Epiomic Epidemiology Series: Niemann–pick Type C Disease Forecast in 11 Major Markets 2017–2027

October 2017 | 54 pages | ID: EE680772C22EN
Black Swan Analysis limited

Abstracts

Black Swan Analysis Epiomic Epidemiology Series Forecast Report on Niemann–Pick Type C Disease in 11 Major Markets

Niemann–Pick type C (NP-C) disease is a rare autosomal recessive lysosomal storage disorder with a variety of neurovisceral manifestations. It is caused by mutations in either NPC1 (more common) or NPC2 (less common) genes that lead to accumulation of cholesterol and glycosphingolipids in tissues. NP-C is divided according to the age at onset of symptoms, with an earlier onset of the disease predicting higher severity and poorer prognosis.

This report provides the current prevalent population for Niemann–Pick type C disease across 11 Major Markets (USA, Canada, France, Germany, Italy, Spain, UK, Japan, China, India, Brazil) 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, subsets and some features of Niemann–Pick type C disease patients, as well as a wide variety of symptoms and co-morbidities 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.

Main symptoms and co-morbidities for Niemann–Pick type C disease include:

• Seizures
• Cataplexy
• Dystonia and tremor
• Spasticity
• Insomnia
• Impaired swallowing
• Cognitive and psychiatric problems

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 Niemann–Pick type C disease market to target the development of future products, pricing strategies and launch plans.
• Gain further insight into the prevalence of the subdivided types of Niemann–Pick type C disease 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 co-morbid conditions on Niemann–Pick type C disease’s prevalent population.
• Identify sub-populations within Niemann–Pick type C disease which require treatment.
• Gain an understanding of the specific markets that have the largest number of Niemann–Pick type C disease 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 NIEMANN–PICK TYPE C DISEASE

FEATURES OF NIEMANN–PICK TYPE C DISEASE PATIENTS

COMORBID CONDITIONS OF NIEMANN–PICK TYPE C DISEASE 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. Classification of Niemann–Pick type C disease with age at onset and clinical features
Table 2. Usual age at death of NP-C patients
Table 3. Prevalence of Niemann–Pick Type C Disease, total (000s)
Table 4. Prevalence of Niemann–Pick Type C Disease, males (000s)
Table 5. Prevalence of Niemann–Pick Type C Disease, females (000s)
Table 6. Patients with NPC1 Gene Mutations by Sex (000s)
Table 7. Patients with NPC2 Gene Mutations by Sex (000s)
Table 8. NP-C Patients by Type of Disease, males (000s)
Table 9. NP-C Patients by Type of Disease, females (000s)
Table 10. NP-C Patients by Ambulation Ability, total (000s)
Table 11. NP-C Patients by Manipulation Ability, total (000s)
Table 12. NP-C Patients by Language Ability, total (000s)
Table 13. NP-C Patients by Swallowing Ability, total (000s)
Table 14. NP-C Patients with Hepatosplenomegaly, total (000s)
Table 15. NP-C Patients with Dysarthria, total (000s)
Table 16. NP-C Patients with Cerebellar Ataxia, total (000s)
Table 17. NP-C Patients with Vertical Supranuclear Ophthalmoplegia, total (000s)
Table 18. NP-C Patients with Cognitive Problems, total (000s)
Table 19. NP-C Patients with Movement Disorders, total (000s)
Table 20. NP-C Patients with Psychiatric Disorders, total (000s)
Table 21. NP-C Patients with Dysphagia, total (000s)
Table 22. NP-C Patients with Splenomegaly, total (000s)
Table 23. NP-C Patients with Hepatomegaly, total (000s)
Table 24. NP-C Patients with Chorea, total (000s)
Table 25. NP-C Patients with Epilepsy, total (000s)
Table 26. NP-C Patients with Parkinsonism, total (000s)
Table 27. NP-C Patients with Sleep Problems, total (000s)
Table 28. NP-C Patients with Jaundice, total (000s)
Table 29. Abbreviations and acronyms used in the report
Table 30. USA Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 31. USA Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 32. Canada Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 33. Canada Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 34. France Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 35. France Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 36. Germany Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 37. Germany Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 38. Italy Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 39. Italy Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 40. Spain Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 41. Spain Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 42. UK Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 43. UK Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 44. Japan Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 45. Japan Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 46. China Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 47. China Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 48. India Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 49. India Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)
Table 50. Brazil Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, males (000s)
Table 51. Brazil Prevalence of Niemann–Pick Type C Disease by 5-yr age cohort, females (000s)