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市場調查報告書

美國的孤兒藥(罕見疾病用醫藥品)的臨床實驗平台分析

US Orphan Drug Clinical Pipeline Insight 2014

出版商 PNS Pharma 商品編碼 310696
出版日期 內容資訊 英文 2000 Pages
商品交期: 最快1-2個工作天內
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美國的孤兒藥(罕見疾病用醫藥品)的臨床實驗平台分析 US Orphan Drug Clinical Pipeline Insight 2014
出版日期: 2014年08月19日 內容資訊: 英文 2000 Pages
簡介

美國孤兒藥的研究開發(R&D),由公共計劃、政府法規及政策促進。孤兒藥的R&D計劃中,包含補助金、研究支援、諸經費的免除、稅制上的優惠措施及孤兒藥市場壟斷權等。2013年美國孤兒藥市值推算為490億美元。該市場由於政府支援、高盈利等要素,預計2018年超過600億美元。

本報告提供美國的孤兒藥市場相關調查、市場概要、各階段、各國臨床實驗分析、上市藥、臨床實驗暫停、中止的藥物簡介及競爭情形等彙整資料,為您概述為以下內容。

第1章 美國的孤兒藥市場:簡介

  • 市場概要
  • 臨床實驗平台分析

第2章 美國的孤兒藥法律規範

  • 孤兒藥指定標準
  • 孤兒藥的醫療費給付政策
  • FDA法規

第3章 美國的孤兒藥臨床實驗平台:各階段、各國

  • 不明階段
  • 研究
  • 臨床前
  • 臨床
  • 階段I
  • 階段I/II
  • 階段II
  • 階段II/III
  • 階段III
  • 預登記
  • 已登記

第4章 已上市孤兒藥臨床分析:各適應症、企業

第5章 在臨床實驗平台上停止、中止的孤兒藥:各階段、適應症、企業

  • 未開發
  • 中止
  • 撤出市場
  • 暫停

第6章 競爭情形

  • Alexion
  • Biogen Idec
  • Bristol Myers Squibb
  • Celgene
  • Eli Lilly
  • Glaxosmithkline
  • Merck
  • Novartis Pharmaceuticals
  • Pfizer
  • Roche
  • Rare Disease Therapeutics
  • Sanofi

圖表清單

目錄

Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, and life-threatening in nature. The rare diseases normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which are extremely life-threatening. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the US and less than 5 in 10,000 in the EU.

The orphan drugs enjoy significant competitive advantages owing to the market exclusivity period after the drug has been authorized. Through this clause, the manufacturer of orphan drugs is given a monopoly status in the market because according to the law, no other company is allowed to market the orphan drugs during the exclusivity period. Additionally, this monopolistic power is further strengthened with the fact that no other alternative health technology exists for many orphan drugs.

In US, the research & development of orphan drugs is facilitated by public programs, government regulations, and policies. The major incentives for research & development of orphan drug include grants, research support, fee waivers, tax incentives, and orphan drug market exclusivity. In a highly specialized market like that of orphan drugs, though the impact of market exclusivity in the US has been positive yet relatively modest, a special platform for continued orphan drug development has been laid down by the economic incentives and public support mechanisms. In 2013, the US market for orphan drugs was estimated worth US$ 49 Billion. With factors like government support, high profitability and many more, this market is expected to surpass US$ 60 Billion by 2018.

"US Orphan Drug Pipeline Insight 2014" Report Highlight:

  • US Orphan Drug Market Insight & Orphan Drug Designation Criteria
  • Reimbursement Policy & FDA Regulatory Framework
  • US Orphan Drug Pipeline by Phase, Indication & Company
  • Marketed Orphan Drug Clinical Insight
  • US Orphan Drug Clinical Pipeline: 570 Drugs
  • Marketed Orphan Drugs in US: 203 Drugs
  • Majority Orphan Drugs in PHASE-II Development: 207 Drugs

Table of Contents

1. Introduction to US Orphan Market

  • 1.1 Market Overview
  • 1.2 Clinical Pipeline Overview

2. US Orphan Drug Regulatory Framework

  • 2.1 Criteria for Orphan Drug Designation
  • 2.2 Reimbursement Policy for Orphan Drugs
  • 2.3 FDA Regulation
    • 2.3.1 Content & Format for Written Recommendations
    • 2.3.2 Provision for Granting & Refusing Written Recommendations
    • 2.3.3 Content & Format for Orphan Drug Designation
    • 2.3.4 Verification of Orphan Drug Status & Resident Agent for Foreign Sponsor
    • 2.3.5 Requests for Orphan Drug Designation & Designation of Already Approved Drugs
    • 2.3.6 Deficiency Letters & Granting Orphan Drug Designation
    • 2.3.7 Refusal of Grant Orphan Drug Designation
    • 2.3.8 Amendment & Change in Ownership to Orphan Drug Designation
    • 2.3.9 Publication & Revocation of Orphan Drug Designations
    • 2.3.10 Annual Reports of Holder of Orphan Drug Designation
    • 2.3.11 Scope & FDA Recognition of Orphan Drug Exclusive Approval
    • 2.3.12 Protocols for Investigations & Availability of Information

3. US Orphan Drug Clinical Pipeline by Phase, Indication & Company

  • 3.1 Unknown
  • 3.2 Research
  • 3.3 Preclinical
  • 3.4 Clinical
  • 3.5 Phase-I
  • 3.6 Phase-I/II
  • 3.7 Phase-II
  • 3.8 Phase-II/III
  • 3.9 Phase-III
  • 3.10 Preregistration
  • 3.11 Registered

4. Marketed Orphan Drugs Clinical Insight by Indication & Company

5. Suspended & Discontinued Orphan Drugs in Clinical Pipeline by Phase, Indication & Company

  • 5.1 No development reported
  • 5.2 Discontinued
  • 5.3 Market Withdrawal
  • 5.4 Suspended

6. Competitive Landscape

  • 6.1 Alexion
  • 6.2 Biogen Idec
  • 6.3 Bristol Myers Squibb
  • 6.4 Celgene
  • 6.5 Eli Lilly
  • 6.6 Glaxosmithkline
  • 6.7 Merck
  • 6.8 Novartis Pharmaceuticals
  • 6.9 Pfizer
  • 6.10 Roche
  • 6.11 Rare Disease Therapeutics
  • 6.12 Sanofi

List of Figures

  • Figure 1-1: US Orphan Drug Market (US$ Billion), 2013-2018
  • Figure 1-2: US Share in Global Orphan Drug Market, 2013 & 2018
  • Figure 1-3: Biological & Non Biological Orphan Drug Segment (%), 2013 & 2018
  • Figure 1-4: Biological & Non Biological Orphan Drug Market (US$ Billion), 2013-2018
  • Figure 1-5: US - Orphan Drug Pipeline by Phase (%)
  • Figure 1-6: US - Orphan Drug Pipeline by Phase (Number)
  • Figure 1-7: US - No Development Reported in Orphan Drug Pipeline by Phase (%)
  • Figure 1-8: US - No Development Reported in Orphan Drug Pipeline by Phase (Number)
  • Figure 1-9: US - Discontinued Orphan Drug in Pipeline by Phase (%)
  • Figure 1-10: US - Discontinued Orphan Drug in Pipeline by Phase (Number)
  • Figure 1-11: US - Suspended Orphan Drug in Pipeline by Phase (%)
  • Figure 1-12: US - Suspended Orphan Drug in Pipeline by Phase (Number)
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