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市場調査レポート

米国のオーファンドラッグ市場見通し

US Orphan Drug Market Outlook 2018

発行 KuicK Research 商品コード 302598
出版日 ページ情報 英文 160 Pages
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米国のオーファンドラッグ市場見通し US Orphan Drug Market Outlook 2018
出版日: 2014年02月01日 ページ情報: 英文 160 Pages
概要

オーファンドラッグを管理するための特別な法律を成立させたのは米国が初です。これがオーファンドラッグへの注目を高めることとなりました。さらにFDAが製薬会社に対してオーファンドラッグの開発を促す取組みを行なうことで、同市場を支えています。これが米国のオーファンドラッグ市場の成長を牽引する主な要因のひとつとなっています。

当レポートでは、米国のオーファンドラッグ市場の概要とオーファンドラッグの指定基準、米国市場の償還政策と法規制、パイプライン動向、解決すべき課題とともに、参入する企業の競合環境をまとめ、概略下記の構成で取り上げております。

第1章 オーファンドラッグのイントロダクション

  • オーファンドラッグの定義
  • オーファンドラッグの人気上昇

第2章 なぜ非オーファンからオーファンドラッグに移行するのか

  • 疲弊する製品パイプライン
  • オーファンドラッグの収益性
  • 研究開発投資の増加
  • 経済的インセンティブの役割
  • 特許保護と市場独占性

第3章 米国のオーファンドラッグ市場見通し

  • オーファンドラッグの指定基準
  • 市場概要
  • オーファンドラッグの償還政策

第4章 米国のオーファンドラッグ法規制枠組み

  • 書面による推奨要求の内容と様式
  • 書面による推奨授与および拒否の提示
  • オーファンドラッグ指定に関する要求内容と様式
  • オーファンドラッグステータスと海外スポンサーに対する在米代理店の検証
  • オーファンドラッグの指定と認可済み薬剤の指定要求タイミング
  • 不備に関する書面とオーファンドラッグの指定の授与
  • オーファンドラッグ指定の拒否
  • オーファンドラッグ指定に対する所有権の変更
  • オーファンドラッグ指定の公表と取消し
  • オーファンドラッグ指定ホルダーの年次レポート
  • オーファンドラッグ特別認可に関する範囲とFDAの認識
  • 調査プロトコルと情報の入手性

第5章 相、適応別オーファンドラッグパイプライン

第6章 米国における上市済みオーファンドラッグ:ブランド名と適応

第7章 解決すべき課題

  • 初期投資の高さ
  • 治験のハードル
  • 法規制のハードル
  • 様々な償還政策

第8章 競合環境

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

図表

目次

In the largest market for orphan drugs, USA, there was a shortage of adequate therapies for treating many rare diseases. These therapies were not developed as companies did not expect these drugs to be highly profitable. Hence there was a lack of interest and thus investment on the part of pharma companies in the USA. Therefore, the FDA introduced incentives for developing such drugs. This step taken by the FDA was successful in creating a thriving market for orphan drugs.

It was in the USA first that a special law exclusively for governing orphan drugs was framed in the form of the Orphan Drug Act of 1983. This led to an increase in the popularity of orphan drugs. The FDA also has been continuously increasing its efforts to support this market by providing significant financial and non-financial incentives to the pharmaceutical companies to attract them. This has been one of the major drivers of growth for the US orphan drugs market.

The orphan drugs enjoy significant competitive advantages in the market 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.

A scenario where orphan drugs would be denied coverage is highly difficult to visualize. However it is most likely that the payer scrutiny would increase with new products entering the market and budgets contracting in a weakening economic environment. The plans would be based on costs and would also include payer resources, philosophies, and available benefit design options, as all these factors could affect patient access. A deep and clear understanding of the clinical and economic value of the drugs will play an increasingly important role in decision-making.

"US Orphan Drug Market Outlook 2018" research report by KuicK Research comprehensive insight on following developments related to US orphan drug market:

  • US Orphan Drug Market Overview
  • Orphan Drug Designation Criteria
  • Market Specific Reimbursement Policy & Regulatory Framework
  • US Orphan Drug Pipeline by Phase, Indication & Originator
  • Marketed Orphan drug List by Indication & Brand Name
  • Key Issue to be Resolved
  • Competitive Landscape

Table of Contents

1. Introduction to Orphan Drugs

  • 1.1. Orphan Drug Defined
  • 1.2. Rising Popularity of Orphan Drugs

2. Why Shift from Non-Orphan to Orphan Drugs?

  • 2.1. Exhausting Product Pipelines
  • 2.2. Profitability of Orphan Drugs
  • 2.3. Increasing R&D Investment
  • 2.4. Role of Economic Incentives
  • 2.5. Patent Protection & Market Exclusivity

3. US Orphan Drug Market Outlook

  • 3.1. Orphan Drug Designation Criteria
  • 3.2. Market Overview
  • 3.3. Reimbursement Policy for Orphan Drugs

4. US Orphan Drug Regulatory Framework

  • 4.1. Content & Format of a Request for Written Recommendations
  • 4.2. Provision for Granting & Refusing Written Recommendations
  • 4.3. Content & Format of a Request for Orphan Drug Designation
  • 4.4. Verification of Orphan Drug Status & Resident Agent for Foreign Sponsor
  • 4.5. Timing of Requests for Orphan Drug Designation & Designation Of Already Approved Drugs
  • 4.6. Deficiency Letters &Granting Orphan Drug Designation
  • 4.7. Refusal to Grant Orphan Drug Designation
  • 4.8. Amendment & Change in Ownership to Orphan Drug Designation
  • 4.9. Publication & Revocation of Orphan Drug Designations
  • 4.10. Annual Reports of Holder of Orphan Drug Designation
  • 4.11. Scope & FDA Recognition of Orphan Drug Exclusive Approval
  • 4.12. Protocols for Investigations & Availability of Information

5. US Orphan Drug Pipeline Insight by Phase & Indication

  • 5.1. Research
  • 5.2. Preclinical
  • 5.3. Phase I
  • 5.4. Phase I/II
  • 5.5. Phase II
  • 5.6. Phase II/III
  • 5.7. Phase III
  • 5.8. Preregistration
  • 5.9. Registered

6. Marketed Orphan Drugs in US: Brand Name & Indication

7. Key Issue to be Resolved

  • 7.1. High Initial Investment
  • 7.2. Clinical Study Hurdles
  • 7.3. Regulatory Hurdles
  • 7.4. Different Reimbursement Policy

8. Competitive Landscape

  • 8.1. Pfizer
  • 8.2. Roche (Genentech)
  • 8.3. Sanofi
  • 8.4. Rare Disease Therapeutics
  • 8.5. Glaxosmithkline
  • 8.6. Merck
  • 8.7. Novartis Pharmaceuticals
  • 8.8. Alexion
  • 8.9. Celgene
  • 8.10. Biogen Idec
  • 8.11. Eli Lilly (Genzyme Corporation)
  • 8.12. Bristol Myers Squibb
  • List of Figures

    • Figure 2-1: Orphan v/s Non-Orphan Drugs Phase II to Launch Clinical Development Time
    • Figure 2-2: Probability of Regulatory Success of Orphan v/s Non-Orphan Drugs
    • Figure 3-1: US Orphan Drug Market (US$ Billion), 2012-2018
    • Figure 3-2: US Share in Global Orphan Drug Market, 2012 & 2018
    • Figure 3-3: Biological & Non Biological Orphan Drug Segment (%), 2012 & 2018
    • Figure 3-4: Biological & Non Biological Orphan Drug Market (US$ Billion), 2012-2018
    • Figure 5-1: US- Orphan Drug Development by Clinical Phase (%), 2014
    • Figure 5-2: US - Number of Orphan Drug by Clinical Phase, 2014
    • Figure 5-3: US - Number of Suspended & Discontinued Number of Orphan Drug, 2014
    • Figure 5-4: US - Number of Discontinued Orphan Drug by Clinical Phase, 2014
    • Figure 5-5: US - Number of Suspended Orphan Drug by Clinical Phase, 2014
    • Figure 5-6: US - Number of No Development Reported Orphan Drug by Clinical Phase, 2014
    • Figure 7-1: Orphan Drug Regulatory Hurdles
    • Figure 8-1: Genzyme Drug Pipeline Chart

    List of Tables

    • Table 2-1: Drugs Patent Expiry in 2014
    • Table 2-2: Drugs Patent Expiry in 2015
    • Table 2-3: Drugs Patent Expiry in 2016
    • Table 2-4: Orphan Drugs in the US and their Cost
    • Table 2-5: Orphan Drugs in the EU and their Cost
    • Table 2-6: Incentives for the Development of Orphan Drugs
    • Table 2-7: Orphan Drug Incentives by Country
    • Table 5-1: US - Orphan Drugs in Research Phase
    • Table 5-2: US - Orphan Drugs in Preclinical Phase
    • Table 5-3: US - Orphan Drugs in Clinical Phase I
    • Table 5-4: US - Orphan Drugs in Clinical Phase I/II
    • Table 5-5: US - Orphan Drugs in Clinical Phase II
    • Table 5-6: US - Orphan Drugs in Clinical Phase II/III
    • Table 5-7: US - Orphan Drugs in Clinical Phase III
    • Table 5-8: US - Orphan Drugs in Preregistration Phase
    • Table 5-9: US - Registered Orphan Drugs
    • Table 6-1: US - Marketed Orphan Drugs by Indication & Brand Name
    • Table 7-1: Orphan Drug Clinical Study Hurdles
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