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市場調查報告書
商品編碼
982085

罕見疾病中的藥物開發:關注臨床試驗和監管狀況

Drug Development in Rare Diseases: Focus on Clinical Trials and the Regulatory Landscape

出版日期: | 出版商: BCC Research | 英文 107 Pages | 訂單完成後即時交付

價格
  • 全貌
  • 簡介
  • 目錄
簡介

在製藥和生物技術領域,罕見疾病藥產業作為重要且有回報的市場正變得越來越重要。特定藥物發現途徑中的技術和科學創新以及遺傳學的重大發展正在推動該行業的發展。

本報告分析了罕見疾病藥的藥物開發,特別側重於臨床試驗和監管狀況,並詳細描述了罕見疾病藥以及生物,化學或非生物藥物。對孤兒藥的比較評估,關於罕見疾病藥的討論,藥品和生物製劑監管的一般框架,以及對罕見疾病藥臨床試驗,基因製造技術的監管見解的分析,它提供有關進展和創新的信息,有關罕見疾病藥名稱申請的信息等。

第1章簡介

  • 調查目標
  • 調查原因
  • 報告範圍
  • 來源
  • 調查方法
  • 分析師職業
  • BCC研究相關報告

第2章定義和背景

  • 罕見疾病的藥物類型
  • 生物罕見病藥物
  • 化學或非生物罕見疾病藥
  • 世界上罕見病的定義:按國家/地區劃分
  • 美國
  • 歐盟
  • 台灣
  • 日本
  • 澳大利亞

第3章FDA對罕見疾病藥臨床試驗的規定

  • 藥品和生物製品監管的一般框架
  • 加速批准
  • 快速通道指定
  • 突破規範
  • 優先審查
  • 管制物質法
  • 特殊協議評估
  • 新的監控和安全要求
  • 罕見疾病藥臨床試驗的規定
  • 書面推薦請求的內容和形式
  • 提供書面建議
  • 拒絕提供書面建議
  • 罕見疾病藥的名稱
  • 罕見疾病藥的獨家批准

第4章:歐洲罕見疾病藥臨床試驗的規定

  • 歐洲罕見疾病藥條例
  • 法律框架
  • 申請罕見疾病藥
  • 罕見病藥物標識申請的挑戰和維護
  • 展示重要利益
  • 時間軸
  • 指定罕見疾病藥物後的活動:年度報告
  • 正在申請銷售批准的活動
  • 維持罕見疾病藥地位

第5章亞洲罕見疾病藥臨床試驗的規定

  • 日本的罕見病藥物指定係統
  • 指定標準
  • 罕見疾病藥/醫療器械指定手續
  • 指定諮詢
  • 台灣罕見病和罕見疾病藥的監管
  • 韓國對罕見病和罕見疾病藥的監管

第6章罕見疾病藥物開發的臨床試驗趨勢

  • 三種註冊表功能
  • 臨床試驗,疾病和藥物之間的關係
  • 三個註冊管理機構的特徵以及疾病與藥物之間的關係

第7章競爭狀況和全球市場

  • 罕見疾病藥的全球銷售:按治療類別
  • 指定的罕見疾病藥數量:按地區/年份

第8章當前趨勢

  • 支持罕見疾病藥市場的因素
  • 罕見疾病藥法和類似法律
  • 技術進步與遺傳密碼
  • 通用藥物競爭
  • 專利到期日期
  • 溢價
  • 製造技術創新
  • 合作和許可協議
  • 挑戰
  • 缺乏訓練有素的專業人員
  • 脆弱的目標群體
  • 多種用法
  • 監管問題

第9章公司簡介

  • ABBVIE INC.
  • ASTELLAS PHARMA INC.
  • ASTRAZENECA PLC.
  • BRISTOL-MYERS SQUIBB
  • EISAI CO. LTD.
  • ELI LILLY AND CO.
  • F. HOFFMANN-LA ROCHE LTD.
  • GILEAD SCIENCES INC.
  • JOHNSON & JOHNSON
  • MERCK & CO.
  • NOVARTIS AG
  • PFIZER INC.
  • SANOFI-AVENTIS
  • TAKEDA PHARMACEUTICALS LTD.
  • TEVA PHARMACEUTICAL INDUSTRIES LTD.

第10章罕見病藥物的臨床試驗

  • 孤兒藥臨床試驗清單

第11章附錄:A

  • 參考
目錄
Product Code: PHM243A

Highlights:

The orphan drug industry is gaining importance as a significant and rewarding market in the pharmaceutical and biotech sectors. Technological and scientific innovations in specific drug discovery pathways as well as major developments in genetics are cultivating growth in this industry. BCC Research’s objective in conducting this report is to provide detailed information on drug development in rare diseases. This report provides a comprehensive analysis and examines the future direction of these drugs as an important means for the treatment of orphan and rare diseases.

Report Includes

An overview of the drug development in rare diseases with special focus on clinical trial and regulatory landscape

Detailed description of different kinds of orphan drugs and a comparative assessment on biological and chemical or non-biological orphan drugs

Discussion on Orphan Drug Act; insights into general framework for the regulation of drugs and biologics, and regulations for clinical trials of orphan designated drugs

Highlights of technological advances and innovations in genetic manufacturing technologies and information on terms such as generic competition, patent expirations, premium pricing and information on orphan drug designation applications

Report Scope:

This report analyzes the regulatory framework for the clinical trials of orphan drugs in rare and orphan diseases. The report also analyzes the trends and characteristics of clinical trials conducted on rare diseases from 1999-2018, using information from various sources. This report covers an overview of the global orphan drugs market and its competitive landscape.

Report Includes:

  • An overview of the drug development in rare diseases with special focus on clinical trial and regulatory landscape
  • Detailed description of different kinds of orphan drugs and a comparative assessment on biological and chemical or non-biological orphan drugs
  • Discussion on Orphan Drug Act; insights into general framework for the regulation of drugs and biologics, and regulations for clinical trials of orphan designated drugs
  • H-ighlights of technological advances and innovations in genetic manufacturing technologies and information on terms such as generic competition, patent expirations, premium pricing and information on orphan drug designation applications

Table of Contents

Chapter 1 Introduction

  • Study Goals and Objectives
  • Reasons for Doing this Study
  • Scope of Report
  • Information Sources
  • Methodology
  • Analyst's Credentials
  • Related BCC Research Reports

Chapter 2 Definitions and Background

  • Types of Orphan Drugs
  • Biological Orphan Drugs
  • Chemical or Non-biological Orphan Drugs
  • Global Definitions of Rare Diseases by Country/Region
  • U.S.
  • EU
  • Taiwan
  • Japan
  • Australia

Chapter 3 FDA Regulation of Clinical Trials on Orphan Designated Drugs

  • General Framework of the Regulation of Drugs and Biologics
  • Accelerated Approvals
  • Fast Track Designation
  • Breakthrough Designation
  • Priority Review
  • Controlled Substances Act
  • Special Protocol Assessments
  • New Surveillance and Safety Requirements
  • Regulation of Clinical Trials on Orphan Designated Drugs
  • Content and Format of a Request for Written Recommendations
  • Providing Written Recommendations
  • Refusal to Provide Written Recommendations
  • Orphan Drug Designation
  • Orphan Drug Exclusive Approval

Chapter 4 European Regulation of Clinical Trials on Orphan Designated Drugs

  • European Orphan Regulation
  • Legal Framework
  • Applying for Orphan Designation
  • Orphan Drug Designation Application Challenges and Maintenance
  • Demonstrating Significant Benefit
  • Timelines
  • Activities After Orphan Designation: Annual Reports
  • Activities During Marketing Authorization Application
  • Maintenance of the Orphan Drug Status

Chapter 5 Asian Regulation of Clinical Trials on Orphan Designated Drugs

  • Orphan Drug Designation System in Japan
  • Designation Criteria
  • Orphan Drug/Medical Device Designation Procedure
  • Designation Consultation
  • Regulation of Rare Diseases and Orphan Drugs in Taiwan
  • Regulation of Rare Diseases and Orphan Drugs in South Korea

Chapter 6 Trends in Clinical Trials for Drug Development in Rare Diseases

  • Characteristics of the Three Registries
  • The Relationship Among Clinical Trials, Diseases and Drugs
  • Characteristics of the Three Registries and Disease-Drug Relationships

Chapter 7 Competitive Landscape and Global Markets

  • Global Orphan Drug Sales by Therapeutic Category
  • Orphan Drug Designation Counts by Region and Year

Chapter 8 Current Trends

  • Factors Boosting the Orphan Drug Market
  • Orphan Drug Act and Similar Legislation
  • Technological Advances and Genetic Codes
  • Generic Competition
  • Patent Expirations
  • Premium Pricing
  • Innovations in Manufacturing Technologies
  • Collaborations and Licensing Agreements
  • Challenges
  • Lack of Trained Professionals
  • Vulnerable Target Groups
  • Multiple Usages
  • Regulatory Challenge

Chapter 9 Company Profiles

  • ABBVIE INC.
  • ASTELLAS PHARMA INC.
  • ASTRAZENECA PLC.
  • BRISTOL-MYERS SQUIBB
  • EISAI CO. LTD.
  • ELI LILLY AND CO.
  • F. HOFFMANN-LA ROCHE LTD.
  • GILEAD SCIENCES INC.
  • JOHNSON & JOHNSON
  • MERCK & CO.
  • NOVARTIS AG
  • PFIZER INC.
  • SANOFI-AVENTIS
  • TAKEDA PHARMACEUTICALS LTD.
  • TEVA PHARMACEUTICAL INDUSTRIES LTD.

Chapter 10 Clinical Trials on Drugs for Rare Diseases

  • List of Clinical Trials on Drugs for Rare Diseases

Chapter 11 Appendix: A

  • References

List of Tables

  • Table 1 : Rare Diseases and their Prevalence
  • Table 2 : List of Documents Included in the Orphan Designation Application
  • Table 3 : Summary of Orphan Designation in Europe
  • Table 4 : Overview of Organizations and Responsibilities in Japan
  • Table 5 : Comparison of the Regulation of Rare Diseases and Orphan Drugs Worldwide
  • Table 6 : Characteristics of the NCT, EUCTR and JPRN Trials
  • Table 7 : Number of Trials in NCT, EUCTR and JPRN Trials, by Recruitment Status
  • Table 8 : Number of Trials in NCT, EUCTR and JPRN Trials, by Gender
  • Table 9 : Number of Trials in NCT, EUCTR and JPRN Trials, by Phase
  • Table 10 : Number of Trials in NCT, EUCTR and JPRN Trials, by Country
  • Table 11 : Top 20 Most Studied Rare Diseases, by Number of Trials
  • Table 12 : Top 20 Most Studied Rare Diseases, by Number of Diseases
  • Table 13 : Top 20 Global Orphan Drug Sales, Through 2025
  • Table 14 : Global Orphan Drug Sales, by Therapeutic Category, 2019-2025
  • Table 15 : Growth in Orphan Designations, by Region
  • Table 16 : Clinical Trials on Drugs for Rare Diseases

List of Figures

  • Figure 1 : Orphan Designation Process, Japan
  • Figure 2 : Orphan Designation Consultation and Evaluation, Japan
  • Figure 3 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Trials
  • Figure 4 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Diseases
  • Figure 5 : Comparison of the Three Registries (NCT, EUCTR, and JPRN), by Number of Drugs
  • Figure 6 : Global Market Share of Top 10 Companies, 2019
  • Figure 7 : Global Market Shares of Top 10 Companies, 2025