Published Date : 2023-12-15
Published Date : 2023-12-15
Updated On : 2024-09-27
Pages : 158
Thelansis’s “Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary hemorrhagic telangiectasia treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
Hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu disease, is a rare genetic disorder that causes abnormal blood vessel formation, leading to the development of arteriovenous malformations (AVMs). These AVMs are abnormal connections between arteries and veins that can form in various organs, including the lungs, liver, and brain. This can result in serious complications such as brain abscesses, stroke, and heart failure. Individuals with HHT often experience frequent and severe nosebleeds, visible blood vessels on the skin and mucous membranes, and gastrointestinal bleeding, which can lead to iron-deficiency anemia. HHT is inherited in an autosomal dominant pattern, with mutations in the ENG (endoglin) and ACVRL1 (ALK1) genes being the primary genetic causes. While there are currently no specific FDA-approved drugs for the treatment of HHT, treatment strategies focus on managing symptoms and complications, such as frequent nosebleeds and pulmonary AVMs.
North America- the United States and Canada
Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)
Other countries- Japan & China
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.
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
- 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
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.
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.
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.
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.
S. no | Asset | Company | Stage |
1 | VAD044 | Vaderis Therapeutics | Phase 1 |
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 |
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.
1. Hereditary Hemorrhagic Telangiectasia (HHT) – 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. Hereditary Hemorrhagic Telangiectasia (HHT) market scenario 2023 |
1.2.2. Hereditary Hemorrhagic Telangiectasia (HHT) market scenario 2028 |
1.2.3. Hereditary Hemorrhagic Telangiectasia (HHT) market scenario 2033 |
2. Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) |
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. Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) management |
2.16. Market Opportunity for Hereditary Hemorrhagic Telangiectasia (HHT) |
2.17. KOL Comments on current and upcoming/expected treatment practices in Hereditary Hemorrhagic Telangiectasia (HHT) |
3. Epidemiology |
3.1. Epidemiology Overview |
3.2. Epidemiology by Geography |
3.2.1. Hereditary Hemorrhagic Telangiectasia (HHT) Epidemiology in US (2023-2033) |
3.2.1.1. Incidence of Hereditary Hemorrhagic Telangiectasia (HHT) |
3.2.1.2. Diagnosed cases |
3.2.1.3. Treatable Patient Pool |
3.2.1.4. Epidemiology Trends |
3.2.2. Hereditary Hemorrhagic Telangiectasia (HHT) Epidemiology in EU-5 (2023-2033) |
3.2.2.1. Incidence of Hereditary Hemorrhagic Telangiectasia (HHT) |
3.2.2.2. Diagnosed cases |
3.2.2.3. Treatable Patient Pool |
3.2.2.4. Epidemiology Trends |
3.2.3. Hereditary Hemorrhagic Telangiectasia (HHT) Epidemiology in Japan (2023-2033) |
3.2.3.1. Incidence of Hereditary Hemorrhagic Telangiectasia (HHT) |
3.2.3.2. Diagnosed cases |
3.2.3.3. Treatable Patient Pool |
3.2.3.4. Epidemiology Trends |
3.2.4. Hereditary Hemorrhagic Telangiectasia (HHT) Epidemiology in China (2023-2033) |
3.2.4.1. Incidence of Hereditary Hemorrhagic Telangiectasia (HHT) |
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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) |
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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 Hereditary Hemorrhagic Telangiectasia (HHT) |
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 Hereditary Hemorrhagic Telangiectasia (HHT) 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 |