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市場調查報告書

孤兒藥 (罕見疾病用醫藥品)的全球市場 - 臨床試驗,專利,指南的考察

Global Orphan Drug Clinical Trials, Patent & Guidelines Insight 2026

出版商 PNS Pharma 商品編碼 928257
出版日期 內容資訊 英文 2400 Pages
商品交期: 最快1-2個工作天內
價格
孤兒藥 (罕見疾病用醫藥品)的全球市場 - 臨床試驗,專利,指南的考察 Global Orphan Drug Clinical Trials, Patent & Guidelines Insight 2026
出版日期: 2020年03月12日內容資訊: 英文 2400 Pages
簡介

預計到2026年全球孤兒藥 (罕見疾病用醫藥品) 市場與2018年相比將增長150%。。

孤兒藥市場最近被認為是一個有前途的治療市場,因為它針對的疾病威脅生命。FDA和EMA已將孤兒藥定義為在美國的病例少於200萬例,在歐盟的病例少於5例(一萬人中)。早期,大型製藥公司忽略了孤兒藥的細分市場,因為開發和銷售這些藥物被認為不太賺​​錢。與孤兒藥有關的大部分研發活動都是由小型製藥公司進行的,只有不到25%的孤兒藥是由大公司進行研發的。

本報告提供孤兒藥 (罕見疾病用醫藥品) 市場相關調查分析,提供世界的市場機會,美國的市場佔有率,美國的市場機會,全球臨床考察,上市藥,FDA·EMA的法規,指定標準·償付政策等相關的系統性資訊。

第1章 所謂孤兒藥

第2章 全球孤兒藥指定的標準

  • 美國
  • 歐洲
  • 亞洲·澳洲

第3章 孤兒藥的市場優先權和專利保護

第4章 全球孤兒藥的償付政策

  • 美國
  • 歐洲
  • 亞洲

第5章 全球孤兒藥市場機會的考察

  • 全球孤兒藥銷售機會
  • 市場:各類別變化
  • 市場:各治療應用
  • 市場:各地區

第6章 FDA臨床試驗孤兒指定藥物法規

  • 推薦書的要求相關內容和形式
  • 推薦書的批准和拒絕相關規定
  • 孤兒藥指定要求相關內容和形式
  • 外國的贊助商的孤兒藥狀況和居住代理商的確認
  • 孤兒藥指定·請求的時間和已批准藥品的指定時間

第7章 對orphan指定藥的臨床試驗的EMA的法規

  • COMP (Committee for Orphan Medicinal Products)
  • 如何在歐洲申請
  • 行銷的認證與市場優先權
  • 變更為另外贊助商的孤兒藥指定
  • 開發相關年度報告書的繳交義務等

第8章 亞洲孤兒藥臨床試驗法規

  • 台灣
  • 日本

第9章 全球孤兒藥臨床實驗平台概要

  • 各企業
  • 藥物分類
  • 劑型
  • 適應
  • 地區
  • 優先情形
  • 患者市場區隔
  • 各相

第10章 不明階段 - 孤兒藥臨床實驗平台:各企業,各國,各適應症

  • 概要
  • 臨床實驗平台的考察

第11章 藥物研發研究階段 - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第12章 前臨床階段 - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第13章 臨床階段 - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第14章 階段I - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第15章 階段I/II - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第16章 階段II - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第17章 階段II/III - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第18章 階段III - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第19章 已申請 - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第20章 已通過核准 - 孤兒藥臨床實驗平台:各企業,各國,各適應症

第21章 已上市孤兒藥臨床考察:各企業,各國,各適應症

第22章 競爭情形

  • AOP Orphan
  • Agenus
  • Alexion
  • Bristol Myers Squibb
  • Biogen Idec
  • Celgene
  • Eli Lilly
  • Genethon
  • Genzyme Corporation
  • Glaxosmithkline
  • Merck
  • Novartis Pharmaceuticals
  • Orphan Europe
  • Pfizer
  • Prosensa
  • Rare Disease Therapeutics
  • Roche
  • Sanofi
  • Shire
  • Teva Pharmaceutical
目錄

"Global Orphan Drug Clinical Trials, Patent & Guidelines Insight 2026" Report Highlights:

  • Global Orphan Drug Market Opportunity: US$ 300 Billion
  • US Dominates Global Orphan Drug Market: 50% Market Share
  • US Orphan Drug Opportunity To Surpass: US$ 150 Billion
  • Global Orphan Drug Clinical Insight: More Than 900 Drugs
  • Clinical Insight on Marketed Orphan Drugs: More Than 400 Drugs
  • Oncology To Dominate Orphan Drug Development: 35% Share
  • FDA & EMA Regulations For Orphan Drugs
  • Orphan Drug Designation Criteria & Reimbursement Policy by Country

The research report “Global Orphan Drug Clinical Trials, Patent & Guidelines Insight 2026” discusses about the recent trends and opportunities that the orphan drug market has brought into the pharmaceutical sector. The information related to the current status of the evolving market strategies and ongoing clinical studies by the companies involved in development of the orphan drugs is elaborately discussed in the report. The research report shares the information related to drugs that have been successfully designated as orphan drugs by respective approval authorities, with an exclusive insight on clinical uniqueness and patent information. In addition to the commercial information, the report brings a deep insight about the efforts that have been put to establish the market as it is now.

" Global Orphan Drug Market Is Estimated To Witness 150% Market Growth By 2026 As Compare To 2018"

Orphan drugs market has been recently recognized as a promising therapeutic market as the diseases that are covered under the market are life-threatening diseases. The FDA & EMA have designated a drug as orphan drug for which the cases in the US are less than 0.2 Million and not more than 5 in 10,000 people across the EU. Earlier the orphan drug segment was overlooked by the big pharmaceutical companies as developing and marketing of these drugs was considered not so profitable. Majority of the research and development activities related to orphan drugs were done by small size pharmaceutical firms and less than 25% of the orphan drugs were being researched and developed by the big firms.

The enactment of 1983 US Orphan Drug Act, as well as similar Acts in 1991 in Singapore, 1993 in Japan, 1997 in Australia and in 2000 by the European Union led to rapid transformation of global orphan drug market landscape which was earlier neglected by the multiple stake holders of the pharmaceutical industry. The structured regulatory and policy framework favoring the research and development of orphan designated drugs resulted in the much needed thrust for the development of global orphan drug market. These laws allowed the various financial incentives, market exclusivity, patent protection, high price allotment and government grants, which resulted in favorable economic environment for the entry of big pharmaceutical companies in the orphan drug segment.

The entry of mid and large size pharmaceutical companies helped in the speeding up the clinical research activities related to orphan drugs. The number of clinical trials increased drastically in last 10 years to more than 500 for orphan drugs as compared to few hundred trials in beginning of 21st century. Currently, more than 400 orphan designated drugs are commercially available in the marketed and close to 1000 drugs are undergoing clinical trials. The number of clinical trials covering the rare diseases has been observed to increase in the recent years with a major participation of the players such as Roche, Celgene, AbbVie, Johnson & Johnson, Shire, Alexion, Novo Nordisk, Sanofi and Bayer. The various major key players and the rising demand of the orphan drugs clearly depicts about the escalation that the market will experience in the future.

The availability of large number of orphan drugs by limited firms provides an excellent fundamental benefit to the emerged market in the present as well as in future. The dynamic interest scenario that has been delivered by the users since its arrival has completely changed the landscape of the market. The drastic change from few users in the past to millions of users till now has been successful in proving the importance of orphan drugs in the market. It is well witnessed from analyzing the market value of orphan drugs that clinicians as well as rare disease patients are now more inclined towards its use, thereby, promoting a form of treatment that is more mainstream.

As per report findings, the orphan drug market is open to serve the globe with an approach that is about to bring a fresh new era for the life threatening diseases. The ongoing clinical research at preclinical and clinical levels and the major trends followed by the regions such as North America and Europe are about to introduce a drastic change in overall scenario of the approach. The market is driven by the anticipation of the players and the huge commercial success that the market has foreseen in few years. The enhancement and the shape that the market has developed since years is about to get evolved as a serious option for rare diseases.

Table of Contents

1. What Are Orphan Drugs?

2. Global Orphan Drug Designation Criteria

  • 2.1. US
  • 2.2. Europe
  • 2.3. Asia & Australia
  • 2.3.1. Japan
  • 2.3.2. Taiwan
  • 2.3.3. South Korea
  • 2.3.4. Australia

3. Market Exclusivity & Patent Protection for Orphan Drugs

4. Global Orphan Drug Reimbursement Policy

  • 4.1. US
  • 4.2. Europe
  • 4.3. Asia

5. Global Orphan Drug Market Opportunity Insight 2026

  • 5.1. Global Orphan Drug Sales Opportunity
  • 5.2. Market by Class Variation
  • 5.3. Market by Therapeutic Application
  • 5.4. Market by Regions

6. FDA Regulation for Clinical Trials Orphan Designated Drugs

  • 6.1. Content & Format Of A Request For Written Recommendations
  • 6.2. Provision For Granting & Refusing Written Recommendations
  • 6.3. Content And Format Of A Request For Orphan Drug Designation
  • 6.4. Verification Of Orphan Drug Status & Resident Agent For Foreign Sponsor
  • 6.5. Timing Of Requests For Orphan Drug Designation & Designation Of Already Approved Drugs
  • 6.6. Deficiency Letters And Granting Orphan Drug Designation
  • 6.7. Refusal To Grant Orphan Drug Designation
  • 6.8. Amendment & Change In Ownership To Orphan Drug Designation
  • 6.9. Publication & Revocation Of Orphan Drug Designations
  • 6.10. Annual Reports Of Holder Of Orphan Drug Designation
  • 6.11. Scope & FDA Recognition Of Orphan Drug Exclusive Approval
  • 6.12. Protocols for Investigations & Availability of Information

7. EMA Regulations for Clinical Trials of Orphan Designated Drugs

  • 7.1. Committee for Orphan Medicinal Products
  • 7.2. How to Apply for Orphan Designation in Europe
  • 7.3. Marketing Authorization & Market Exclusivity
  • 7.4. Transferring An Orphan Designation To Another Sponsor
  • 7.5. Mandatory Submission Of Annual Report On Development
  • 7.6. Incentives For Micro, Small And Medium-Sized Enterprises
  • 7.7. Fee Reductions For Designated Orphan Medicinal Products
  • 7.8. Procedure for Orphan Designation & Incentives for R&D ( Regulation (EC) No 141/2000 )

8. Asian Regulations for Clinical Trials of Orphan Designated Drugs

  • 8.1. Taiwan Rare Disease and Orphan Drug Act
  • 8.2. Japan Orphan Drug Regulation

9. Global Orphan Clinical Pipeline Overview

  • 9.1. By Company
  • 9.2. Drug Class
  • 9.3. Formulation
  • 9.4. Indication
  • 9.5. Region
  • 9.6. Priority Status
  • 9.7. Patient Segment
  • 9.8. By Phase

10. Unknown Phase-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 10.1. Overview
  • 10.2. Clinical Pipeline Insight

11. Research Phase-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 11.1. Overview
  • 11.2. Clinical Pipeline Insight

12. Preclinical Phase-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 12.1. Overview
  • 12.2. Clinical Pipeline Insight

13. Clinical Phase-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 13.1. Overview
  • 13.2. Clinical Pipeline Insight

14. Phase-I-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 14.1. Overview
  • 14.2. Clinical Pipeline Insight

15. Phase-I/II-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 15.1. Overview
  • 15.2. Clinical Pipeline Insight

16. Phase-II-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 16.1. Overview
  • 16.2. Clinical Pipeline Insight

17. Phase-II/III-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 17.1. Overview
  • 17.2. Clinical Pipeline Insight

18. Phase-III-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 18.1. Overview
  • 18.2. Clinical Pipeline Insight

19. Preregistration-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 19.1. Overview
  • 19.2. Clinical Pipeline Insight

20. Registered-Orphan Drugs Clinical Pipeline By Company, Country & Indication

  • 20.1. Overview
  • 20.2. Clinical Pipeline Insight

21. Marketed Orphan Drugs Clinical Insight By Company, Country & Indication

  • 21.1. Overview
  • 21.2. Clinical Pipeline Insight

22. Competitive Landscape

  • 22.1. AOP Orphan
  • 22.2. Agenus
  • 22.3. Alexion
  • 22.4. Bristol Myers Squibb
  • 22.5. Biogen Idec
  • 22.6. Celgene
  • 22.7. Eli Lilly
  • 22.8. Genethon
  • 22.9. Genzyme Corporation
  • 22.10. Glaxosmithkline
  • 22.11. Merck
  • 22.12. Novartis Pharmaceuticals
  • 22.13. Orphan Europe
  • 22.14. Pfizer
  • 22.15. Prosensa
  • 22.16. Rare Disease Therapeutics
  • 22.17. Roche
  • 22.18. Sanofi
  • 22.19. Shire
  • 22.20. Teva Pharmaceutical

List of Figures

  • Figure 5-1: Global-Orphan Drugs Market Value (US$ Billion), 2018-2026
  • Figure 5-2: US-Orphan Drugs Market Value (US$ Billion), 2018-2026
  • Figure 5-3: Europe-Orphan Drugs Market Value (US$ Billion), 2018-2026
  • Figure 5-4: Asia*-Orphan Drugs Market Value (US$ Billion), 2018-2026
  • Figure 5-5: Biological & Non Biological Orphan Drug Segment (%), 2018 & 2026
  • Figure 5-6: Biological & Non Biological Orphan Drug Market (US$ Billion), 2018-2026
  • Figure 5-7: Global Orphan Drugs Market by Therapeutic Area, 2018 & 2026
  • Figure 5-8: Regional Markets for Orphan Drugs, 2018 & 2026
  • Figure 8-1: Japan Orphan Drug/Medical Device Designation System
  • Figure 8-2: Japan Orphan Drug/Device Designation Process
  • Figure 9-1: Global-Orphan Drugs Clinical Pipeline by Company (Number), 2020 till 2026
  • Figure 9-2: Global-Orphan Drugs Clinical Pipeline by Drug Class (Number), 2020 till 2026
  • Figure 9-3: Global-Orphan Drugs Clinical Pipeline by Formulation (Number), 2020 till 2026
  • Figure 9-4: Global-Orphan Drugs Clinical Pipeline by Indication (Number), 2020 till 2026
  • Figure 9-5: Global-Orphan Drugs Clinical Pipeline by Region (Number), 2020 till 2026
  • Figure 9-6: Global-Orphan Drugs Clinical Pipeline by Priority Status (Number), 2020 till 2026
  • Figure 9-7: Global-Orphan Drugs Clinical Pipeline by Patient Segment (Number), 2020 till 2026
  • Figure 9-8: Global-Orphan Drugs Clinical Pipeline by Phase (Number), 2020 till 2026
  • Figure 9-9: Global-Orphan Drugs Clinical Pipeline by Phase (%), 2020 till 2026
  • Figure 10-1: Share of Initial Development Drug in Total Orphan Drug Pipeline, 2020
  • Figure 11-1: Share of in Research Phase Orphan Drugs in Total Pipeline, 2020
  • Figure 12-1: Share of in Preclinical Phase Orphan Drugs in Total Pipeline, 2020
  • Figure 13-1: Share of in Clinical Phase Orphan Drugs in Total Pipeline, 2020
  • Figure 14-1: Share of in Phase-I Orphan Drugs in Total Pipeline, 2020
  • Figure 15-1: Share of in Phase-I/II Orphan Drugs in Total Pipeline, 2020
  • Figure 16-1: Share of in Phase-II Orphan Drugs in Total Pipeline, 2020
  • Figure 17-1: Share of in Phase-II/III Orphan Drugs in Total Pipeline, 2020
  • Figure 18-1: Share of in Phase-III Orphan Drugs in Total Pipeline, 2020
  • Figure 19-1: Share of in Preregistration Orphan Drugs in Total Pipeline, 2020
  • Figure 20-1: Share of in Registered Orphan Drugs in Total Pipeline, 2020
  • Figure 21-1: Share of in Marketed Orphan Drugs in Total Pipeline, 2020