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市場調査レポート

米国におけるオーファンドラッグ(希少疾病用医薬品)の臨床パイプライン分析

US Orphan Drug Clinical Pipeline Insight 2014

発行 PNS Pharma 商品コード 310696
出版日 ページ情報 英文 2000 Pages
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米国におけるオーファンドラッグ(希少疾病用医薬品)の臨床パイプライン分析 US Orphan Drug Clinical Pipeline Insight 2014
出版日: 2014年08月19日 ページ情報: 英文 2000 Pages
概要

米国におけるオーファンドラッグの研究開発(R&D)は、公共プログラム、政府規制および政策によって促進されています。オーファンドラッグのR&Dイニシアチブには、補助金、研究支援、諸経費の免除、税制上の優遇措置およびオーファンドラッグ市場の独占権などが含まれます。2013年における米国のオーファンドラッグ市場額は490億米ドルと推計されます。同市場は政府支援、高い収益性などの要因により、2018年までに600億米ドルを上回ると予測されています。

当レポートでは、米国のオーファンドラッグ市場について調査し、市場概要、フェーズ・国別の治験分析、上市薬、治験が停止・中止された薬剤のプロファイルおよび競合情勢などをまとめ、お届けいたします。

第1章 米国のオーファンドラッグ市場:イントロダクション

  • 市場概要
  • 臨床パイプライン分析

第2章 米国におけるオーファンドラッグ規制の枠組み

  • オーファンドラッグ指定基準
  • オーファンドラッグの医療費償還政策
  • FDA規制

第3章 米国におけるオーファンドラッグ臨床パイプライン:フェーズ・国別

  • 段階不明
  • 研究
  • 前臨床
  • 臨床
  • フェーズI
  • フェーズI/II
  • フェーズII
  • フェーズII/III
  • フェーズIII
  • 事前登録
  • 登録済み

第4章 上市済みオーファンドラッグ臨床分析:適応症・企業別

第5章 臨床パイプラインにおいて停止・中止されたオーファンドラッグ:フェーズ・適応症・企業別

  • 未開発
  • 中止
  • 市場撤退
  • 停止

第6章 競合情勢

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

図表リスト

目次

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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