Epiomic Epidemiology Series: Cystinosis Forecast in 19 Major Markets 2017-2027

September 2017 | 58 pages | ID: E2663405C41EN
Black Swan Analysis limited

Abstracts

Black Swan Analysis Epiomic Epidemiology Series Forecast Report on Cystinosis in 19 Major Markets

Cystinosis is a rare inherited lysosomal storage disorder caused by mutations in the CTNS gene that encodes cystinosin, a lysosomal cystine–proton co-transporter. This results in an abnormal accumulation of the amino acid cystine within the lysosomes of all body cells and tissues. Although it is a monogenic, autosomal recessive disease, there are three clinical presentations of cystinosis based on the age at diagnosis and the magnitude of cellular cystine deposition

This report provides the current prevalent population for cystinosis across 19 Major Markets (USA, Canada, France, Germany, Italy, Spain, UK, Poland, Austria, The Netherlands, Ireland, Turkey, Japan, South Korea, India, Australia, Brazil, Mexico, Argentina) split by gender and 5-year age cohort. Along with the current prevalence, the report also contains a disease overview of the risk factors, disease diagnosis and prognosis along with specific variations by geography and ethnicity.

Providing a value-added level of insight from the analysis team at Black Swan, the three clinical presentations of cystinosis have been quantified and presented alongside the overall prevalence figures. These sub-populations within the main disease are also included at a country level across the 10-year forecast snapshot.

This report is built using data and information sourced from the proprietary Epiomic patient segmentation database. To generate accurate patient population estimates, the Epiomic database utilises a combination of several world class sources that deliver the most up to date information form patient registries, clinical trials and epidemiology studies. All of the sources used to generate the data and analysis have been identified in the report.

Reason to buy

• Able to quantify patient populations in global cystinosis market to target the development of future products, pricing strategies and launch plans.
• Gain further insight into the prevalence of the subdivided types of cystinosis and identify patient segments with high potential.
• Delivery of more accurate information for clinical trials in study sizing and realistic patient recruitment for various countries.
• Provide a level of understanding on the impact from specific clinical presentations of the disease on cystinosis prevalent population.
• Identify sub-populations within cystinosis which require treatment.
• Gain an understanding of the specific markets that have the largest number of cystinosis patients.

Contents

INTRODUCTION

CAUSE OF THE DISEASE

RISK FACTORS & PREVENTION

DIAGNOSIS OF THE DISEASE

VARIATION BY GEOGRAPHY/ETHNICITY

DISEASE PROGNOSIS & CLINICAL COURSE

KEY COMORBID CONDITIONS / FEATURES ASSOCIATED WITH THE DISEASE

METHODOLOGY FOR QUANTIFICATION OF PATIENT NUMBERS

TOP-LINE PREVALENCE FOR CYSTINOSIS

TYPES OF CYSTINOSIS IN PATIENTS

ABBREVIATIONS USED IN THE REPORT

OTHER BLACK SWAN ANALYSIS PUBLICATIONS

BLACK SWAN ANALYSIS ONLINE PATIENT-BASED DATABASES

PATIENT-BASED OFFERING

ONLINE PRICING DATA & PLATFORMS

REFERENCES

APPENDIX

List Of Tables

LIST OF TABLES AND FIGURES

Table 1. Prevalence of Cystinosis, total (000s)
Table 2. Prevalence of Cystinosis, males (000s)
Table 3. Prevalence of Cystinosis, females (000s)
Table 4. Patients with Nephropathic Infantile Cystinosis, total (000s)
Table 5. Patients with Late-onset Nephropathic Cystinosis, total (000s)
Table 6. Patients with Non-nephropathic Cystinosis, total (000s)
Table 7. Abbreviations and Acronyms used in the report
Table 8. USA Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 9. USA Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 10. Canada Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 11. Canada Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 12. France Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 13. France Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 14. Germany Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 15. Germany Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 16. Italy Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 17. Italy Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 18. Spain Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 19. Spain Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 20. UK Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 21. UK Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 22. Poland Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 23. Poland Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 24. Austria Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 25. Austria Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 26. The Netherlands Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 27. The Netherlands Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 28. Ireland Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 29. Ireland Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 30. Turkey Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 31. Turkey Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 32. Japan Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 33. Japan Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 34. South Korea Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 35. South Korea Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 36. India Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 37. India Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 38. Australia Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 39. Australia Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 40. Brazil Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 41. Brazil Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 42. Mexico Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 43. Mexico Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Table 44. Argentina Prevalence of Cystinosis by 5-yr age cohort, males (000s)
Table 45. Argentina Prevalence of Cystinosis by 5-yr age cohort, females (000s)
Figure 1. Schematic representation of the CTNS gene and all reported mutations in cystinosis patients.