CHAPTER 4 TREATMENT OPTIONS 29

Standard COPD therapy 29

Augmentation therapy 29

Therapeutic rationale 29

Market analysis 32

Cost and reimbursement 33

Antibiotic therapy 34

Organ transplant 34

Pulmonary rehabilitation, supplementary oxygen and genetic counseling 35

CHAPTER 5 FUTURE TRENDS 36

Inhaled alpha-1 antitrypsin augmentation therapy 36

Arriva/Hyland 37

Kamada 37

Talecris 37

Recombinant alpha-1 antitrypsin augmentation therapy 37

Gamma retinoid agonists 38

Gene therapy is in the far future 38

Alpha-1 antitrypsin replacement therapy in cystic fibrosis 38

Clinical trial endpoints 39

Continuing medical education 39

The role of patient support groups 40

Neonatal genetic screening 42

Transfer of treatments to the general emphysema population 43

CHAPTER 6 BIBLIOGRAPHY 44

Articles 44

Websites 49

List of Tables

Table 1: Alpha-1 antitrypsin levels in common genotypes 9

Table 2: Estimated prevalence of the five main phenotypes of alpha-1 antitrypsin deficiency in selected countries 11

Table 3: Estimated numbers of each of the five main phenotypes of alpha-1 antitrypsin deficiency in selected countries 12

Table 4: Classification of recommendations for genetic testing 24

Table 5: Comparison of augmentation therapies in the US 30

List of Figures

Figure 1: Alpha-1 antitrypsin production and activity 8

Figure 2: An example of three-generation pedigree with alpha-1 antitrypsin deficiency 10

Figure 3: Estimated numbers of PiZZ individuals in selected European countries 13

Figure 4: Liver disease in PiZZ patients by age 15

Figure 5: Association between liver dysfunction and age in PiZZ infants 16

Figure 6: Flow diagram of anticipated liver disease outcomes in PiZZ infants 17

Figure 7: Decline in lung function by smoking status 19

Figure 8: Hospital admissions for alpha-1 antitrypsin deficient patients in Norway, 2005 26

Figure 9: SWOT analysis of human, plasma derived augmentation therapy 33

Stakeholder Opinions: Alpha 1 Antitrypsin Deficiency

To order this report:

www.reportlinker.com/p075995/2008/02/Stakeholder-Opinions-Alpha-1- Antitrypsin-Deficiency.html

More market research reports?

Go to http://www.reportlinker.com