Published Date : 2023-04-17
Published Date : 2023-04-17
Updated On : 2024-01-31
Pages : 159
Thelansis’s “Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
Pulmonary arterial hypertension (PAH) is a progressive and chronic condition marked by antiproliferative changes in the pulmonary arterioles, resulting in endothelial and smooth muscle proliferation and dysfunction, inflammation, and the formation of blood clots. PAH is primarily caused by remodeling pulmonary arteries, narrowing and obstructing the smallest arteries. This increased resistance in the pulmonary circulation results in elevated pressure within the right ventricle. Specific gene mutations, including BMPR2 (2q33), ACVRL1 (12q13), Endoglin (9q34), CAV1 (7q31), KCNK3 (2p23), Smad9 (13q12), and TBX4 (17q21), have been associated with heritable PAH. Most cases of PAH typically develop in adults, with occasional occurrences in children. Women are twice as likely as men to be affected by this condition. Initial symptoms include shortness of breath, fainting, chest discomfort, palpitations, and swelling in the lower extremities. Clinical signs include a prominent and palpable second heart sound, a pulmonary ejection click, and murmurs related to pulmonary and tricuspid regurgitation—approximately 70% of patients present with severe heart failure. Less frequently, symptoms such as abdominal swelling, clubbing of the fingers, and Raynaud's phenomenon (predominantly in females) may also manifest. Hemoptysis has been reported but is relatively rare. Treatment strategies encompass general measures, such as aggressive management of respiratory tract infections, annual influenza vaccinations, family planning, and medical therapies like oxygen supplementation, anticoagulation, diuretics, and digoxin. Calcium channel blockers are appropriate for patients with acute vasodilator response to nitric oxide during right heart catheterization. Additional therapeutic options include endothelin receptor agonists like ambrisentan, prostanoids like epoprostenol, and phosphodiesterase inhibitors like sildenafil. Lung transplantation remains the last resort for patients who do not respond to other treatment modalities. The prognosis varies depending on the specific form of PAH, but it is generally grim, with a mean survival rate of less than three years following diagnosis. However, early intervention with appropriate therapies can improve survival rates.
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 | CS1 | Cereno Scientific AB | Phase 2 |
2 | AV-101 | Aerovate Therapeutics | Phase 3 |
3 | Treprostinil Palmitil | Insmed Incorporated | Phase 2 |
4 | KER-012 | Keros Therapeutics, Inc. | Phase 2 |
5 | L608 | Pharmosa Biopharm Inc. | Phase 1 |
6 | Tyvaso® (treprostinil) | United Therapeutics | Phase 3 |
7 | Seralutinib | Gossamer Bio Inc. | Phase 2 |
8 | GMA301 | Gmax Biopharm LLC. | Phase 1 |
9 | Ubenimex | Eiger BioPharmaceuticals | Phase 2 |
10 | L606 | Liquidia Technologies, Inc. | Phase 3 |
Continued...
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. Pulmonary Arterial Hypertension (PAH) – 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. Pulmonary Arterial Hypertension (PAH) market scenario 2023 |
1.2.2. Pulmonary Arterial Hypertension (PAH) market scenario 2028 |
1.2.3. Pulmonary Arterial Hypertension (PAH) market scenario 2033 |
2. Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) |
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. Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) management |
2.16. Market Opportunity for Pulmonary Arterial Hypertension (PAH) |
2.17. KOL Comments on current and upcoming/expected treatment practices in Pulmonary Arterial Hypertension (PAH) |
3. Epidemiology |
3.1. Epidemiology Overview |
3.2. Epidemiology by Geography |
3.2.1. Pulmonary Arterial Hypertension (PAH) Epidemiology in US (2023-2033) |
3.2.1.1. Incidence of Pulmonary Arterial Hypertension (PAH) |
3.2.1.2. Diagnosed cases |
3.2.1.3. Treatable Patient Pool |
3.2.1.4. Epidemiology Trends |
3.2.2. Pulmonary Arterial Hypertension (PAH) Epidemiology in EU-5 (2023-2033) |
3.2.2.1. Incidence of Pulmonary Arterial Hypertension (PAH) |
3.2.2.2. Diagnosed cases |
3.2.2.3. Treatable Patient Pool |
3.2.2.4. Epidemiology Trends |
3.2.3. Pulmonary Arterial Hypertension (PAH) Epidemiology in Japan (2023-2033) |
3.2.3.1. Incidence of Pulmonary Arterial Hypertension (PAH) |
3.2.3.2. Diagnosed cases |
3.2.3.3. Treatable Patient Pool |
3.2.3.4. Epidemiology Trends |
3.2.4. Pulmonary Arterial Hypertension (PAH) Epidemiology in China (2023-2033) |
3.2.4.1. Incidence of Pulmonary Arterial Hypertension (PAH) |
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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) |
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 Pulmonary Arterial Hypertension (PAH) 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 Pulmonary Arterial Hypertension (PAH) |
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 Pulmonary Arterial Hypertension (PAH) 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 |