Epiomic Epidemiology Series: Primary Carnitine Deficiency Forecast in 19 Major Markets 2018–2028

December 2017 | 58 pages | ID: E3FF2D8E0DBEN
Black Swan Analysis limited

Abstracts

Black Swan Analysis Epiomic Epidemiology Forecast Report on Primary Carnitine Deficiency in 19 Major Markets

Systemic Primary Carnitine Deficiency (CDSP) is a rare autosomal recessive disorder caused by mutations in the SLC22A5 gene encoding organic cation transporter type 2 (OCTN2) that transports carnitine across cell membranes. To date, more than 160 unique mutations in the SLC22A5 gene have been reported, and the type of mutation affects the severity of symptoms, with some carriers of less severe mutations being asymptomatic.

This report provides the current prevalent population for CDSP across 19 Major Markets (USA, Canada, France, Germany, Italy, Spain, UK, Poland, Netherlands, Russia, Turkey, Japan, China, South Korea, India, Australia, Brazil, Mexico, Argentina) split by gender and 5-year age cohort. In addition to the current prevalence, the report provides an overview of the risk factors, diagnosis and prognosis of the disease, along with specific variations by geography and ethnicity.

Providing a value-added level of insight from the analysis team at Black Swan, several features of CDSP patients, as well as the main symptoms and comorbidities of the disease 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 of CDSP include:

• Hepatic and cardiac signs and symptoms
• Anaemia
• Proximal muscle weakness
• Developmental delay
• Respiratory distress

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

• Ability to quantify patient populations in global CDSP market to target the development of future products, pricing strategies and launch plans.
• Further insight into the prevalence of the subdivided types of CDSP and identification of patient segments with high potential.
• Delivery of more accurate information for clinical trials in study sizing and realistic patient recruitment for various countries.
• Better understanding of the impact of specific co-morbid conditions on the prevalent population of CDSP patients.
• Identification of CDSP patient sub-populations that require treatment.
• Better understanding of the specific markets that have the largest number of CDSP 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 PRIMARY CARNITINE DEFICIENCY

FEATURES OF PRIMARY CARNITINE DEFICIENCY PATIENTS

ABBREVIATIONS USED IN THE REPORT

OTHER BLACK SWAN SERVICES & SOLUTIONS

REPORTS & PUBLICATIONS

ONLINE EPIDEMIOLOGY DATABASES

ONLINE PHARMACEUTICAL PRICING DATABASE

REFERENCES

APPENDIX

List Of Tables

LIST OF TABLES AND FIGURES

Table 1. Prevalence of primary carnitine deficiency, total (000s)
Table 2. Prevalence of primary carnitine deficiency, males (000s)
Table 3. Prevalence of primary carnitine deficiency, females (000s)
Table 4. Patients with primary carnitine deficiency by presence of clinical symptoms, total (000s)
Table 5. Patients with primary carnitine deficiency by phenotype, total (000s)
Table 6. Primary carnitine deficiency patients with cardiomyopathy, total (000s)
Table 7. Primary carnitine deficiency patients with hyperammonaemia, total (000s)
Table 8. Primary carnitine deficiency patients with hypoglycaemia, total (000s)
Table 9. Primary carnitine deficiency patients with metabolic acidosis, total (000s)
Table 10. Primary carnitine deficiency patients with Reye-like syndrome, total (000s)
Table 11. Primary carnitine deficiency patients with less energetic behaviour, total (000s)
Table 12. Abbreviations and acronyms used in the report
Table 13. USA prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 14. USA prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 15. Canada prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 16. Canada prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 17. France prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 18. France prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 19. Germany prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 20. Germany prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 21. Italy prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 22. Italy prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 23. Spain prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 24. Spain prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 25. UK prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 26. UK prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 27. Poland prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 28. Poland prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 29. Netherlands prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 30. Netherlands prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 31. Russia prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 32. Russia prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 33. Turkey prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 34. Turkey prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 35. Japan prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 36. Japan prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 37. China prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 38. China prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 39. South Korea prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 40. South Korea prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 41. India prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 42. India prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 43. Australia prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 44. Australia prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 45. Brazil prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 46. Brazil prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 47. Mexico prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 48. Mexico prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)
Table 49. Argentina prevalence of primary carnitine deficiency by 5-yr age cohort, males (000s)
Table 50. Argentina prevalence of primary carnitine deficiency by 5-yr age cohort, females (000s)