Adrenoleukodystrophy - Market Outlook, Market Forecast And Competitve Landscape Report - 2018 To 2030
Thelansis Knowledge Partners 'Adrenoleukodystrophy' - Market Outlook, Market Forecast and Competitive Landscapet report – 2018 to 2030' report comprises of the Disease overview, classifications, stages or severity level, pathogenesis, identified and emerging biomarkers, pipeline assessment, competitive landscape, KOLs perspective, Market access - Country Specific Regulatory scenario
s, Patent landscape, reimbursement scenario; Public, Private, Medicare, Medicaid, Government, Private, Co-payment, Out of pocket, Epidemiology; Incidence, Prevalence, Mortality, and current therapy market share, Order of entry, patient share, Market uptake, Peak patient share, Disease burden, Annual cost of therapy, market forecast for USA, Germany, France, Italy, Spain, UK, Japan and China 2018 to 2030
Geographic Coverage
Report brief:
Epidemiology
This section provide the insights about historical and current patient pool (Incidence, Prevalence and mortality) and forecasted trend for 8 major markets (US, Germany, France, Italy, Spain, UK, Japan and China). The epidemiology research is one the key element of the report driven by meta-analysis, Systemic literature review and insights from KOL’s
Treatment Algorithm
The comprehensive details on country specific treatment and disease management for the patients in the US, Europe, Japan, and China. Future treatment paradigm and KOL’s perspective (HCP’s, Payers and Researcher) are the key strength of the report
Competitive Landscapet
A thorough analysis of reported revenue, patient share and marketed trend for approved therapies however for pipeline assets we consider Phase I, Phase II and Phase III including their major milestones, study result, interim result, safety & efficacy data, key drivers, mechanism of action designation (Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review) geography level prioritization, asset level profile and probable launch date estimation
Market Outlook & Market Forecast
Historic, current and forecasted market trend (High, Base case, Low) of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better diagnosis of biomarkers. Key patient segment and market based on type, severity and stages of the disease. Disease burden, compliance rate, annual cost of therapy, reimbursement scenario, Patient share, Patient access
Market uptake and benchmarking
The market penetration for pipeline assets being derived on the basis of benchmarking the sales of launched assets specifically targeting the patient segment or overall indication or therapy area or country or region specific. Order of entry benchmark works as a critical element for defining the uptake curve for the pipeline assets. Many drug utilization study are being benchmarked to arrive at the current patient share for marketed therapies as well as off-label products. Market events are the major key factors which applied to the forecast model to optimize the year on year revenue estimation
Regulatory scenario
Country specific healthcare structure, regulatory bodies, HTA bodies, approval process for diagnostic and therapies, and work flow
Patent landscape
Country specific patent filings for approved therapies as well as pipeline assets, formulation patent, process patent (FDA, EMA, EPO, Orange book, JPO; Japan Patent Office) and fillings
Reimbursement Scenario
Country specific reimbursement Scenario; Public, Private, Medicare, Medicaid, Government, Private, Co-payment, Out of pocket
Key Report highlights
s, Patent landscape, reimbursement scenario; Public, Private, Medicare, Medicaid, Government, Private, Co-payment, Out of pocket, Epidemiology; Incidence, Prevalence, Mortality, and current therapy market share, Order of entry, patient share, Market uptake, Peak patient share, Disease burden, Annual cost of therapy, market forecast for USA, Germany, France, Italy, Spain, UK, Japan and China 2018 to 2030
Geographic Coverage
- United States
- EU5 (Germany, France, Italy, Spain and the United Kingdom)
- Japan
- China
Report brief:
Epidemiology
This section provide the insights about historical and current patient pool (Incidence, Prevalence and mortality) and forecasted trend for 8 major markets (US, Germany, France, Italy, Spain, UK, Japan and China). The epidemiology research is one the key element of the report driven by meta-analysis, Systemic literature review and insights from KOL’s
Treatment Algorithm
The comprehensive details on country specific treatment and disease management for the patients in the US, Europe, Japan, and China. Future treatment paradigm and KOL’s perspective (HCP’s, Payers and Researcher) are the key strength of the report
Competitive Landscapet
A thorough analysis of reported revenue, patient share and marketed trend for approved therapies however for pipeline assets we consider Phase I, Phase II and Phase III including their major milestones, study result, interim result, safety & efficacy data, key drivers, mechanism of action designation (Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review) geography level prioritization, asset level profile and probable launch date estimation
Market Outlook & Market Forecast
Historic, current and forecasted market trend (High, Base case, Low) of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better diagnosis of biomarkers. Key patient segment and market based on type, severity and stages of the disease. Disease burden, compliance rate, annual cost of therapy, reimbursement scenario, Patient share, Patient access
Market uptake and benchmarking
The market penetration for pipeline assets being derived on the basis of benchmarking the sales of launched assets specifically targeting the patient segment or overall indication or therapy area or country or region specific. Order of entry benchmark works as a critical element for defining the uptake curve for the pipeline assets. Many drug utilization study are being benchmarked to arrive at the current patient share for marketed therapies as well as off-label products. Market events are the major key factors which applied to the forecast model to optimize the year on year revenue estimation
Regulatory scenario
Country specific healthcare structure, regulatory bodies, HTA bodies, approval process for diagnostic and therapies, and work flow
Patent landscape
Country specific patent filings for approved therapies as well as pipeline assets, formulation patent, process patent (FDA, EMA, EPO, Orange book, JPO; Japan Patent Office) and fillings
Reimbursement Scenario
Country specific reimbursement Scenario; Public, Private, Medicare, Medicaid, Government, Private, Co-payment, Out of pocket
Key Report highlights
- 4 years of historical epidemiology data Epidemiology; Incidence, Prevalence, Mortality
- 11 years of epidemiology and market projections
- Incidence/ Prevalence patient population
- Mortality, survival and diagnosed patient pool
- Treated patient pool
- Treatment guidelines
- Competitive landscape (Marketed therapies, Off-label therapies, Pipeline assessment, Phase – I, II, III)
- Patient share
- Country specific revenue share and trend
- Market Opportunities assessment
- Key market and product event analysis
- Disease burden and annual cost of therapy
- Regulatory scenario
- Reimbursement Scenario
- Patent landscape
- HCP’s/ KOL’s perspective
- Payer’s perspective
- Analyst commentary Bluebird Bio, Medday Pharmaceuticals, Minoryx Therapeutics, Orpheris, Inc, Magenta Therapeutics
1. DISEASE – KEY FINDINGS SUMMARY
1. Clinical findings
1. Disease overview
2. Therapeutic practices
3. Future outlook
2. Commercial findings
1. Disease market scenario 2020
2. Disease market scenario 2025
3. Disease market scenario 2030
2. INDICATION - DISEASE OVERVIEW
1. Disease introduction
2. Classification
3. Genetic Subtypes and subtypes
4. Signs and Symptoms
5. Risk Groups
6. Histology
7. Etiology
8. Pathogenesis
9. Diagnosis
1. Biomarker testing
10. Treatment Algorithm
1. Treatment in US (guidelines and real-world management)
2. Treatment in EU-5 (guidelines and real-world management)
3. Treatment in Japan (guidelines and real-world management)
4. Treatment in China (guidelines and real-world management)
11. Current Treatment and Management Goals for Disease
12. Referral Patterns
1. Referral Scenario in US
2. Referral Scenario in EU-5
3. Referral Scenario in Japan
4. Referral Scenario in China
13. Disease Prognosis
14. Disease Progression Pattern
15. Disease Biomarkers – Current and Future Paradigm
16. Disease Observing Scores
17. Healthcare burden
1. Healthcare burden in US
2. Healthcare burden in EU-5
3. Healthcare burden in Japan
4. Healthcare burden in China
18. Unmet Needs
3. EPIDEMIOLOGY
1. Epidemiology Overview
2. Epidemiology by Geography
1. Disease Incidence/ Prevalence in US
2. Disease Incidence/ Prevalence in EU-5
3. Disease Incidence/ Prevalence in Japan
4. Disease Incidence/ Prevalence in China
3. Epidemiology by Type and subtype
4. Epidemiology by stages
5. Epidemiology Trends
4. MARKET OUTLOOK
1. US - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
2. UK - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
3. France - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
4. Germany - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
5. Italy - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
6. Spain - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
7. Japan - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
8. China - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
9. Key Expected Milestones Impacting Market
5. COMPETITIVE LANDSCAPE
1. Pipeline Therapies Overview
1. Phase III Therapies
1. Assets - Company
1. Current Status
2. Trials
3. Approval Timeline
4. Expected Product Positioning
5. Market Forecast (Till 2030)
6. Attribute analysis - Phase III Therapies
2. Phase II Therapies
1. Assets - Company
1. Current Status
2. Trials
3. Approval Timeline
4. Expected Product Positioning
5. Market Forecast (Till 2030)
6. Attribute analysis - Phase III Therapies)
2. Assets - Company
1. Current Status
2. Trials
3. Approval Timeline
4. Expected Product Positioning
5. Market Forecast (Till 2030)
6. Attribute analysis - Phase III Therapies
3. Phase I Therapies
1. Assets - Company
1. Current Status
2. Trials
3. Approval Timeline
4. Expected Product Positioning
5. Market Forecast (Till 2030)
6. Attribute analysis
4. Pre-clinical Therapies
1. Asset - Company
1. Current Status
2. Pre-clinical Trials
3. Estimated Timelines
2. Asset - Company
1. Current Status
2. Pre-clinical Trials
3. Estimated Timelines
3. Asset - Company
1. Current Status
2. Pre-clinical Trials
3. Estimated Timelines
5. Inactive Therapies
1. Asset - Company
1. Current Status
2. Reasons for Inactivation
2. Asset - Company
1. Current Status
2. Reasons for Inactivation
3. Asset - Company
1. Current Status
2. Reasons for Inactivation
6. REGULATORY FRAMEWORK SCENARIO
1. Regulatory Framework in US
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
2. Regulatory Framework in UK
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
3. Regulatory Framework in France
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
4. Regulatory Framework in Germany
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
5. Regulatory Framework in Italy
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
6. Regulatory Framework in Spain
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
7. Regulatory Framework in Japan
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
8. Regulatory Framework in China
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
7. CLINICAL TRIAL ASSESSMENT – CURRENT AND FUTURE PARADIGM
1. Distribution of Primary Endpoints across trials
2. Distribution of Secondary Endpoints across trials
3. Evolution and acceptance of surrogate endpoints
4. Key Investigator initiated trials
5. Attrition analysis
1. Failed Trials, Reasons and Business Impact
2. Terminated Trials, Reasons and Business Impact
3. Withdrawn Trials, Reasons and Business Impact
6. Trial enrollment scenario and challenges
7. Clinical Trial Guidance (across geographies)
8. THELANSIS’ COMMENTARY
1. Key Unmet needs in Indication-Disease
2. Possible Best-case Clinical Trial Strategies
3. Possible Best-case Market positioning strategies
4. Possible Best-case Market Access Strategies
5. Overall View on Indication-Disease Market in Dollar Value
9. REPORT METHODOLOGY
1. Secondary research
2. Primary research
3. Data collation
4. Insight Generation
10. ABOUT THELANSIS
1. Our Capabilities
2. Our Services
3. Our Contacts
4. Disclaimer
1. Clinical findings
1. Disease overview
2. Therapeutic practices
3. Future outlook
2. Commercial findings
1. Disease market scenario 2020
2. Disease market scenario 2025
3. Disease market scenario 2030
2. INDICATION - DISEASE OVERVIEW
1. Disease introduction
2. Classification
3. Genetic Subtypes and subtypes
4. Signs and Symptoms
5. Risk Groups
6. Histology
7. Etiology
8. Pathogenesis
9. Diagnosis
1. Biomarker testing
10. Treatment Algorithm
1. Treatment in US (guidelines and real-world management)
2. Treatment in EU-5 (guidelines and real-world management)
3. Treatment in Japan (guidelines and real-world management)
4. Treatment in China (guidelines and real-world management)
11. Current Treatment and Management Goals for Disease
12. Referral Patterns
1. Referral Scenario in US
2. Referral Scenario in EU-5
3. Referral Scenario in Japan
4. Referral Scenario in China
13. Disease Prognosis
14. Disease Progression Pattern
15. Disease Biomarkers – Current and Future Paradigm
16. Disease Observing Scores
17. Healthcare burden
1. Healthcare burden in US
2. Healthcare burden in EU-5
3. Healthcare burden in Japan
4. Healthcare burden in China
18. Unmet Needs
3. EPIDEMIOLOGY
1. Epidemiology Overview
2. Epidemiology by Geography
1. Disease Incidence/ Prevalence in US
2. Disease Incidence/ Prevalence in EU-5
3. Disease Incidence/ Prevalence in Japan
4. Disease Incidence/ Prevalence in China
3. Epidemiology by Type and subtype
4. Epidemiology by stages
5. Epidemiology Trends
4. MARKET OUTLOOK
1. US - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
2. UK - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
3. France - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
4. Germany - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
5. Italy - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
6. Spain - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
7. Japan - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
8. China - Disease Market Forecast
1. Market Evolution (Historic)
2. Market Progression (Futuristic)
3. Market Trends and Expectations
1. Worst case scenario
2. Base Case Scenario
3. Best Case Scenario
4. Drivers and Barriers
9. Key Expected Milestones Impacting Market
5. COMPETITIVE LANDSCAPE
1. Pipeline Therapies Overview
1. Phase III Therapies
1. Assets - Company
1. Current Status
2. Trials
3. Approval Timeline
4. Expected Product Positioning
5. Market Forecast (Till 2030)
6. Attribute analysis - Phase III Therapies
2. Phase II Therapies
1. Assets - Company
1. Current Status
2. Trials
3. Approval Timeline
4. Expected Product Positioning
5. Market Forecast (Till 2030)
6. Attribute analysis - Phase III Therapies)
2. Assets - Company
1. Current Status
2. Trials
3. Approval Timeline
4. Expected Product Positioning
5. Market Forecast (Till 2030)
6. Attribute analysis - Phase III Therapies
3. Phase I Therapies
1. Assets - Company
1. Current Status
2. Trials
3. Approval Timeline
4. Expected Product Positioning
5. Market Forecast (Till 2030)
6. Attribute analysis
4. Pre-clinical Therapies
1. Asset - Company
1. Current Status
2. Pre-clinical Trials
3. Estimated Timelines
2. Asset - Company
1. Current Status
2. Pre-clinical Trials
3. Estimated Timelines
3. Asset - Company
1. Current Status
2. Pre-clinical Trials
3. Estimated Timelines
5. Inactive Therapies
1. Asset - Company
1. Current Status
2. Reasons for Inactivation
2. Asset - Company
1. Current Status
2. Reasons for Inactivation
3. Asset - Company
1. Current Status
2. Reasons for Inactivation
6. REGULATORY FRAMEWORK SCENARIO
1. Regulatory Framework in US
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
2. Regulatory Framework in UK
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
3. Regulatory Framework in France
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
4. Regulatory Framework in Germany
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
5. Regulatory Framework in Italy
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
6. Regulatory Framework in Spain
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
7. Regulatory Framework in Japan
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
8. Regulatory Framework in China
1. Approval Framework(s)
2. Clinical Trial Guidance
3. Macro Policies (if any)
4. Expectations on Future Pricing and Reimbursement Outlook
7. CLINICAL TRIAL ASSESSMENT – CURRENT AND FUTURE PARADIGM
1. Distribution of Primary Endpoints across trials
2. Distribution of Secondary Endpoints across trials
3. Evolution and acceptance of surrogate endpoints
4. Key Investigator initiated trials
5. Attrition analysis
1. Failed Trials, Reasons and Business Impact
2. Terminated Trials, Reasons and Business Impact
3. Withdrawn Trials, Reasons and Business Impact
6. Trial enrollment scenario and challenges
7. Clinical Trial Guidance (across geographies)
8. THELANSIS’ COMMENTARY
1. Key Unmet needs in Indication-Disease
2. Possible Best-case Clinical Trial Strategies
3. Possible Best-case Market positioning strategies
4. Possible Best-case Market Access Strategies
5. Overall View on Indication-Disease Market in Dollar Value
9. REPORT METHODOLOGY
1. Secondary research
2. Primary research
3. Data collation
4. Insight Generation
10. ABOUT THELANSIS
1. Our Capabilities
2. Our Services
3. Our Contacts
4. Disclaimer
