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市場調查報告書

美國的孤兒藥市場預測

US Orphan Drug Market Outlook 2018

出版商 KuicK Research 商品編碼 302598
出版日期 內容資訊 英文 160 Pages
商品交期: 最快1-2個工作天內
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美國的孤兒藥市場預測 US Orphan Drug Market Outlook 2018
出版日期: 2014年02月01日 內容資訊: 英文 160 Pages
簡介

美國第一次為了管理孤兒藥而成立特別法律。這提高了孤兒藥的關注。再加上由於FDA進行促進對製藥公司孤兒藥開發的配合措施,支持了該市場。這成為帶動美國的孤兒藥市場成長的主要原因之一。

本報告提供美國的孤兒藥市場概要和孤兒藥的指定標準、美國市場給付政策與法律規章、開發平台趨勢、應解決的課題、參與企業的競爭環境彙整,為您概述為以下內容。

第1章 孤兒藥的簡介

  • 孤兒藥定義
  • 孤兒藥的日益流行

第2章 為何從非孤兒藥轉移到孤兒藥

  • 疲乏的產品平台
  • 孤兒藥的盈利
  • 研究開發投資的增加
  • 經濟獎勵所扮演的角色
  • 專利保護與市場壟斷性

第3章 美國的孤兒藥市場預測

  • 孤兒藥的指定標準
  • 市場概要
  • 孤兒藥的給付政策

第4章 美國的孤兒藥法律規章架構

  • 書面的推薦要求的內容和格式
  • 書面的推薦授予及拒絕的出示
  • 孤兒藥指定相關要求內容和格式
  • 孤兒藥現狀與美國代理店對海外贊助商的檢驗
  • 孤兒藥的指定和已經過核准藥物的指定要求時機
  • 關於不足的文件和孤兒藥指定的授予
  • 孤兒藥指定的拒絕
  • 對於孤兒藥指定的所有權變更
  • 孤兒藥指定的公佈和取消
  • 孤兒藥指定持有人的年度報告
  • 孤兒藥特別認證相關範圍和FDA的認識
  • 調查通訊協定與資訊的取得

第5章 各相、適應症之孤兒藥開發平台

第6章 美國的已上市孤兒藥:品牌名與適應症

第7章 應解決的課題

  • 初期投資的高度
  • 臨床實驗的門檻
  • 法律規章的門檻
  • 各種給付政策

第8章 競爭環境

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

圖表

目錄

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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