Junctional Epidermolysis Bullosa (JEB) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Junctional Epidermolysis Bullosa (JEB) Market Outlook

Thelansis’s “Junctional Epidermolysis Bullosa (JEB) 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 Junctional Epidermolysis Bullosa treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Junctional Epidermolysis Bullosa (JEB) Overview

Junctional epidermolysis bullosa (JEB) is characterized by skin and mucous membrane fragility, resulting in blistering with minimal or no trauma. The blistering can be severe, and granulation tissue may develop around the oral and nasal cavities, fingers and toes, and internally around the upper airway. In contrast to many other conditions, blisters in JEB typically heal without significant scarring. JEB is broadly classified into two categories: generalized severe and generalized intermediate. In JEB, generalized severe blisters are present at birth or appear in the neonatal period, often accompanied by congenital malformations of the urinary tract and bladder. JEB generalized intermediate may present with milder blistering localized to specific areas, such as hands, feet, knees, and elbows, with or without renal or ureteral involvement. Some individuals may not experience blistering after the newborn period. Common features shared with other forms of Epidermolysis bullosa (EB) include congenital localized absence of skin, milia, nail dystrophy, scarring alopecia, hypotrichosis, pseudo syndactyly, and other contractures. Diagnosis of JEB is typically confirmed through molecular genetic testing, identifying biallelic pathogenic variants in genes associated with JEB, such as COL17A1, ITGB4, LAMA3, LAMB3, or LAMC2. Although skin biopsy using transmission electron microscopy (TEM) and immunofluorescent antibody/antigen mapping can be performed, it is no longer the preferred diagnostic method. Patients with JEB often require neonatal intensive care due to severe skin lesions and extracutaneous manifestations. Hospitalization is necessary for monitoring and treating issues such as water-electrolyte balance, failure to thrive, anemia, and infectious and respiratory complications. Pain management, often involving opioids, is crucial. Coordinated care from a multidisciplinary team is essential, including meticulous skin management to prevent blistering through protective padding, avoidance of trauma, lancing and draining new blisters, and prevention of secondary infection through careful wound care.

• The estimated incidence and prevalence of Junctional Epidermolysis Bullosa are 2.68 and 0.49 cases per one million live births, respectively. In the United States, the reported incidence rate is 20 per million births.

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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