Recessive Dystrophic Epidermolysis Bullosa (RDEB) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Outlook and Forecast

Recessive Dystrophic Epidermolysis Bullosa (RDEB) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Published Date : 2023-12-14

Updated On : 2024-09-27

Pages : 152

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Outlook

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

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Overview

Recessive dystrophic epidermolysis bullosa (RDEB) is a subgroup of hereditary blistering diseases known for their recurring injuries and healing processes, often leading to extensive scarring. These are part of a broader category of genetic skin disorders characterized by high susceptibility to blister formation and erosion triggered by minor injuries. There are more than 12 hereditary variations of epidermolysis bullosa (EB), grouped into four primary forms: dominant EB simplex, recessive junctional EB, dominant dystrophic EB, and recessive dystrophic EB. Dystrophic forms of EB entail repetitive skin injuries and subsequent healing, resulting in severe scarring. Notably, individuals with RDEB are prone to developing aggressive squamous cell carcinomas in regions with severe scarring, leading to significant health challenges and a shortened lifespan. The underlying mechanisms of Recessive dystrophic epidermolysis bullosa (RDEB) remain unclear, but researchers have explored two main hypotheses. One hypothesis posits that RDEB-affected skin has fewer anchoring fibrils (AF), contributing to its fragility.

  • According to data from the National EB Registry, the prevalence of recessive dystrophic epidermolysis bullosa (RDEB) is 1.35 cases per one million live 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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Recessive Dystrophic Epidermolysis Bullosa (RDEB) Competitive Landscape

S. no Asset Company Stage
1 Prademagene zamikeracel (pz-cel) Abeona Therapeutics, Inc Phase 3
2 PTR-01 Phoenix Tissue Repair, Inc. Phase 2
3 FCX-007 Castle Creek Biosciences, LLC. Phase 3
4 Rigosertib Sodium Traws Pharma, Inc. Phase 1
5 allo-APZ2-EB RHEACELL GmbH & Co. KG Phase 1/2

KOLs across 8 MM market 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.

COUNTRY No. Of KOLs
USA 17
GERMANY 4
UK 4
SPAIN 3
FRANCE 2
ITALY 3
JAPAN 3
CHINA 4

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

Data Inputs with sourcing, Market Event, Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast

1.       Recessive Dystrophic Epidermolysis Bullosa (RDEB) – Key Findings Summary

         1.1.    Clinical findings
                     1.1.1. Disease overview
                     1.1.2. Therapeutic practices
                     1.1.3. Future outlook
         1.2.    Commercial findings
                     1.2.1. Recessive Dystrophic Epidermolysis Bullosa (RDEB) market scenario 2023
                     1.2.2. Recessive Dystrophic Epidermolysis Bullosa (RDEB) market scenario 2028
                     1.2.3. Recessive Dystrophic Epidermolysis Bullosa (RDEB) market scenario 2033

2.       Recessive Dystrophic Epidermolysis Bullosa (RDEB) Overview

         2.1.    Disease Introduction
         2.2.    Pathophysiology
         2.3.    Signs and Symptoms
         2.4.    Risk Factors
         2.5.    Etiology
         2.6.    Classification
         2.7.    Pathogenesis
         2.8.    Diagnosis
         2.9.    Complications
         2.10. Treatment Algorithm
                     2.10.1.    Treatment in US (guidelines)
                     2.10.2.    Treatment in EU-5 (guidelines)
                     2.10.3.    Treatment in Japan (guidelines)
                     2.10.4.    Treatment in China (guidelines)
         2.11. Treatment Goals for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
         2.12. Referral Patterns
                     2.12.1.    Referral Scenario in US
                     2.12.2.    Referral Scenario in EU-5
                     2.12.3.    Referral Scenario in Japan
                     2.12.4.    Referral Scenario in China
         2.13. Recessive Dystrophic Epidermolysis Bullosa (RDEB) Prognosis
         2.14. Healthcare burden
                     2.14.1.    Healthcare burden in US
                     2.14.2.    Healthcare burden in EU-5
                     2.14.3.    Healthcare burden in Japan
                     2.14.4.    Healthcare burden in China
         2.15.  Unmet Needs in Recessive Dystrophic Epidermolysis Bullosa (RDEB) management
         2.16.  Market Opportunity for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
         2.17. KOL Comments on current and upcoming/expected treatment practices in Recessive Dystrophic Epidermolysis Bullosa (RDEB)

3.       Epidemiology

         3.1.    Epidemiology Overview
         3.2.    Epidemiology by Geography
                     3.2.1.  Recessive Dystrophic Epidermolysis Bullosa (RDEB) Epidemiology in US (2023-2033)
                             3.2.1.1.              Incidence of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
                             3.2.1.2.              Diagnosed cases
                             3.2.1.3.              Treatable Patient Pool
                             3.2.1.4.              Epidemiology Trends
                     3.2.2.  Recessive Dystrophic Epidermolysis Bullosa (RDEB) Epidemiology in EU-5 (2023-2033)
                             3.2.2.1.              Incidence of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
                             3.2.2.2.              Diagnosed cases
                             3.2.2.3.              Treatable Patient Pool
                             3.2.2.4.              Epidemiology Trends
                     3.2.3.  Recessive Dystrophic Epidermolysis Bullosa (RDEB) Epidemiology in Japan (2023-2033)
                             3.2.3.1.              Incidence of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
                             3.2.3.2.              Diagnosed cases
                             3.2.3.3.              Treatable Patient Pool
                             3.2.3.4.              Epidemiology Trends
                     3.2.4.  Recessive Dystrophic Epidermolysis Bullosa (RDEB) Epidemiology in China (2023-2033)
                             3.2.4.1.              Incidence of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
                             3.2.4.2.              Diagnosed cases
                             3.2.4.3.              Treatable Patient Pool
                             3.2.4.4.              Epidemiology Trends
         3.3.    Epidemiology Trends (World-wide)

4.       Market Outlook

         4.1.    US Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast 2023-2033
                     4.1.1. Market Progression (Futuristic)
                     4.1.2. Market Trends and Expectations
                             4.1.2.1.              Worst case scenario
                             4.1.2.2.              Base Case Scenario
                             4.1.2.3.              Best Case Scenario
                     4.1.3. Drivers and Barriers
         4.2.    UK Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast 2023-2033
                     4.2.1. Market Progression (Futuristic)
                     4.2.2. Market Trends and Expectations
                             4.2.2.1.              Worst case scenario
                             4.2.2.2.              Base Case Scenario
                             4.2.2.3.              Best Case Scenario
                     4.2.3. Drivers and Barriers
         4.3.    France Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast 2023-2033
                     4.3.1. Market Progression (Futuristic)
                     4.3.2. Market Trends and Expectations
                             4.3.2.1.              Worst case scenario
                             4.3.2.2.              Base Case Scenario
                             4.3.2.3.              Best Case Scenario
                     4.3.3. Drivers and Barriers
         4.4.    Germany Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast 2023-2033
                     4.4.1. Market Progression (Futuristic)
                     4.4.2. Market Trends and Expectations
                             4.4.2.1.              Worst case scenario
                             4.4.2.2.              Base Case Scenario
                             4.4.2.3.              Best Case Scenario
                     4.4.3. Drivers and Barriers
         4.5.    Italy Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast 2023-2033
                     4.5.1. Market Progression (Futuristic)
                     4.5.2. Market Trends and Expectations
                             4.5.2.1.              Worst case scenario
                             4.5.2.2.              Base Case Scenario
                             4.5.2.3.              Best Case Scenario
                     4.5.3. Drivers and Barriers
         4.6.    Spain Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast 2023-2033
                     4.6.1. Market Progression (Futuristic)
                     4.6.2. Market Trends and Expectations
                             4.6.2.1.              Worst case scenario
                             4.6.2.2.              Base Case Scenario
                             4.6.2.3.              Best Case Scenario
                     4.6.3. Drivers and Barriers
         4.7.    Japan Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast 2023-2033
                     4.7.1. Market Progression (Futuristic)
                     4.7.2. Market Trends and Expectations
                             4.7.2.1.              Worst case scenario
                             4.7.2.2.              Base Case Scenario
                             4.7.2.3.              Best Case Scenario
                     4.7.3. Drivers and Barriers
         4.8.    China Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market Forecast 2023-2033
                     4.8.1. Market Progression (Futuristic)
                     4.8.2. Market Trends and Expectations
                             4.8.2.1.              Worst case scenario
                             4.8.2.2.              Base Case Scenario
                             4.8.2.3.              Best Case Scenario
                     4.8.3. Drivers and Barriers
         4.9.    Key Expected Milestones (world-wide) Impacting the Market

5.       Competitive Landscape

         5.1.    Pipeline Therapies Overview
                     5.1.1. Phase III Therapies
                                      5.1.1.1.                     Current Status
                                      5.1.1.2.                     Trial details, results
                                      5.1.1.3.                     Approval Timeline
                                      5.1.1.4.                     Likelihood of approval
                                      5.1.1.5.                     Expected Product Positioning
                             5.1.1.2.              All other Phase III Therapies …..
                             5.1.1.3.              Attribute Analysis of Phase III molecules
                     5.1.2.  Phase II and Phase I/II Therapies
                                      5.1.2.1.                     Current Status
                                      5.1.2.2.                     Trial details, results
                                      5.1.2.3.                     Approval Timelines
                     5.1.3. List of active Pre-clinical Therapies
                                      5.1.3.1.                     Status in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
                                      5.1.3.2.                     Company positioning
                             5.1.3.2.              All other pre-clinical therapies
                     5.1.4. List of Inactive/discontinued assets
                             5.1.4.1.              Business impact of discontinuations on current pipeline
                     5.1.5. Potential winners from Recessive Dystrophic Epidermolysis Bullosa (RDEB) Pipeline
                             5.1.5.1.              Potential Blockbusters across the pipeline

6.       Regulatory/Approval Scenario

         6.1.    Regulatory/Approval Framework in US
                     6.1.1. Policy Framework
                     6.1.2. Payer Expectations
         6.2.    Regulatory/Approval Framework in UK
                     6.2.1. Policy Framework
                     6.2.2. Payer Expectations
         6.3.    Regulatory/Approval Framework in France
                     6.3.1. Policy Framework
                     6.3.2. Payer Expectations
         6.4.    Regulatory/Approval Framework in Germany
                     6.4.1. Policy Framework
                     6.4.2. Payer Expectations
         6.5.    Regulatory/Approval Framework in Italy
                     6.5.1. Policy Framework
                     6.5.2. Payer Expectations
         6.6.    Regulatory/Approval Framework in Spain
                     6.6.1. Policy Framework
                     6.6.2. Payer Expectations
         6.7.       Regulatory/Approval Framework in Japan
                     6.7.1. Policy Framework
                     6.7.2. Payer Expectations
         6.8.       Regulatory/Approval Framework in China
                     6.8.1. Policy Framework
                     6.8.2. Payer Expectations

7.       Clinical Trial Assessment – Current and Future Paradigm

         7.1.    Distribution of Primary Endpoints across trials
         7.2.    Distribution of Secondary Endpoints across trials
         7.3.    Evolution and acceptance of surrogate endpoints
         7.4.    Key Investigator initiated trials
         7.5.    Attrition analysis
                     7.5.1. Suspended/Discontinued Assets
                     7.5.2. Failed Trials, Reasons and Business Impact
                     7.5.3. Terminated Trials, Reasons and Business Impact
                     7.5.4. Withdrawn Trials, Reasons and Business Impact
         7.6.    Trial enrollment scenario and challenges
         7.7.    Clinical Trial Guidance (across geographies)

8.       Thelansis Commentary

         8.1.    Key Unmet needs in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
         8.2.    Possible Best-case Clinical Trial Strategies
         8.3.    Possible Best Case Targeted Product Profile (TPP)
         8.4.    Possible Best-case Market positioning strategies
         8.5.    Possible Best-case Market Access Strategies
         8.6.    Possible Best-case LCM Strategies
         8.7.    Overall View on Recessive Dystrophic Epidermolysis Bullosa (RDEB) Market in Dollar Value

9.       Report Methodology

         9.1.    Secondary research
         9.2.    Primary research
         9.3.    Data collation
         9.4.    Insight Generation

10.   About Thelansis

         10.1.    Our Capabilities
         10.2.    Our Services
         10.3.    Our Contacts
         10.4.    Disclaimer