Camurati-Engelmann Disease (CED) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Camurati-Engelmann Disease (CED) Market Outlook

Thelansis’s “Camurati-Engelmann Disease (CED) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 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 Camurati-Engelmann Disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Camurati-Engelmann Disease (CED) Overview

Camurati-Engelmann Disease (CED) is a rare genetic condition characterized by excessive bony growth affecting the long bones, skull, spine, and pelvis. Excessive growth leads to clinical symptoms such as severe pain in the extremities, joint contractures, muscle weakness, and easy fatigability. The onset of the disease is variable, with an average onset of around 13 years and almost always before 30 years of age. The symptoms can worsen with time and result in decreased muscle mass, joint contractures, and facial abnormalities such as frontal bossing and enlarged mandible. In some cases, the orbit involvement can lead to vision problems and hearing loss. Diagnosis of CED is based on clinical and radiographic findings and can be confirmed by genetic testing, which reveals mutations in the TGFB1 gene in over 90% of patients. Currently, there are no universally agreed-upon recommendations for treating CED, and the prognosis is poor, with a significant impact on quality of life due to pain and reduced mobility. To manage the symptoms, treatment options include pain management, physical therapy, orthopedic surgery, and in some cases, bisphosphonate therapy.

• To date, over 300 cases of CED have been reported, with both males and females being affected equally by the condition.

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: 2022-2032

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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