Netherton syndrome (NS) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2019 To 2030

Nethertan syndrome

Netherton syndrome (NS) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2019 To 2030

Indication Overview:

Definition- Netherton syndrome (NS) is a skin disorder characterized by congenital ichthyosiform erythroderma (CIE), a distinctive hair shaft defect (trichorrhexis invaginata; TI) and atopic manifestations.

Etiology-

NS is caused by mutations in the SPINK5 gene (5q31-q32) encoding the serine protease inhibitor LEKTI. LEKTI deficiency results in an increase in trypsin-like hydrolytic activity in the stratum corneum (SC) leading to SC premature desquamation and a severe skin barrier defect.

Epidemiology-

Incidence is estimated at 1/200,000 births.

Geography Covered

  • North America- United States and Canada
  • EU5- Germany, France, Italy, Spain, and the United Kingdom
  • Other EU Countries- Sweden, Denmark, Austria, Finland, Belgium, Netherlands, Switzerland, Poland, Russia
  • Japan
  • APAC- Singapore, China, India, New Zealand, Australia
  • LATAM- Brazil, Mexico, Argentina
  • Middle East (Israel, Saudi Arabia and the United Arab Emirates, Morocco, Egypt, Turkey, Algeria)

Study Period: 2019–2030

Current Clinical Practice and Treatment Algorithm

Country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs are being covered under this section of the study. Retrospective analysis and bench-marking of clinical study outcomes are being presented in terms of Pr-treatment & post-treatment clinical and demographic patient characteristics. Essentially the evolution of the current competitive landscape and its impact on the future treatment paradigm is being well covered under this section.

KOL Insights:

KOLs across 8 MM market from 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 investment throughout the product development process. Rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape: 

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 being tracked and supplemented with analyst commentary.

Clinical Trial Assessment- 

Detailed clinical trial data analysis and key product positioning includes 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 in determining the potential of the key assets as well as 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 important unmet needs identified through our research, include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

S. No Asset Company Partner Stage
1 BPR277 ointment Novartis Pharmaceuticals Novartis Phase 1

KOLs across 8 MM market from 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 4
GERMANY 3
UK 4
SPAIN 2
FRANCE 3
ITALY 3
JAPAN 3
CHINA 3

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.

Netherton Syndrome (NS) forecast

1.       Indication's – 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. Indication's market scenario 2020
1.2.2. Indication's market scenario 2025
1.2.3. Indication's market scenario 2030
2.       Indication's 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 Indication's
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.Indication's 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 Indication's management
2.16.  Market Opportunity for Indication's
2.17. KOL Comments on current and upcoming/expected treatment practices in Indication's
3.       Epidemiology
3.1.    Epidemiology Overview
3.2.    Epidemiology by Geography
3.2.1.  Indication's Epidemiology in US (2019-2030)
3.2.1.1.              Incidence of Indication's
3.2.1.2.              Diagnosed cases
3.2.1.3.              Treatable Patient Pool
3.2.1.4.              Epidemiology Trends
3.2.2.  Indication's Epidemiology in EU-5 (2019-2030)
3.2.2.1.              Incidence of Indication's
3.2.2.2.              Diagnosed cases
3.2.2.3.              Treatable Patient Pool
3.2.2.4.              Epidemiology Trends
3.2.3.  Indication's Epidemiology in Japan (2019-2030)
3.2.3.1.              Incidence of Indication's
3.2.3.2.              Diagnosed cases
3.2.3.3.              Treatable Patient Pool
3.2.3.4.              Epidemiology Trends
3.2.4.  Indication's Epidemiology in China (2019-2030)
3.2.4.1.              Incidence of Indication's
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 Indication's Market Forecast 2019-2030
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 Indication's Market Forecast 2019-2030
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 Indication's Market Forecast 2019-2030
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 Indication's Market Forecast 2019-2030
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 Indication's Market Forecast 2019-2030
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 Indication's Market Forecast 2019-2030
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 Indication's Market Forecast 2019-2030
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 Indication's Market Forecast 2019-2030
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 Indication's
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 Indication's 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 Indication's
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 Indication's 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