Prader-Willi Syndrome (PWS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Prader-Willi Syndrome (PWS) Market Outlook

Thelansis’s “Prader-Willi Syndrome (PWS) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" 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 Prader-Willi Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Prader-Willi Syndrome (PWS) Overview

Prader-Willi Syndrome (PWS) is a rare genetic disorder affecting multiple body systems. It is caused by a deletion of genes on chromosome 15. Infants with PWS have weak muscle tone and feeding difficulties, which can lead to poor weight gain and growth. They also have distinctive facial features. Children with PWS are typically short, have small genitals, develop an excessive appetite, and lack satiety. This can lead to severe obesity if not managed carefully. Other associated endocrine abnormalities include growth hormone deficiency, hypogonadism, hypothyroidism, premature pubarche, and rarely corticotropin deficiency. Cognitive dysfunction is common in PWS, ranging from mild to moderate. Children with PWS may have learning disabilities, impaired speech and language development, and emotional control difficulties. The syndrome may also result in comorbidities such as diabetes, sleep-related breathing disorders, gastrointestinal problems, and infections. Early genetic testing and multidisciplinary care are essential for individuals with PWS. Growth hormone treatment is a key component of treatment, as it can stabilize body mass index, improve linear growth and cognitive development, and reduce comorbidities. While full autonomy cannot be reached, growth hormone treatment can help individuals with PWS lead better lives.

• The incidence of PWS at birth in the United States is estimated to be 1/10,000 to 1/30,000. There are 10,000 to 20,000 living individuals with PWS in the United States.

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: 2023-2033

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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