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市場調查報告書

稀有兒童疾病的新藥開發:2019-2025

New Drug Development for Rare Pediatric Disorders Sustaining High Growth in the Global Orphan Drugs Market, 2019-2025

出版商 Frost & Sullivan 商品編碼 954126
出版日期 內容資訊 英文 99 Pages
商品交期: 最快1-2個工作天內
價格
稀有兒童疾病的新藥開發:2019-2025 New Drug Development for Rare Pediatric Disorders Sustaining High Growth in the Global Orphan Drugs Market, 2019-2025
出版日期: 2020年08月12日內容資訊: 英文 99 Pages
簡介

稀有疾病兒童藥產品可以滿足未滿足的需求,現有製藥公司和新興製藥公司有很多進入市場並建立絕對優勢的機會。但是,需要解決許多挑戰,例如研發,臨床試驗,小批量生產,報銷和定價,並且需要大力偏離製藥業傳統方法的努力。

本報告調查了用於治療稀有兒童疾病的新藥市場,包括市場概況和動態,稀有兒童疾病藥市場預測和趨勢,競爭格局,主要合作,產品線分析,我們提供諸如增長機會之類的信息。

目錄

行政儀表板

  • 調查目的
  • 主要發現
  • 市場工程評估
  • 調查範圍和細分市場
  • 大市場主題
  • 增長的戰略要求

市場概況和動態

  • 定義
  • 市場背景
  • 市場增長動力
  • 市場增長的障礙因素
  • 罕見病藥物標準
  • 指定和批准的孤兒藥
  • 批准用於罕見病的新化合物(NME)
  • 立法和監管政策
  • 財政和非貨幣激勵措施
  • 世界市場吸引力
  • 可用資源
  • 各廠商的功率圖

預測和趨勢-稀有兒童疾病藥物市場整體

  • 市場工程評估
  • 調查過程和調查方法
  • 預測先決條件
  • 總收入預測
  • 按產品組劃分的總收入預測
  • 按產品組細分的收入
  • 按地區分的總收入預測
  • 按地區劃分的收入明細
  • 按治療藥物類型劃分的收入預測
  • 除腫瘤外的前4個疾病領域
  • 腫瘤收入細分
  • 其他分部收入細分
  • 稀有兒童疾病藥物的機會

競爭情況

  • 各公司的市場佔有率
  • 競爭情況的討論
  • 競爭環境

主要合作關係

  • 併購評估
  • 合作評價
  • 值得關注的重點公司

管道分析

  • 管道分析
  • 按治療領域劃分的管線概況
  • 按技術類型分類的管道摘要
  • 有前景的管道產品

視覺場景

  • 從宏觀到微觀
  • 救贖挑戰與機遇
  • 競爭策略
  • 競爭策略

願景和戰略增長管道

遠景和戰略增長機會

  • 願景與戰略-增長機會
  • 增長機會1基因療法的開發和製造外包
  • 基因治療的現狀
  • 增長機會2-CDx個性化醫學
  • 對CDx的需求
  • 稀有兒童疾病情況下CDx的現狀
  • 增長機會3雜交臨床試驗
  • 稀有疾病混合試驗的定義和應用
  • 生態系統參與者促進罕見病混合臨床試驗
  • 增長機會4藥物轉化(DPRx)
  • 稀有藥物轉移藥物的現狀
  • 藥物轉移策略
  • 精選公司-Healx
  • 成長機會5兒童罕見疾病
  • 治療領域的商機
  • 主要資產面臨與仿製藥/生物仿製藥競爭的風險
  • 增長機會影響分析

結論

  • 要避免的陷阱
  • 增長的戰略要求
  • 案例研究-Takeda、Bayer
  • 案例研究-Amicus Therapeutics
  • 案例研究-Strimvelis
  • 案例研究-Glybera
  • 3大預測
  • 免責聲明

附錄

  • 縮寫
  • 圖表列表
目錄
Product Code: MF5C-52

AI-powered Early Disease Diagnosis and Drug Repurposing will Create New Growth Opportunities

Rare diseases have been an increasing area of focus as three waves have converged in recent years: genomic research and innovations becoming mainstream; the forthcoming regulatory policies and financial incentives put in place by the federal agencies; and the increasingly patient-centric and coordinated efforts of patient advocacy groups, caregivers, and centers of excellence (COEs).

This research service provides an overview of the orphan drugs market dynamics, estimating the market size broken down by different segments. It also dives deeper into the different technological, geographical, and therapeutic opportunities with an objective assessment of the disclosed pipelines. Notably, the study has an expanded coverage of not only rare diseases, but also hard-to-cure and "commercially non-viable" diseases.

The orphan drugs market has been characterized by severe unmet needs with therapies available for less than 8% of the identified diseases, creating an abundant opportunity for established and new companies alike to not only enter the market but also establish absolute dominance. However, this will require companies to navigate through R&D, clinical trial, small-volume manufacturing, and reimbursement/pricing challenges as there is a measurable departure from traditional pharma rules.

This study captures the competitive landscape and the different strategies employed by companies to stay ahead of the curve. It also identifies game-changing companies leading innovation from the front. Acknowledging that the market continues to be dominated by larger pharma companies, the study identifies opportunities for mid and small segment companies to tap this market. The study thus elaborates on opportunities in drug repurposing, personalized medicine, hybrid clinical trials, and untapped therapeutic areas.

Furthermore, this research service lays down strategic imperatives for prioritizing markets for new product launch, working with the right communities which act as conduits to the right patient populations, and the right set of technologies to be leveraged to accelerate innovation. In conjunction with the core manufacturers, the research service also provides in-depth analysis of other ecosystem stakeholders such as logistic service providers, contract service providers, diagnostic companies, investors, and advocacy groups.

Information is also given on some of the leading M&A activities impacting the market as well as unconventional collaboration agreements laying the foundation for propelling innovations toward licensure and delivery. Furthermore, present and future market trends like increasing price transparency and drug affordability, which would shape the market, are discussed.

Key Issues Addressed:

  • What is the total market size and what is the projected growth for the global orphan drugs market? Which are the largest growth segments to be pursued?
  • How are the various regions around the globe positioned to monetize the market opportunities? What are the key regional barriers to entry?
  • What are the new risk mitigation strategies and alternative big bets to circumvent R&D challenges? Which are the lucrative partnership models for the long term?
  • What are the market-defining macro trends mandating the need to deploy new digital solutions suite and which cross sections of the value chain will benefit the most?
  • How are product offerings in this market likely to emerge? What is the rate of innovation adoption and how is it solving the unmet needs of customers?
  • Which are the key game-changing companies in the market and how are they riding the growth curve?

Table of Contents

Executive Dashboard

  • Purpose of this Experiential Study
  • 5-step Process to Transformational Growth
  • Key Questions this Study will Answer
  • Key Findings
  • Key Findings (continued)
  • Market Engineering Measurements
  • Scope and Segmentation
  • Big Market Themes
  • Strategic Imperatives for Growth

Market Overview and Dynamics

  • Definitions
  • Market Background
  • Market Drivers
  • Market Restraints
  • Orphan Drug Criteria
  • Designated and Approved Orphan Drugs
  • Approved Orphan New Molecular Entities (NMEs)
  • Legislative and Regulatory Policies
  • Financial and Non-Financial Incentives
  • Global Market Attractiveness Outlook
  • Resources to Leverage
  • Vendor Landscape

Forecast and Trends-Total Orphan Drugs Market

  • Market Engineering Measurements
  • Research Process and Methodology
  • Forecast Assumptions
  • Total Revenue Forecast
  • Total Revenue Forecast by Product Group
  • Percent Revenue Breakdown by Product Group
  • Total Revenue Forecast by Region
  • Percent Revenue Breakdown by Geography
  • Percent Revenue Forecast by Therapy Type
  • Magnified View of the Top 4 Non-Oncology Disease Areas
  • Magnified View of the Top 4 Non-Oncology Disease Areas (continued)
  • Percent Revenue Breakdown by Oncology
  • Percent Revenue Breakdown for Other Segments
  • Total Opportunity with Orphan Drugs

Competitive Landscape

  • Market Share by Companies
  • Competitive Landscape Discussion
  • Competitive Environment

Key Collaboration Agreements

  • M&A Assessment
  • M&A Assessment (continued)
  • Collaboration Assessment
  • Key Companies to Watch
  • Key Companies to Watch (continued)

Pipeline Analysis

  • Pipeline Analysis
  • Pipeline Summary by Therapy Area
  • Pipeline Summary by Technology Type
  • High-potential Pipeline Products

Visioning Scenario

  • Macro to Micro Visioning
  • Reimbursement-Challenges and Opportunity
  • Competitive Strategies
  • Competitive Strategies-First-to-market vs Late Entrant

Vision and Strategy-Growth Pipeline

  • Levers for Growth

Vision and Strategy-Growth Opportunities

  • Vision and Strategy-Growth Opportunities
  • Growth Opportunity 1-Outsourcing Gene Therapy Development and Manufacturing
  • Current Gene Therapy Landscape
  • Growth Opportunity 2-CDx enabled Personalized Medicine
  • Need for CDx
  • Current State of CDx in the Orphan Drugs Landscape
  • Growth Opportunity 3-Hybrid Clinical Trials
  • Definition and Applications of Hybrid Trials for Rare Diseases
  • Ecosystem Participants Facilitating Hybrid Clinical Trials for Rare Diseases
  • Growth Opportunity 4-Drug Repurposing (DPRx)
  • Current State of Repurposed Drugs in the Orphan Drugs Landscape
  • Drug Repurposing Strategies
  • Key Company to Watch-Healx
  • Growth Opportunity 5-Rare Pediatric Disorders
  • Business Opportunity by Therapy Areas
  • Key Assets at the Risk of Generic/Biosimilar Competition
  • Growth Opportunity Impact Analysis

The Last Word

  • Pitfalls to Avoid
  • Strategic Imperatives for Growth
  • Case Study-Takeda and Bayer
  • Case Study-Amicus Therapeutics
  • Case Study-Strimvelis
  • Case Study-Glybera
  • Top 3 Predictions for the Market
  • Legal Disclaimer

Appendix

  • Abbreviations
  • List of Exhibits
  • List of Exhibits (continued)
  • List of Exhibits (continued)