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市場調查報告書

OpportunityAnalyzer:高雪氏症 - 機會分析與預測

OpportunityAnalyzer: Gaucher Disease - Opportunity Analysis and Forecast to 2024

出版商 GlobalData 商品編碼 353074
出版日期 內容資訊 英文 244 Pages
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OpportunityAnalyzer:高雪氏症 - 機會分析與預測 OpportunityAnalyzer: Gaucher Disease - Opportunity Analysis and Forecast to 2024
出版日期: 2015年12月01日 內容資訊: 英文 244 Pages
簡介

主要7個國家(美國、法國、德國、義大利、西班牙、英國、以色列) 的高雪氏症的治療藥市場,從2014年的8億7,500萬美元,預計2024年擴大到11億6,000萬美元規模,以2.9%的年複合成長率 (CAGR) 增加。

本報告提供高雪氏症的治療藥市場相關調查分析,疾病概要,流行病學,目前治療選項,未滿足需求和機會,研究開發策略,開發平台評估等相關的系統性資訊。

第1章 目錄

第2章 簡介

第3章 疾病概要

  • 病因、病理生理學
  • 症狀
  • 預後
  • QOL

第4章 流行病學

  • 疾病的背景
  • 危險因素和合併症
  • 全球趨勢
    • 美國
    • EU5個國家
    • 以色列
  • 預測手法
    • 利用之資訊來源
    • 未利用之資訊來源
    • 預測的前提條件與手法
  • 高雪氏症的流行病學預測
    • I型和確診的患者數
    • I型和確診的患者數 (各年齡)
    • I型和確診的患者數 (性別)
    • I型和確診的患者數 (年齡標準化) 、等
  • 討論
    • 流行病學預測的考察
    • 分析的限制
    • 分析的優勢

第5章 目前治療選項

  • 概要
  • 產品簡介

第6章 未滿足需求評估與機會分析

  • 概要
  • 高雪氏症的神經學的症狀的治療
  • 高雪氏症的早期診斷與疾病的重要度的評估
  • 高雪氏症治療的低價格化
  • 給藥途徑的改善

第7章 研究開發 (R&D) 策略

  • 概要
  • 臨床試驗設計

第8章 開發平台評估

  • 臨床開發中的有前途藥物
  • 創新的初期階段的方法

第9章 開發平台評估分析

  • 主要藥臨床基準
  • 主要藥的商業基準
  • 競爭評估
  • 銷售額的10年預測
    • 美國
    • EU5個國家
    • 以色列
    • 推動因素、障礙

第10章 附錄

圖表

目錄
Product Code: GDHC047POA

GlobalData estimated the 2014 sales for the Gaucher Disease (GD) market at approximately $875m across the 7MM (US, France, Germany, Italy, Spain, UK, and Israel). The US market contributed the majority of these sales, generating an estimated $482m. By the end of the forecast period in 2024, GD sales are expected to increase to $1.16 billion at a Compound Annual Growth Rate (CAGR) of 2.9%. The majority of these sales will continue to come from the US, which will represent approximately 56.2% of the GD market in the 7MM in 2024. GlobalData forecasts an increase in the number of diagnosed prevalent cases of GD, and therefore, an increase in the treated GD population. Improved diagnosis will be driven by increasing physician education and awareness of GD and its signs and symptoms. Despite the anticipated increase in the number of diagnosed prevalent cases of GD over the forecast period, it is expected that a large portion of the GD population will remain undiagnosed. This is due to continued late diagnosis and misdiagnosis of GD as a result of the heterogeneous manifestations of the disease. The sparsely populated GD pipeline, with no drugs in late-stage development, will further hinder the growth of the GD market.

Highlights

Key Questions Answered

  • Although the use of enzyme replacement therapies (ERTs) and substrate reduction therapies (SRTs) has significantly changed the natural history of GD, with a marked decrease in morbidity, especially in Type 1 GD patients, GD market is still marked by the presence of a number of unmet needs in current treatments. What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs of the GD market?
  • The late-stage GD pipeline is very sparsely populated and there no drugs in late-stage development. Nevertheless, the GD market will experience substantial growth. What are the main drivers of this growth during 2014-2024? Which of the marketed drugs will have the highest peak sales at the highest CAGR, and why?

Key Findings

  • GlobalData forecasts an increase in the number of diagnosed prevalent cases of GD, and therefore, an increase in the treated GD population. Improved diagnosis will be driven by increasing physician education and awareness of GD and its signs and symptoms. This is largely attributable to Genzyme's and Shire's continued efforts to raise awareness of rare diseases through organized events and established websites, and by sponsoring patient registries.
  • Genzyme's recently approved oral substrate reduction therapy (SRT), Cerdelga (eliglustat), is more effective and safer than the other oral SRT, Zavesca (miglustat), and therefore will be used more commonly as an oral alternative to IV ERT in adult Type 1 GD patients. As Cerdelga is more expensive than the other GD therapies, it is expected to drive the growth of the GD market.
  • None of the therapies fulfilled the greatest unmet need in GD treatment - need for the drugs that can treat the neurological manifestations of the disease. One of the main R&D strategies in the GD space is the focus on the development of new SRT formulations and pharmacological chaperones (PCs) that can cross the blood brain barrier and thereby improve the neurological manifestations of GD.

Scope

  • Overview of GD, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Annualized GD therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2014 and forecast for ten years to 2024.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the GD therapeutics market.
  • Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
  • Analysis of the current and future market competition in the global GD therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

The report will enable you to -

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Additionally a list of acquisition targets included in the pipeline product company list.
  • Develop business strategies by understanding the trends shaping and driving the GD therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the GD therapeutics market in future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Track drug sales in the 7MM GD therapeutics market from 2014-2024.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Table of Contents

1. Table of Contents

  • 1.1. List of Tables
  • 1.2. List of Figures

2. Introduction

  • 2.1. Catalyst
  • 2.2. Related Reports

3. Disease Overview

  • 3.1. Etiology and Pathophysiology
    • 3.1.1. Etiology
    • 3.1.2. Pathophysiology
  • 3.2. Symptoms
  • 3.3. Prognosis
  • 3.4. Quality of Life

4. Epidemiology

  • 4.1. Disease Background
  • 4.2. Risk Factors and Comorbidities
  • 4.3. Global Trends
    • 4.3.1. US
    • 4.3.2. 5EU
    • 4.3.3. Israel
  • 4.4. Forecast Methodology
    • 4.4.1. Sources Used
    • 4.4.2. Sources Not Used
    • 4.4.3. Forecast Assumptions and Methods
  • 4.5. Epidemiological Forecast for Gaucher Disease (2014-2024)
    • 4.5.1. Total Prevalent Cases of Type 1 Gaucher Disease
    • 4.5.2. Age-Specific Total Prevalent Cases of Type 1 Gaucher Disease
    • 4.5.3. Sex-Specific Total Prevalent Cases of Type 1 Gaucher Disease
    • 4.5.4. Age-Standardized Total Prevalence of Type 1 Gaucher Disease
    • 4.5.5. Total Prevalent Cases of Type 2 Gaucher Disease
    • 4.5.6. Sex-Specific Total Prevalent Cases of Type 2 Gaucher Disease
    • 4.5.7. Total Prevalent Cases of Type 3 Gaucher Disease
    • 4.5.8. Age-Specific Total Prevalent Cases of Type 3 Gaucher Disease
    • 4.5.9. Sex-Specific Total Prevalent Cases of Type 3 Gaucher Disease
    • 4.5.10. Age-Standardized Total Prevalence of Type 3 Gaucher Disease
  • 4.6. Discussion
    • 4.6.1. Epidemiological Forecast Insight
    • 4.6.2. Limitations of the Analysis
    • 4.6.3. Strengths of the Analysis

5. Current Treatment Options

  • 5.1. Overview
    • 5.1.1. Enzyme Replacement Therapy
    • 5.1.2. Substrate Reduction Therapy
    • 5.1.3. ERT Dosing Issues
    • 5.1.4. Treatment Guidelines
  • 5.2. Product Profiles
    • 5.2.1. Enzyme Replacement Therapies
    • 5.2.2. Substrate Reduction Therapies

6. Unmet Needs Assessment and Oppportunity Analysis

  • 6.1. Overview
  • 6.2. Treatment of the Neurological Manifestations of Gaucher Disease
    • 6.2.1. Unmet Needs
    • 6.2.2. Gap Analysis
    • 6.2.3. Opportunity
  • 6.3. Earlier Gaucher Disease Diagnosis and Disease Severity Assessment
    • 6.3.1. Unmet Needs
    • 6.3.2. Gap Analysis
    • 6.3.3. Opportunity
  • 6.4. Lower Cost of Gaucher Disease Treatment
    • 6.4.1. Unmet Needs
    • 6.4.2. Gap Analysis
    • 6.4.3. Opportunity
  • 6.5. Improved Route of Administration
    • 6.5.1. Unmet Needs
    • 6.5.2. Gap Analysis
    • 6.5.3. Opportunity

7. R&D Strategies

  • 7.1. Overview
    • 7.1.1. Substrate Reduction Therapy
    • 7.1.2. Pharmacological Chaperones and Their Combination with ERT
  • 7.2. Clinical Trial Design
    • 7.2.1. Efficacy Endpoints
    • 7.2.2. Challenges Involved in Gaucher Disease Clinical Trials

8. Pipeline Assessment

  • 8.1. Promising Drugs in Clinical Development
  • 8.2. Innovative Early-Stage Approaches
    • 8.2.1. Overview

9. Pipeline Valuation Analysis

  • 9.1. Clinical Benchmarking of the Key Gaucher Disease Drugs
  • 9.2. Commercial Benchmarking of the Key Gaucher Disease Drugs
  • 9.3. Competitive Assessment
  • 9.4. Top-Line 10-Year Forecast
    • 9.4.1. US
    • 9.4.2. 5EU
    • 9.4.3. Israel
    • 9.4.4. Drivers and Barriers

10. Appendix

  • 10.1. Bibliography
  • 10.2. Abbreviations
  • 10.3. Methodology
  • 10.4. Forecasting Methodology
    • 10.4.1. Percentage of Diagnosed Gaucher Disease Patients
    • 10.4.2. Percentage of Drug-Treated Gaucher Disease Patients
    • 10.4.3. Drugs Included in Each Therapeutic Class
    • 10.4.4. Key Product Launch and Patent Expiry Dates
    • 10.4.5. General Pricing Assumptions
    • 10.4.6. Individual Drug Assumptions
    • 10.4.7. Generic Erosion
  • 10.5. Physicians and Specialists Included in This Study
  • 10.6. About the Authors
    • 10.6.1. Author
    • 10.6.2. Therapy Area Director
    • 10.6.3. Epidemiologist
    • 10.6.4. Global Head of Healthcare
    • 10.6.5. Global Director of Therapy Analysis and Epidemiology
  • 10.7. About GlobalData
  • 10.8. Disclaimer

List of Tables

  • Table 1: Types of Gaucher Disease and Their Main Characteristics
  • Table 2: Organ-Specific Clinical Presentation of Gaucher Disease
  • Table 3: 7MM, Sources Used to Forecast the Total Prevalent Cases of Gaucher Disease
  • Table 4: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, All Ages, Both Sexes, N, 2014-2024
  • Table 5: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, by Age, Both Sexes, N (Row %), 2014
  • Table 6: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, All Ages, N (Row %), by Sex, 2014
  • Table 7: 7MM, Total Prevalent Cases of Type 2 Gaucher Disease, Age ≤4 Years, Both Sexes, N, 2014-2024
  • Table 8: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, Age <40 Years, Both Sexes, N, 2014-2024
  • Table 9: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, by Age, Both Sexes, N (Row %), 2014
  • Table 10: Leading Branded Products for the Treatment of Gaucher Disease
  • Table 11: Gaucher Disease: Risk Assessment
  • Table 12: Recommendations and Guidelines for the Management of Gaucher Disease
  • Table 13: Product Profile - Cerezyme
  • Table 14: Mean Changes in Clinical Parameters from Baseline to Month 9 in Patients with Type 1 Gaucher Disease in Response to Cerezyme Versus Ceredase
  • Table 15: Cerezyme SWOT Analysis, 2015
  • Table 16: Product Profile - VPRIV
  • Table 17: Mean Changes in Clinical Parameters from Baseline to 12 Months in Patients with Type 1 Gaucher Disease in Response to VPRIV in the First Phase III Study
  • Table 18: Mean Changes in Clinical Parameters from Baseline to Month 9 in Patients with Type 1 Gaucher Disease in Response to VPRIV or Cerezyme
  • Table 19: VPRIV SWOT Analysis, 2015
  • Table 20: Product Profile - Elelyso
  • Table 21: Mean Changes in Clinical Parameters from Baseline to Month Nine in Patients with Type 1 GD in Response to Elelyso in the Phase III Study
  • Table 22: Mean Increases in Height and Weight from Baseline to Month 12 in Pediatric Patients with Type 1 GD in Response to Elelyso in a Phase III Study
  • Table 23: Mean Changes in Clinical Parameters at Baseline and at Month Nine in Patients with Type 1 GD in Response to Elelyso in a Phase III Study
  • Table 24: Number of Patients with Drug-Related Adverse Events in the Phase III Study of Elelyso by Dose Group
  • Table 25: Elelyso SWOT Analysis, 2015
  • Table 26: Product Profile - Zavesca
  • Table 27: Changes in the Clinical Parameters in Response to Zavesca 100mg Three Times Daily in Study 1 and Its Extension Study
  • Table 28: Changes in the Clinical Parameters in Response to Zavesca 50mg Three Times Daily in Study 2 and Its Extension Study
  • Table 29: Changes in the Clinical Parameters in Response to Treatment with Zavesca 100mg Three Times Daily in the Two-Year Maintenance Study
  • Table 30: Patients Experiencing Adverse Events During the Randomized and Extension Phases of Study 3 of Zavesca
  • Table 31: Most Frequently Reported Adverse Events in Patients Receiving Zavesca in Combination with Cerezyme in the Two-Year Maintenance Study
  • Table 32: Zavesca SWOT Analysis, 2015
  • Table 33: Recommended Cerdelga Dosage by CYP2D6 Metabolizer Status
  • Table 34: Product Profile - Cerdelga
  • Table 35: Changes in the Primary and Secondary Endpoints After Nine Months of Treatment with Cerdelga Versus Placebo in the ENGAGE Trial
  • Table 36: CYP2D6 Metabolizer Status of Patients Receiving Cerdelga or Cerezyme in the ENCORE Trial
  • Table 37: Mean Changes in the Clinical Parameters from Baseline to Month 12 in Patients with Type 1 GD Who Were Switched to Cerdelga or Received Cerezyme in the ENCORE Trial
  • Table 38: Adverse Events that Occurred in ≥10% of Patients Treated with Cerdelga and More Frequently Than with Placebo in the ENGAGE Trial
  • Table 39: Adverse Events Occurring in ≥10% of Patients and More Frequently Than with Placebo in the ENGAGE Trial
  • Table 40: Cerdelga SWOT Analysis, 2015
  • Table 41: Unmet Need and Opportunity in Gaucher Disease
  • Table 42: Early-Stage Pipeline Products for Gaucher Disease - US, 5EU, Israel, 2014-2024
  • Table 43: Clinical Benchmarking of the Key Gaucher Disease Drugs - ERTs and SRTs
  • Table 44: Commercial Benchmarking of the Key Drugs in Gaucher Disease - ERTs and SRTs
  • Table 45: Top-Line Sales Forecasts ($m) for GD, 2014-2024
  • Table 46: Key Events Impacting Sales for the Gaucher Disease Market, 2014-2024
  • Table 47: Gaucher Disease Market - Drivers and Barriers, 2014-2024
  • Table 48: Key Product Launch Dates in the Gaucher Disease Market, 2014-2024
  • Table 49: Key Patent Expiry Dates in the Gaucher Disease Market, 2014-2024

List of Figures

  • Figure 1: Classification of Gaucher Disease
  • Figure 2: Simultaneous Accumulation of Unfolded Proteins and GC in Lysosomal Macrophages in Gaucher Disease - Current Therapeutic Interventions
  • Figure 3: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, All Ages, Both Sexes, N, 2014-2024
  • Figure 4: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, by Age, Both Sexes, N
  • Figure 5: 7MM, Total Prevalent Cases of Type 1 Gaucher Disease, All Ages, N, by Sex, 2014
  • Figure 6: 7MM, Age-Standardized Total Prevalence of Type 1 Gaucher Disease (%), All Ages, by Sex, 2014
  • Figure 7: 7MM, Total Prevalent Cases of Type 2 Gaucher Disease, Age ≤4 Years, Both Sexes, N, 2014-2024
  • Figure 8: 7MM, Total Prevalent Cases of Type 2 Gaucher Disease, All Ages, by Sex, N, 2014
  • Figure 9: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, Age <40 Years, Both Sexes, N, 2014-2024
  • Figure 10: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, by Age, Both Sexes, N, 2014
  • Figure 11: 7MM, Total Prevalent Cases of Type 3 Gaucher Disease, Age <40 Years, by Sex, N, 2014
  • Figure 12: 7MM, Age-Standardized Total Prevalence of Type 3 Gaucher Disease (%), Age <40 Years, by Sex, 2014
  • Figure 13: Competitive Assessment of Gaucher Disease Therapies in the 7MM, 2014-2024
  • Figure 14: Sales for the Gaucher Disease Market in the 7MM, 2014-2024
  • Figure 15: Sales of Gaucher Disease Drugs by Country, 2014 and 2024
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