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市場調查報告書

孤兒藥的全球市場:到2020年的未來發展預測

Global Orphan Drug Market Future Outlook 2020

出版商 KuicK Research 商品編碼 331910
出版日期 內容資訊 英文 2200 Pages
商品交期: 最快1-2個工作天內
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孤兒藥的全球市場:到2020年的未來發展預測 Global Orphan Drug Market Future Outlook 2020
出版日期: 2015年06月05日 內容資訊: 英文 2200 Pages
簡介

孤兒藥,由於患者數少而被製藥公司忽略。可是自1983年美國孤兒藥法通過以來,製藥公司著手孤兒藥的開發,之後歐洲法律規章也施行了。

本報告提供全球孤兒藥市場相關調查、市場概要、FDA、EMA臨床實驗相關法規、全球臨床實驗平台趨勢等,以及參與企業簡介彙整。

第1章 孤兒藥的簡介

第2章 孤兒藥商品化的有利市場動向

  • 景氣變動
  • 藥物專利的失效
  • 金融、非金融主張
  • 專利及市場壟斷性
  • 研究開發投資

第3章 全球孤兒藥市場概要

第4章 全球孤兒藥市場區隔分析

  • 生物學及非生物學性孤兒藥
  • 治療市場區隔
  • 各地區市場區隔

第5章 全球孤兒藥臨床開發平台概要

第6章 美國的孤兒藥市場概要

  • 孤兒藥指定標準
  • 市場概要
  • 償付政策

第7章 歐洲的孤兒藥市場概要

  • 孤兒藥指定標準
  • 市場概要
  • 償付政策

第8章 亞洲的孤兒藥市場概要

  • 孤兒藥指定標準
  • 市場概要
  • 償付政策

第9章 FDA對孤兒藥指定藥物臨床實驗的法律規章

第10章 EMA對孤兒藥指定藥物臨床實驗的法律規章

第11章 亞洲對孤兒藥指定藥物臨床實驗的法律規章

  • 台灣
  • 日本

第12章 全球臨床實驗平台趨勢及專利分析:各企業、國家、適應、Phase

第13章 已上市孤兒藥的臨床性考察及專利分析:各企業、國家、適應症

第14章 在臨床實驗中中止、中斷的孤兒藥

  • 沒有開發報告
  • 中止
  • 預登記提交撤回
  • 撤出市場
  • 中斷

第15章 競爭環境

目錄

Disease incidences are rapidly escalating across the globe and few rare diseases have emerged for which effective medications are not available. Occurrence of such disease is not unusual and their roots could be traced back to genetic or metabolic anomalies. They affect limited number of individuals and widely uncommon in nature. Such diseases are called rare diseases and corresponding drugs are known as orphan drugs. The orphan drug status to a particular formulation depends upon the public health policy due to which number of individuals may vary from country to country. This fact reflects the marketing opportunity in particular region but largely limited market size is observed. Previously, the market for orphan drugs was not as lucrative as it is today. Due to small patient population, the pharmaceutical companies generally ignored them and hence these drugs were given the designation "orphan". However, in 1983, the Orphan Drug Act was passed in the US in order to encourage pharmaceutical companies to develop orphan drugs followed by enactment of law for orphan drugs by European Medical Agency in 1999.

Encouragingly, large numbers of orphan drugs have been introduced in global market and many more are at different stages of clinical trials. Innovative technologies are being employed for the development of these orphan drugs. For instance, bone marrow stem-cells are being explored for treatment of Amyotrophic Lateral Sclerosis (ALS). Targeted RNAi therapies for Duchenne Muscular Dystrophy (DMD) are under investigation. Market introduction of orphan drugs based on these innovative therapies may take time but their future seems optimistic due to increasing activity of pharmaceutical companies in this segment. Moreover, owing to continuous effort of orphan drug developers for several decade, many medical breakthroughs have been achieved which are also applicable for other diseases. For instance, statins drugs developed for homozygous familial hypercholesterolemia is widely prescribed for cardiac disorders.

Strong clinical pipeline of orphan drugs for different rare diseases using innovative technologies makes them worthy candidates having high pharmacological and commercialization potential. But, challenges in clinical trials may hamper their growth and limit their marketing potential. Owing to low patient base it is not always possible to generate high quality data in clinical trials. Regulatory authorities also understand this issue due to which some relaxations have been made which is imperative in such scenarios. Their clinical trials are quite risky and orphan drug developer may run the chances of having high failure rates. This may lead to loss of valuable time and resources in clinical trials resulting in dominance of big pharmaceutical companies in this segment. However, few small/mid-sized orphan drug developers could also be found in this segment which is developing autoimmune and cancer therapeutics.

In recent years, several new rare diseases are being included in clinical trials for the development of orphan drug candidate. Oncology, Genetic diseases, Autoimmune, pulmonary disease, Central nervous system and others are main focus of orphan drug developers. All of these diseases have large patient base but their rare forms doesn't have suitable therapeutics which reflects the marketing potential of orphan drugs in these segments. Out of these, oncology segment has got maximum attention followed by rare genetic disorders, neurological disorder and autoimmune disorders. In a bigger view, orphan drug segment is expected to grow several folds in coming years due to several favorable factors. Introduction of innovative technologies, favorable pricing, reimbursement, unmet medical necessities and strong clinical pipeline could be attributed as important factors responsible for their growth.

"Global Orphan Drug Market Future Outlook 2020" report highlights:

  • Global Orphan Drug Market Overview
  • Global Orphan Drug Market Segment Analysis
  • FDA & EMA Regulation for Clinical Trials Orphan Designated Drugs
  • Orphan Drug Designation Criteria & Reimbursement Policy by Region
  • Comprehensive Insight on Global Orphan Drugs Clinical Pipeline & Patent Analysis by Company, Country, Indication & Phase
  • Global Orphan Drugs Clinical Pipeline: 697 Drugs
  • Majority Orphan Drugs in Phase-II Trials: 249 Drugs
  • Marketed Orphan Drugs: 274 Drugs

Table of Contents

1. Introduction to Orphan Drug

2. Favorable Market Dynamics for Orphan Drugs Commercialization

  • 2.1 Economic Viability
  • 2.2 Drug Patent Expiry
  • 2.3 Financial & Non-Financial Incentives
  • 2.4 Patent & Market Exclusivity
  • 2.5 R&D Investment

3. Global Orphan Drug Market Overview

4. Global Orphan Drug Market Segment Analysis

  • 4.1 Biological & Non-Biological Orphan Drugs
  • 4.2 Therapeutic Segmentation
  • 4.3 Regional Segmentation

5. Global Orphan Drug Clinical Pipeline Overview

6. US Orphan Drug Market Overview

  • 6.1 Orphan Drug Designation Criteria
  • 6.2 Market Overview
  • 6.3 Reimbursement Policy

7. Europe Orphan Drug Market Overview

  • 7.1 Orphan Drug Designation Criteria
  • 7.2 Market Overview
  • 7.3 Reimbursement Policy

8. Asia Orphan Drug Market Overview

  • 8.1 Orphan Drug Designation Criteria
  • 8.2 Market Overview
  • 8.3 Reimbursement Policy

9. FDA Regulation for Clinical Trials Orphan Designated Drugs

  • 9.1 Content & Format of a Request for Written Recommendations
  • 9.2 Provision for Granting & Refusing Written Recommendations
  • 9.3 Content and Format of a Request for Orphan Drug Designation
  • 9.4 Verification of Orphan Drug Status & Resident Agent for Foreign Sponsor
  • 9.5 Timing of Requests for Orphan Drug Designation & Designation of Already Approved Drugs
  • 9.6 Deficiency Letters and Granting Orphan Drug Designation
  • 9.7 Refusal to Grant Orphan Drug Designation
  • 9.8 Amendment & Change in Ownership to Orphan Drug Designation
  • 9.9 Publication & Revocation of Orphan Drug Designations
  • 9.10 Annual Reports of Holder of Orphan Drug Designation
  • 9.11 Scope & FDA Recognition of Orphan Drug Exclusive Approval
  • 9.12 Protocols for Investigations & Availability of Information

10. EMA Regulations for Clinical Trials of Orphan Designated Drugs

  • 10.1 Committee for Orphan Medicinal Products
  • 10.2 How to Apply for Orphan Designation in Europe
  • 10.3 Marketing Authorization & Market Exclusivity
  • 10.4 Transferring an Orphan Designation to another Sponsor
  • 10.5 Mandatory Submission of Annual Report On Development
  • 10.6 Incentives for Micro, Small and Medium-Sized Enterprises
  • 10.7 Fee Reductions for Designated Orphan Medicinal Products
  • 10.8 Procedure for Orphan Designation & Incentives for R&D (Regulation (EC) No 141/2000)

11. Asian Regulations for Clinical Trials of Orphan Designated Drugs

  • 11.1 Taiwan Rare Disease and Orphan Drug Act
  • 11.2 Japan Orphan Drug Regulation

12. Global Orphan Drugs Clinical Pipeline Insight & Patent Analysis by Company, Country, Indication & Phase

  • 12.1 Unknown
  • 12.2 Research
  • 12.3 Preclinical
  • 12.4 Clinical
  • 12.5 Phase-I
  • 12.6 Phase-I/II
  • 12.7 Phase-II
  • 12.8 Phase-II/III
  • 12.9 Phase-III
  • 12.10 Preregistration
  • 12.11 Registered

13. Marketed Orphan Drugs Clinical Insight & Patent Analysis by Company, Country & Indication

14. Discontinued & Suspended Orphan Drugs in Clinical Trials

  • 14.1 No Development Reported
  • 14.2 Discontinued
  • 14.3 Preregistration-Submission Withdrawal
  • 14.4 Market Withdrawal
  • 14.5 Suspended

15. Competitive Landscape

  • 15.1 AOP Orphan
  • 15.2 Agenus
  • 15.3 Alexion
  • 15.4 Bristol Myers Squibb
  • 15.5 Biogen Idec
  • 15.6 Celgene
  • 15.7 Eli Lilly
  • 15.8 Genethon
  • 15.9 Genzyme Corporation
  • 15.10 Glaxosmithkline
  • 15.11 Merck
  • 15.12 Novartis Pharmaceuticals
  • 15.13 Orphan Europe
  • 15.14 Pfizer
  • 15.15 Prosensa
  • 15.16 Rare Disease Therapeutics
  • 15.17 Roche
  • 15.18 Sanofi
  • 15.19 Shire
  • 15.20 Teva Pharmaceutical

List of Tables

  • Table 1-1: Orphan Drugs in the US and their Costs
  • Table 1-2: Orphan Drugs in the EU and their Costs

List of Figures

  • Figure 2-1: Orphan v/s Non-Orphan Drugs -Phase II to Launch Clinical Development Time
  • Figure 2-2: Probability of Regulatory Success of Orphan v/s Non-Orphan Drugs
  • Figure 3-1: Global Orphan Drugs Market Value (US$ Billion), 2013-2020
  • Figure 3-2: US Orphan Drugs Market Value (US$ Billion), 2013-2020
  • Figure 3-3: Europe Orphan Drugs Market Value (US$ Billion), 2013-2020
  • Figure 3-4: Asia* Orphan Drugs Market Value (US$ Billion), 2013-2020
  • Figure 4-1: Biological & Non Biological Orphan Drug Segment (%), 2014 & 2020
  • Figure 4-2: Biological & Non Biological Orphan Drug Market (US$ Billion), 2013-2020
  • Figure 4-3: Global Orphan Drugs Market by Therapeutic Area, 2012 & 2020
  • Figure 4-4: Regional Markets for Orphan Drugs, 2014 & 2020
  • Figure 5-1: Global - Orphan Drug Pipeline by Phase (%), 2015
  • Figure 5-2: Global - Orphan Drug Pipeline by Phase (Numbers), 2015
  • Figure 5-3: Global - No Development Reported in Orphan Drug Pipeline by Phase (%), 2015
  • Figure 5-4: Global - No Development Reported in Orphan Drug Pipeline by Phase (Numbers), 2015
  • Figure 5-5: Global - Discontinued Orphan Drug Pipeline by Phase (%), 2015
  • Figure 5-6: Global - Discontinued Orphan Drug Pipeline by Phase (Numbers), 2015
  • Figure 5-7: Global - Suspended Orphan Drug Pipeline by Phase (%), 2015
  • Figure 5-8: Global - Suspended Orphan Drug Pipeline by Phase (Numbers), 2015
  • Figure 6-1: US Market for Orphan Drugs (US$ Billion), 2012-2020
  • Figure 6-2: US - Orphan Drug Pipeline by Phase (%), 2015
  • Figure 6-3: US - Orphan Drug Pipeline by Phase (Number), 2015
  • Figure 6-4: US - No Development Reported in Orphan Drug Pipeline by Phase (%), 2015
  • Figure 6-5: US - No Development Reported in Orphan Drug Pipeline by Phase (Number), 2015
  • Figure 6-6: US - Discontinued Orphan Drug Pipeline by Phase (%), 2015
  • Figure 6-7: US - Discontinued Orphan Drug Pipeline by Phase (Number), 2015
  • Figure 6-8: US - Suspended Orphan Drug Pipeline by Phase (%), 2015
  • Figure 6-9: US - Suspended Orphan Drug Pipeline by Phase (Number), 2015
  • Figure 7-1: European Market for Orphan Drugs (US$ Billion), 2012-2020
  • Figure 7-2: Europe - Orphan Drug Pipeline by Phase (%), 2015
  • Figure 7-3: Europe - Orphan Drug Pipeline by Phase (Numbers), 2015
  • Figure 7-4: Europe - No Development Reported in Orphan Drug Pipeline by Phase (%), 2015
  • Figure 7-5: Europe - No Development Reported in Orphan Drug Pipeline by Phase (Numbers), 2015
  • Figure 7-6: Europe - Discontinued Orphan Drug Pipeline by Phase (%), 2015
  • Figure 7-7: Europe - Discontinued Orphan Drug Pipeline by Phase (Numbers), 2015
  • Figure 7-8: Europe - Suspended Orphan Drug Pipeline by Phase (%), 2015
  • Figure 7-9: Europe - Suspended Orphan Drug Pipeline by Phase (Numbers), 2015
  • Figure 8-1: Asia Pipeline by Phase (%), 2015
  • Figure 8-2: Asia Pipeline by Phase (Number), 2015
  • Figure 8-3: No Development Reported Asia Pipeline by Phase (%), 2015
  • Figure 8-4: No Development Reported Asia Pipeline by Phase (Number), 2015
  • Figure 8-5: Discontinued Asia Pipeline by Phase (%), 2015
  • Figure 8-6: Discontinued Asia Pipeline by Phase (Number), 2015
  • Figure 8-7: Asia - Number of Orphan Drug by Clinical Phase
  • Figure 11-1: Japan Orphan Drug/Medical Device Designation System
  • Figure 11-2: Japan Orphan Drug/Device Designation Process
  • Figure 15-1: AOP Orphan
  • Figure 15-2: Agenus Pipeline
  • Figure 15-3: Genethon Drug Pipeline
  • Figure 15-4: Genzyme Drug Pipeline
  • Figure 15-5: Orphan Europe Pipeline
  • Figure 15-6: Prosensa Drug Pipeline
  • Figure 15-7: Shire Drug Pipeline
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