Alström Syndrome (AS) – Market Outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030

Alström Syndrome (AS) Market Outlook

Thelansis’s “Alström Syndrome (AS) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Alström Syndrome (AS) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Alström Syndrome (AS) Overview

Alström Syndrome (AS) is an ultra-rare multisystem genetic disorder caused by pathogenic variants of the ALMS1 gene. The disease is relentlessly progressive in nature which can result in premature death. Misdiagnosis is common. Because so many features of Alström Syndrome develop as children get older, more than seventy percent of the known Alström Syndrome patients have received previous misdiagnoses, and almost half of these have received multiple misdiagnoses. It progresses to severe visual impairment by the end of the second decade in 75%. Obesity develops in early childhood. Progressive bilateral sensorineural hearing loss is more variable, often presenting with high-frequency loss in the first decade but not detectable until much later in the minority. More than 60% of individuals with AS develop cardiomyopathy. This can present as potentially reversible dilated cardiomyopathy in infancy or presenting de novo or recurrence in adolescence with progression to a restrictive pattern. Insulin resistance is present from infancy- and progression to glucose intolerance is partly related to the degree of obesity. Molecular genetic testing of ALMS1, the only gene in which mutations are known to cause Alström syndrome, is estimated to detect mutations in 70%-80% of individuals of northern European descent and approximately 40% worldwide.

• • Prevalence range from 1:10,000 in communities where first cousin marriages are customary to fewer than 1:1,000,000 in populations with low levels of consanguineous marriages.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2021-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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