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表紙:オーファンドラッグ (希少疾病用医薬品) の世界市場 - 臨床試験、特許、ガイドラインの考察
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オーファンドラッグ (希少疾病用医薬品) の世界市場 - 臨床試験、特許、ガイドラインの考察

Global Orphan Drug Clinical Trials, Patent & Guidelines Insight 2026

出版日: | 発行: PNS Pharma | ページ情報: 英文 2400 Pages | 納期: 即日から翌営業日

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オーファンドラッグ (希少疾病用医薬品) の世界市場 - 臨床試験、特許、ガイドラインの考察
出版日: 2020年03月12日
発行: PNS Pharma
ページ情報: 英文 2400 Pages
納期: 即日から翌営業日
  • 全表示
  • 概要
  • 図表
  • 目次
概要

世界のオーファンドラッグ (希少疾病用医薬品) 市場は、2018年と比較すると、2026年までに150%の市場成長が予測されています。

オーファンドラッグ市場は、対象の疾患が生命を脅かすものであることから、近年、有望な治療市場として認識されています。FDAとEMAは、米国での症例数が200万人未満、EUでの症例数が1万人に5人以下の場合、オーファンドラッグに指定しています。これまでオーファンドラッグは、大手製薬会社が開発・販売しても採算が合わないと判断し、見過ごされていましたが、現在では、オーファンドラッグの研究開発が主流となっています。オーファンドラッグに関連した研究開発活動の大部分は小規模の製薬会社によって行われており、大企業によって研究開発されたオーファンドラッグは25%未満となっています。

当レポートでは、オーファンドラッグ (希少疾病用医薬品) 市場について調査分析し、世界の市場機会、米国の市場シェア、米国の市場機会、世界の臨床考察、上市薬、FDA・EMAの規制、指定基準・償還政策などについて、体系的な情報を提供しています。

第1章 オーファンドラッグとは

第2章 世界のオーファンドラッグ指定の基準

  • 米国
  • 欧州
  • アジア・オーストラリア

第3章 オーファンドラッグの市場優先権と特許保護

第4章 世界のオーファンドラッグの償還政策

  • 米国
  • 欧州
  • アジア

第5章 世界のオーファンドラッグ市場機会の考察

  • 世界のオーファンドラッグ販売機会
  • 市場:クラスの多様性別
  • 市場:治療応用別
  • 市場:地域別

第6章 オーファン指定薬の臨床試験に対するFDAの規制

  • 推薦状のリクエストに関する内容と形式
  • 推薦状の付与・拒否に関する規定
  • オーファンドラッグ指定のリクエストに関する内容と形式
  • 外国のスポンサーに対するオーファンドラッグの状況と居住エージェントの確認
  • オーファンドラッグ指定・承認済み薬の指定のリクエストに関するタイミング、など

第7章 オーファン指定薬の臨床試験に対するEMAの規制

  • COMP (Committee for Orphan Medicinal Products)
  • 欧州でオーファン指定を申請する方法
  • マーケティングの承認と市場優先権
  • 別のスポンサーにオーファン指定を変更する
  • 開発に関する年次報告書の提出義務、など

第8章 オーファン指定薬の臨床試験に対するアジアの規制

  • 台湾
  • 日本

第9章 世界のオーファン臨床パイプラインの概要

  • 企業別
  • 薬剤分類
  • 剤形
  • 適応
  • 地域
  • 優先状況
  • 患者セグメント
  • 相別

第10章 不明フェーズ - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

  • 概要
  • 臨床パイプラインの考察

第11章 創薬研究フェーズ - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第12章 前臨床フェーズ - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第13章 臨床フェーズ - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第14章 フェーズI - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第15章 フェーズI/II - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第16章 フェーズII - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第17章 フェーズII/III - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第18章 フェーズIII - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第19章 申請済み - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第20章 承認済み - オーファンドラッグ臨床パイプライン:企業別、国別、適応別

第21章 上市済みオーファンドラッグの臨床考察:企業別、国別、適応別

第22章 競合情勢

  • 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
図表

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
目次

"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
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