Pompe Disease - Epidemiology Forecast to 2030

Pompe Disease - Epidemiology Forecast to 2030

  • May 2020 •
  • 100 pages •
  • Report ID: 5897618 •
  • Format: PDF
‘Pompe Disease (PD) - Epidemiology Forecast to 2030’ report delivers an in-depth understanding of the disease, historical & forecasted epidemiology of Pompe Disease in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom) and Japan.

Pompe Disease (PD) Understanding

Pompe disease (PD), also known as glycogen storage disease type II (GSDII) or “acid maltase deficiency”, is caused by the absence or deficiency of acid alpha-glucosidase (GAA), a lysosomal enzyme that is responsible for the cleavage of the ?-1, 4- and ?-1, 6-glycosidic bonds of glycogen to glucose. The disease is caused pathogenic variations in the acid alpha-glucosidase (GAA) gene. Close to 500 different GAA gene variations have been identified in families with this disorder.

Pompe Disease Epidemiology Perspective

The Pompe Disease (PD) epidemiology division provide the insights about historical and current patient pool and forecasted trend for every seven major countries. The epidemiology data for Pompe Disease are studied through all possible division to give a better understanding about the Disease scenario in 7MM. It also helps to recognize the causes of current and forecasted trends by exploring numerous studies, survey reports and views of key opinion leaders.

Pompe Disease Detailed Epidemiology Segmentation

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total live birth cases of PD, Total Adult Prevalent cases of PD, Total Prevalent cases of PD, Comorbidities Prevalence of PD by Onset Types, Incidence of IOPD based on clinical phenotypes) scenario of Pompe Disease (PD) in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017–2030.
The PD report also provides the epidemiology trends observed in the 7MM during the study period, along with the assumptions undertaken. The calculated data are presented with relevant tables and graphs to give a clear view of the epidemiology at first sight.

Scope of the Report
• The report covers detailed overview of Pompe Disease explaining its causes, symptoms, classification, pathophysiology, diagnosis and treatment patterns
• The Pompe Disease (PD) Report and Model provide an overview of the risk factors and global trends of PD in the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan)
• The report provides the insight about the historical and forecasted patient pool of Pompe Disease in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK) and Japan
• The Report assesses the disease risk and burden and highlights the unmet needs of the disease
• The Report helps to recognize the growth opportunities in the 7MM with respect to the patient population
• The report provides the segmentation of the disease epidemiology by Total live birth cases of PD, Total Adult Prevalent cases of PD, Total Prevalent cases of PD, Comorbidities Prevalence of PD by Onset Types, Incidence of IOPD based on clinical phenotypes in 7MM

Report Highlights

• 11 Years Forecast
• 7MM Coverage
• Total Diagnosed Prevalent Cases of Pompe Disease
• Prevalent Cases according to segmentation: Total live birth cases of PD, Total Adult Prevalent cases of PD, Total Prevalent cases of PD, Comorbidities Prevalence of PD by Onset Types, Incidence of IOPD based on clinical phenotypes
KOL- Views

We interview, KOLs and SME’s opinion through primary research to fill the data gaps and validate our secondary research. The opinion helps to understand the total patient population and current treatment pattern. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the indications.

Key Questions Answered

• What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Pompe Disease?
• What are the key findings pertaining to the Pompe Disease epidemiology across 7MM and which country will have the highest number of patients during the forecast period (2020–2030)?
• What would be the total number of patients of Pompe Disease across the 7MM during the forecast period (2020–2030)?
• Among the EU5 countries, which country will have the highest number of patients during the forecast period (2020–2030)?
• At what CAGR the patient population is expected to grow in 7MM during the forecast period (2020–2030)?
• What is the disease risk, burden and unmet needs of the Pompe Disease?
• What are the current available treatments of Pompe Disease?

Reasons to buy
The Pompe Disease Epidemiology report will allow the user to -
• Develop business strategies by understanding the trends shaping and driving the global Pompe Disease market
• Quantify patient populations in the global Pompe Disease market to improve product design, pricing, and launch plans
• Organize sales and marketing efforts by identifying the age groups that present the best opportunities for Pompe Disease therapeutics in each of the markets covered
• Understand the magnitude of Pompe Disease population by its severity
• The Pompe Disease epidemiology report and model were written and developed by Masters and PhD level epidemiologists
• The Pompe Disease Epidemiology Model developed is easy to navigate, interactive with dashboards, and epidemiology based with transparent and consistent methodologies. Moreover, the model supports data presented in the report and showcases disease trends over 11-year forecast period using reputable sources

Key Assessments

• Patient Segmentation
• Disease Risk and Burden

• Risk of disease by the segmentation
• Factors driving growth in a specific patient population

Geographies Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan
Study Period: 2017–2030
Although Pompe disease is a rare autosomal recessive disorder there have been attempts at estimating the overall worldwide incidence with results yielding a value of ~1/40,000. Organizations such as the National Organization for Rare Disorders (NORD) and United Pompe Foundation have also quoted such figures in their publications and journals. However, a fear of underestimation has always prevailed despite adequate precautionary measures due to the inherent difficulty in diagnosis
Newborn screening studies have forced experts to revise previously accepted incidence/prevalence numbers and DelveInsight has kept its focus on these recent happenings while formulating the Epidemiology model for 7 MM