市場調査レポート

希用薬(オーファンドラッグ)の世界市場

The World Market for Orphan Drugs (Markets for Oncology, Hematology, Neurology, Endocrine, Metabolic and Other Drugs)

発行 Kalorama Information 商品コード 274431
出版日 ページ情報 英文 400 Pages
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本日の銀行送金レート: 1USD=102.95円で換算しております。
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希用薬(オーファンドラッグ)の世界市場 The World Market for Orphan Drugs (Markets for Oncology, Hematology, Neurology, Endocrine, Metabolic and Other Drugs)
出版日: 2013年06月13日 ページ情報: 英文 400 Pages
概要

希用薬(オーファンドラッグ:Orphan Drugs)とは治療対象となる患者数が少ないことから医薬品メーカーが参入したがらないため、行政機関が補助することで開発が進められる薬剤のこと。ただし、近年は製品数が増加する傾向を見せています。

当レポートでは、この希用薬に注目し、全世界および米国、欧州、日本をはじめとする各国・各地域の市場動向や発展への課題、競合状況などについての最新情報を集め、概略以下の構成でお届けします。

第1章 エグゼクティブサマリー

  • イントロダクション
  • 調査の範囲と方法
  • 市場セグメント
  • 課題と動向
  • 世界市場サマリー
  • 競合企業

第2章 イントロダクション

  • 概要
  • 米国希用薬市場の歴史
  • 医薬品研究開発の概要
  • 医薬品産業の重要規制条件
  • 疾患と希用薬の影響
  • 疾患の解説
  • 血液疾患
  • がん
  • ホルモン障害
  • 感染性疾患
  • 希用薬のコスト面

第3章 米国の希用薬

  • 概要
  • 最近の希用薬指定
  • 企業別に見る希用薬承認

第4章 EUの希用薬

  • 概要
  • 最近の希用薬指定
  • 主要各社の希用薬活動

第5章 日本の希用薬

  • 概要
  • 最近の希用薬指定
  • 主要各社の希用薬活動

第6章 競合企業の動向と業務

  • 買収および協定
  • 米国競合市場分析

第7章 希用薬産業動向

  • イントロダクション
  • 希用薬セグメントを形成する米国規定
  • 1983年の希用薬法
  • 2002年の希少疾患法
  • 連邦食品医薬品局(FDA)希用製品開発部(Office of Orphan Products Development)
  • Humanitarian Use Device Program
  • 希用薬指定の功罪
  • 米国における希用薬指定
  • EUにおける希用薬指定
  • 日本における希用薬指定
  • カナダにおける希用薬指定
  • オーストラリアにおける希用薬指定
  • 健康産業への影響ファクター
  • 老齢化
  • 人口統計
  • 世界の人口動向
  • 世界の65歳超人口
  • 米国の人口動向
  • 米国の65歳超人口
  • 世界の平均寿命動向
  • 世界の出生率動向
  • 世界の医療支出動向
  • 国別景気動向
  • 米国医療の動向
  • 米国医療の課題
  • 米国の景気動向
  • 米国の方式別健康支出

第8章 米国希用薬市場の規模と機会

  • 概要
  • 市場規模および予測
  • 製品分類別市場
  • 腫瘍学
  • サプライヤー別市場シェア
  • 血液学
  • サプライヤー別市場シェア
  • 筋骨格系薬
  • サプライヤー別市場シェア
  • 神経学
  • サプライヤー別市場シェア
  • 内分泌学
  • サプライヤー別市場シェア
  • 代謝系薬
  • サプライヤー別市場シェア
  • その他
  • サプライヤー別市場シェア

第9章 世界市場サマリー:希用薬

  • 概要
  • 市場流通:米国および世界市場

第10章 企業プロフィール

  • イントロダクション
  • 企業別希用薬指定
  • 企業プロフィール

規制官庁および機関

企業一覧

目次
Product Code: KLI5061933

In recent years, there has been a substantial increase in the number of drugs available to treat rare ("orphan") diseases. This comprehensive report, The World Market for Orphan Drugs, looks at this trend and provides estimates for the market opportunity in the U.S., Europe, Japan and globally. With over 400 pages of market analysis and over 120 figures and tables, the report provides in-depth coverage of key competitors and important trends and challenges for makers of orphan drug treatments.

Osteoporosis, arthritis, Alzheimer's, and Parkinson's are major factors affecting quality of life. Mortality from cancer, diabetes, liver and kidney diseases has been slow to change, offering significant areas for research breakthroughs. Cancer, poorly served by traditional chemotherapies, is a major opportunity for biotech firms because the investment compared to return can be favorable: the field has high priority with regulatory authorities who are willing to give it fast track status on the basis of smaller (and therefore cheaper) clinical trials (a few extra months of survival could be enough to win FDA approval); the clinical community is highly concentrated; and the market size is often larger than the approved indication because of a high off-label use (for other cancers). Infectious diseases, the third most common cause of death in many geographic regions, highlight the pressing need for new therapies with novel mechanisms of action to avoid growing issues affecting this industry including a top area of concern-drug resistance.

This report provides estimates for the world market for orphan drugs, as well as the E.U. and Japan markets. Given the size of the U.S. opportunity, the report offers in-depth market data for the U.S. market. Several segments are discussed, including:

  • Oncology
  • Hematology
  • Musculoskeletal
  • Neurology
  • Endocrine
  • Metabolic
  • Others

These segments are the most common areas of development and success for orphan drug developers/marketers which required providing detailed information about these orphan drug market segments.

In the U.S., a drug may be designated an orphan drug if the disease or condition for which it is indicated, affects less than 200,000 Americans. Orphan medicinal products in the E.U. member states are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than 5 in 10,000 persons. In Japan an orphan drug may be designated for a patient population of less than 50,000 and provide for an unmet medical need. Other countries have similar designations. These three regional markets for drugs are examined in separate sections of the report.

Analysis of The “Pure” Orphan Drug Market

Some publicized estimates of orphan drug markets include brand revenue that is not truly related to the orphan status of the drug. In this unique research effort, Kalorama Information separates strictly orphan revenue from total brand revenue where necessary to provide a true picture of the pure orphan drug market opportunity. The world orphan drug market is difficult to determine due to the variances in orphan drug status and requirements by country. Products may be available as an orphan drug on one country and be a traditional marketing approval for another country. In addition, some products gain orphan status, are granted approval and then removed from the orphan drug database at the request of the developer. There are a number of factors involved, including reimbursement by country, regulation by country, disease status by country and general development by country.

For this market research report, market numbers discussed in this report are focused on the global market in brief with in-depth market coverage of the U.S. market. Additionally, there is select coverage for the European Union and Japanese market. All sales are displayed at the manufacturers' level in U.S. dollars.

This report includes orphan drugs. In cases where sales are generated by indications for both orphan indications and non-orphan indications, we have attempted to extract just the orphan portion of sales. The base year is 2012, with forecasts provided for each year through 2017. The market was generated using estimates of individual product sales and compared with market data from a combination of other methods including disease prevalence trends, population trends, pricing trends, government regulations and reimbursements. Both primary and secondary sources were consulted in developing market estimates.

Demographics, Costs, Regulation and Other Trends Examined

The list of trends and factors affecting the orphan drug market remain lengthy. The orphan drug market is affected by both trends that affect the pharmaceutical industry as a whole and trends that specifically affect the orphan drug market:

  • Orphan drug pricing
  • Aging of the population
  • Increasing life expectancy
  • Trends in birth rates
  • Cost effectiveness
  • Regulatory environments
  • Orphan drug incentives
  • Population demographics
  • Product availability by country/region
  • Due to the wide variety of products to receive orphan designations, there are numerous developers with products in this area. Some of the developers that have received an orphan designation have followed through to receive a full product approval but others may not gain additional approvals.

Extensive Company Coverage

The report looks extensively at what major pharmaceutical companies and smaller concerns are doing within this drug category. The report provides company market share for both the US and global markets.

A variety of companies with both designations and designation/approvals are discussed in this report:

  • Abbott Laboratories
  • AbbVie
  • Actelion
  • Amgen
  • Astellas
  • AstraZeneca
  • Baxter
  • Bayer
  • Bio Products Laboratory
  • Biogen IDEC
  • BioMarin Pharmaceuticals
  • Boehringer-Ingelheim
  • Bristol-Myers Squibb
  • Cangene
  • Celgene
  • CSL Behring
  • Depomed
  • Eisai
  • Eli Lilly & Co.
  • EMD Serono
  • Exelixis
  • Gilead Sciences
  • GlaxoSmithKline
  • ImmunoGen
  • Insmed
  • Johnson & Johnson
  • Luitpold Pharmaceuticals
  • Merck & Co
  • Millennium Pharmaceuticals
  • NBI Pharmaceuticals
  • Novartis
  • Novo Nordisk
  • NPS Pharmaceuticals
  • Pfizer
  • Roche
  • Sanofi
  • Seattle Genetics
  • Shire
  • Sigma-Tau
  • Soligenix
  • Spectrum Pharmaceuticals
  • Swedish Orphan Biovitrum
  • TEVA
  • United Therapeutics
  • Valeant Pharmaceuticals
  • XenoPort

Biological therapies have increasingly become the treatment of choice for many diseases. Several hundred million individuals worldwide have been helped by more than 150 therapies. Many of these areas of treatment have huge numbers of individuals affected each year while other areas are small but growing due to the advancement in treatment, superior therapies, and development incentives--such as those that accompany orphan drug designation/approvals.

Telephone interviews and email correspondence were the primary method of gathering information. For the purpose of this study Kalorama Information conducted interviews with more than 130 key industry officials, consultants, health care providers, and government personnel. These sources were the primary basis in gathering information specifically relating to revenue and market share data presented in this report. Specific interviews with leading orphan drug developers' representatives including marketing directors, division managers, and product representatives, were consulted. Secondary sources such as company literature, databases, investment reports, and business journals were used to complement primary information.

Table of Contents

CHAPTER ONE: EXECUTIVE SUMMARY

  • Introduction
  • Scope and Methodology
  • Market Segments
  • Issues and Trends
  • World Market Summary
  • Competitors

CHAPTER TWO: INTRODUCTION

  • Overview
  • History of the U.S. Orphan Drug Market
  • Pharmaceutical Research & Development Overview
  • Pharmaceutical Industry Important Regulatory Terms
  • Diseases and Orphan Drug Impact
  • Disease Descriptions
  • Blood Disorders
  • Cancer
  • Hormone Disorders
  • Infectious Diseases
  • The Cost Side of Orphan Drugs

CHAPTER THREE: U.S. ORPHAN DRUGS

  • Overview
  • Recent Orphan Drug Designations
  • Orphan Drug Approvals by Company
  • Abbott Laboratories
  • Acorda Therapeutics
  • Actelion Pharmaceuticals
  • Aegerion Pharmaceuticals
  • Alexion Pharmaceuticals
  • Allergan, Inc.
  • Amgen
  • Ariad Pharmaceuticals
  • Astellas
  • AstraZeneca
  • Auxilium Pharmaceuticals, Inc.
  • Bausch & Lomb
  • Baxter International
  • Bayer AG
  • Biogen IDEC
  • BioMarin Pharmaceutical, Inc.
  • Boehringer Ingelheim
  • Bristol-Myers Squibb
  • Cangene Corporation
  • Celgene Corporation
  • CSL Behring
  • Eisai Co., Ltd.
  • Eli Lilly & Company
  • EMD Serono
  • Endo Health Solutions, Inc.
  • (previously Endo Pharmaceuticals, Inc.)
  • Exelixis, Inc.
  • Gilead Sciences, Inc.
  • GlaxoSmithKline
  • Grifols, S.A.
  • Hyperion Therapeutics, Inc.
  • Incyte Corporation
  • Ipsen, Inc.
  • Jazz Pharmaceuticals plc
  • Johnson & Johnson
  • KV Pharmaceutical Company
  • Merck & Company
  • Novartis
  • Novo Nordisk
  • NPS Pharmaceuticals
  • Octapharma USA, Inc.
  • Onyx Pharmaceuticals, Inc.
  • Otsuka Pharmaceutical Co., Inc.
  • Pacira Pharmaceuticals, Inc.
  • Pfizer, Inc.
  • Rare Disease Therapeutics, Inc.
  • Reckitt Benckiser Group plc
  • Regeneron Pharmaceuticals, Inc.
  • Roche
  • Salix Pharmaceuticals, Inc.
  • Sanofi
  • Savient Pharmaceuticals, Inc.
  • Seattle Genetics, Inc.
  • Shire Plc
  • Sigma-Tau Pharmaceuticals, Inc.
  • Spectrum Pharmaceuticals
  • Takeda Pharmaceutical Company Ltd.
  • Talon Therapeutics, Inc.
  • Teva Pharmaceuticals
  • United Therapeutics Corporation
  • Valeant Pharmaceuticals International, Inc.
  • Vertex Pharmaceuticals, Inc.
  • ViroPharma, Inc.

CHAPTER FOUR: E.U. ORPHAN DRUGS

  • Overview
  • Recent Orphan Drug Designations
  • Select Company Orphan Drug Activities
  • Actelion Pharmaceuticals
  • Adienne Pharma & Biotech
  • Alexion Pharmaceuticals
  • Amgen
  • Bayer AG
  • BioMarin Pharmaceutical, Inc.
  • Bristol-Myers Squibb
  • Celgene Corporation
  • Eisai Co., Ltd.
  • Gilead Sciences, Inc.
  • GlaxoSmithKline
  • Novartis
  • Orphan Europe S.A.R.L.
  • Pfizer, Inc.
  • Sanofi
  • Shire Plc
  • Vertex Pharmaceuticals, Inc.

CHAPTER FIVE: JAPAN ORPHAN DRUGS

  • Overview
  • Recent Orphan Drug Designations
  • Select Company Orphan Drug Activities
  • Actelion Pharmaceuticals
  • Alexion Pharmaceuticals
  • Bristol-Myers Squibb
  • Celgene Corporation
  • GlaxoSmithKline
  • Johnson & Johnson
  • Merck Serono
  • Mitsubishi Tanabe
  • Novartis
  • Pfizer, Inc.
  • Roche
  • Sanofi

CHAPTER SIX: COMPETITOR TRENDS AND PRACTICES

  • Acquisitions and Agreements
  • U.S. Competitor Market Analysis
  • Amgen
  • Roche
  • Celgene
  • Novartis
  • Abbott
  • Biogen Idec
  • Bayer
  • Eli Lilly
  • Bristol-Myers Squibb
  • Novo Nordisk
  • Sanofi
  • Allergan
  • Other Companies

CHAPTER SEVEN: ORPHAN DRUG INDUSTRY TRENDS

  • Introduction
  • U.S. Regulation Shaping the Orphan Drug Segment
  • Orphan Drug Act of 1983
  • Rare Disease Act of 2002
  • Office of Orphan Products Development
  • Humanitarian Use Device Program
  • Positives and Negatives of the Orphan Drug Designation
  • Orphan Drug Designation in the U.S.
  • Orphan Drug Designation in the E.U.
  • Orphan Drug Designation in Japan
  • Orphan Drug Designation in Canada
  • Orphan Drug Designation in Australia
  • Factors Affecting the Health Industry
  • Aging of the Population
  • Population Statistics
  • Global Population Trends
  • Global Population over 65
  • U.S. Population Trends
  • U.S. Population over 65
  • Global Trends in Life Expectancy
  • Global Trends in Birth Rates
  • Global Healthcare Spending Trends
  • General Economic Trends by Country
  • U.S. Health Industry Trends
  • U.S. Healthcare Challenges
  • Economic Trends in the U.S.
  • U.S. Health Expenditures by Type

CHAPTER EIGHT: U.S. ORPHAN DRUG MARKET SIZE AND OPPORTUNITY

  • Overview
  • Market Size and Forecast
  • Markets by Product Classification
  • Oncology
  • Suppliers Market Share
  • Hematology
  • Suppliers Market Share
  • Musculoskeletal
  • Suppliers Market Share
  • Neurology
  • Suppliers Market Share
  • Endocrinology
  • Suppliers Market Share
  • Metabolic
  • Suppliers Market Share
  • Others
  • Suppliers Market Share

CHAPTER NINE: WORLD MARKET SUMMARY? ORPHAN DRUGS

  • Overview
  • Market Distribution: United States and World Markets

CHAPTER TEN: CORPORATE PROFILES

  • Introduction
  • Orphan Drug Designations by Company: A U.S. Analysis
  • Company Profiles
  • Abbott Laboratories
    • Company Overview
    • Performance Review
  • AbbVie
    • Company Overview
    • Performance Review
  • Actelion
    • Company Overview
    • Performance Review
    • Overview
    • Performance Review
  • Astellas
    • Company Overview
    • Performance Review
  • AstraZeneca
    • Company Overview
    • Performance Review
  • Baxter
    • Company Overview
    • Performance Review
  • Bayer
    • Company Overview
    • Performance Review
  • Bio Products Laboratory
    • Company Overview
  • Biogen Idec
    • Company Overview
    • Performance Review
  • BioMarin
    • Company Overview
    • Performance Review
  • Boehringer Ingelheim
    • Company Overview
    • Performance Review
  • Bristol-Myers Squibb
    • Company Overview
    • Performance Review
  • Cangene
    • Company Overview
    • Performance Review
  • Celgene
    • Company Overview
    • Performance Review
  • CSL Behring
    • Company Overview
    • Performance Review
  • Depomed
    • Company Overview
    • Performance Review
  • Eisai
    • Company Overview
    • Performance Review
  • Eli Lilly
    • Company Overview
    • Performance Review
  • EMD Serono
    • Company Overview
    • Performance Review
  • Exelixis
    • Company Overview
    • Performance Review
  • Gilead Sciences
    • Company Overview
    • Performance Review
  • GlaxoSmithKline
    • Company Overview
    • Performance Review
  • ImmunoGen
    • Company Overview
    • Performance Review
  • Insmed
    • Company Overview
    • Performance Review
  • Johnson & Johnson
    • Company Overview
    • Performance Review
  • LUITPOLD
    • Company Overview
    • Performance Review
  • Merck & Co.
    • Company Overview
    • Performance Review
  • Millennium Pharmaceuticals
    • Company Overview
    • Performance Review
  • NBI Pharmaceuticals
    • Company Overview
  • Novartis
    • Company Overview
    • Performance Review
  • Novo Nordisk
    • Company Overview
    • Performance Review
  • NPS Pharmaceuticals
    • Company Overview
    • Performance Review
  • Pfizer
    • Company Overview
    • Performance Review
  • Roche
    • Company Overview
    • Performance Review
  • Sanofi
    • Company Overview
    • Performance Review
  • Seattle Genetics
    • Company Overview
    • Performance Review
  • Shire
    • Company Overview
    • Performance Review
  • Sigma Tau
    • Company Overview
    • Performance Review
  • Soligenix
    • Company Overview
    • Performance Review
  • Spectrum
    • Company Overview
    • Performance Review
  • Swedish Orphan Biovitrum
    • Company Overview
    • Performance Review
  • TEVA
    • Company Overview
    • Performance Review
  • United Therapeutics
    • Company Overview
    • Performance Review
  • Valeant
    • Company Overview
    • Performance Review
  • XenoPort
    • Company Overview
    • Performance Review

REGULATORY AUTHORITIES AND ORGANIZATIONS

LIST OF COMPANIES

LIST OF EXHIBITS

CHAPTER ONE: EXECUTIVE SUMMARY

  • Table 1-1: World Orphan Drug Market 2007-2017
  • Figure 1-1: World Orphan Drug Market

CHAPTER TWO: INTRODUCTION

  • Table 2-1: Phases of Clinical Drug Development
  • Table 2-2: Total Cancer Incidence by Sex and Country
  • Figure 2-1: Total Cancer Incidence by Sex and Country
  • Table 2-3: Average Annual Drug Costs to Patients, Selected Drugs
  • Figure 2-2: Average Annual Drug Costs to Patients, Selected Drugs

CHAPTER THREE: U.S. ORPHAN DRUGS

  • Table 3-1: Orphan Drug Designations Granted and Approvals by the FDA 1983-2013*
  • Figure 3-1: Orphan Drug Designations Granted and Approvals by the FDA 1983-2013*
  • Table 3-2: 12-Months Orphan Drug Designations Granted by FDA (May 1, 2012 - May 1, 2013)
  • Figure -3-2: May 2012 - May 2013 FDA Orphan Drug Designations by Target Classification
  • Table 3-3: Abbott Laboratories' Orphan Drug Approvals Granted by FDA
  • Table 3-4: Acorda Therapeutics' Orphan Drug Approvals Granted by FDA
  • Table 3-5: Actelion Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-6: Aegerion Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-7: Alexion Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-8: Allergan's Orphan Drug Approvals Granted by FDA
  • Table 3-9: Amgen's Orphan Drug Approvals Granted by FDA
  • Table 3-9 (continued): Amgen's Orphan Drug Approvals Granted by FDA
  • Table 3-10: Ariad Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-11: Astellas' Orphan Drug Approvals Granted by FDA
  • Table 3-12: AstraZeneca's Orphan Drug Approvals Granted by FDA
  • Table 3-13: MedImmune's Orphan Drug Approvals Granted by FDA
  • Table 3-14: Auxilium Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-15: Bausch & Lomb's Orphan Drug Approvals Granted by FDA
  • Table 3-16: Baxter International's Orphan Drug Approvals Granted by FDA
  • Table 3-17: Bayer's Orphan Drug Approvals Granted by FDA
  • Table 3-18: Biogen IDEC's Orphan Drug Approvals Granted by FDA
  • Table 3-19: BioMarin Pharmaceutical's Orphan Drug Approvals Granted by FDA
  • Table 3-20: Boehringer Ingelheim's Orphan Drug Approvals Granted by FDA
  • Table 3-21: Bristol-Myers's Orphan Drug Approvals Granted by FDA
  • Table 3-22: Cangene's Orphan Drug Approvals Granted by FDA
  • Table 3-23: Celgene Corporation's Orphan Drug Approvals Granted by FDA
  • Table 3-24: CSL Behring's Orphan Drug Approvals Granted by FDA
  • Table 3-25: Eisai's Orphan Drug Approvals Granted by FDA
  • Table 3-26: Eli Lilly's Orphan Drug Approvals Granted by FDA
  • Table 3-27: EMD Serono's Orphan Drug Approvals Granted by FDA
  • Table 3-28: Endo Health Solutions' Orphan Drug Approvals Granted by FDA
  • Table 3-29: Exelixis' Orphan Drug Approvals Granted by FDA
  • Table 3-30: Gilead Sciences' Orphan Drug Approvals Granted by FDA
  • Table 3-31: GlaxoSmithKline's Orphan Drug Approvals Granted by FDA
  • Table 3-32: Grifols' Orphan Drug Approvals Granted by FDA
  • Table 3-33: Hyperion Therapeutics' Orphan Drug Approvals Granted by FDA
  • Table 3-34: Incyte Corporation's Orphan Drug Approvals Granted by FDA
  • Table 3-35: Ipsen's Orphan Drug Approvals Granted by FDA
  • Table 3-36: Jazz Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-37: Johnson & Johnson Pharmaceutical's Orphan Drug Approvals Granted by FDA
  • Table 3-38: Alza's Orphan Drug Approvals Granted by FDA
  • Table 3-39: Janssen's Orphan Drug Approvals Granted by FDA
  • Table 3-40: KV Pharmaceutical's Orphan Drug Approvals Granted by FDA
  • Table 3-41: Merck & Co.'s Orphan Drug Approvals Granted by FDA
  • Table 3-41 (continued): Merck & Co.'s Orphan Drug Approvals Granted by FDA
  • Table 3-42: Novartis' Orphan Drug Approvals Granted by FDA
  • Table 3-43: Alcon's Orphan Drug Approvals Granted by FDA
  • Table 3-44: Novo Nordisk's Orphan Drug Approvals Granted by FDA
  • Table 3-45: NPS Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-46: Octapharma's Orphan Drug Approvals Granted by FDA
  • Table 3-47: Onyx Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-48: Otsuka Pharmaceutical's Orphan Drug Approvals Granted by FDA
  • Table 3-49: Pacira Pharmaceutical's Orphan Drug Approvals Granted by FDA
  • Table 3-49: Pfizer's Orphan Drug Approvals Granted by FDA
  • Table 3-49 (continued): Pfizer's Orphan Drug Approvals Granted by FDA
  • Table 3-50: Rare Disease Therapeutics' Orphan Drug Approvals Granted by FDA
  • Table 3-51: Reckitt Benckiser's Orphan Drug Approvals Granted by FDA
  • Table 3-52: Regeneron Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-53: Roche's Orphan Drug Approvals Granted by FDA
  • Table 3-54: Genentech's Orphan Drug Approvals Granted by FDA
  • Table 3-55: Salix Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-56: Sanofi's Orphan Drug Approvals Granted by FDA
  • Table 3-57: Genzyme's Orphan Drug Approvals Granted by FDA
  • Table 3-58: Savient Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-59: Seattle Genetics' Orphan Drug Approvals Granted by FDA
  • Table 3-60: Shire's Orphan Drug Approvals Granted by FDA
  • Table 3-61: Sigma Tau's Orphan Drug Approvals Granted by FDA
  • Table 3-62: Spectrum Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-63: Millennium Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-64: Talon Therapeutics' Orphan Drug Approvals Granted by FDA
  • Table 3-65: Teva's Orphan Drug Approvals Granted by FDA
  • Table 3-66: Cephalon's Orphan Drug Approvals Granted by FDA
  • Table 3-67: United Therapeutics' Orphan Drug Approvals Granted by FDA
  • Table 3-68: Valeant Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-69: Vertex Pharmaceuticals' Orphan Drug Approvals Granted by FDA
  • Table 3-70: ViroPharma's Orphan Drug Approvals Granted by FDA

CHAPTER FOUR: E.U. ORPHAN DRUGS

  • Table 4-1: Orphan Drug Designations and Approvals Granted by the EMA 2000-2013*
  • Figure 4-1: Orphan Drug Designations and Approvals Granted by the EMA 2000-2013*
  • Figure 4-2: Orphan Drugs Approved by EMA by Target Category
  • Table 4-2: Orphan Drug Designations Granted by EMA (January 1, 2012 - December 31, 2012)
  • Figure -4-3: 2012 European Union Orphan Drug Designations by Target Classification
  • Table 4-3: Orphan Drug Designations Granted by EMA (2013, January - April)

CHAPTER FIVE: JAPAN ORPHAN DRUGS

  • Table 5-1: Orphan Drug Designations and Approvals Granted by the MHLW 1993-2012*
  • Figure 5-1: Orphan Drug Designations and Approvals Granted by the MHLW 1993-2012*
  • Figure 5-2: Orphan Drugs Approved by MHLW by Target Category
  • Table 5-2: Orphan Drug Designations Granted by MHLW (January 1, 2012 - November 14, 2012)
  • Figure 5-3: 2012 Ministry of Health, Labour, and Welfare Orphan Drug Designations by Target Classification

CHAPTER SIX: COMPETITOR TRENDS AND PRACTICES

  • Figure 6-1: Amgen's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-2: Roche's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-3: Celgene's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-4: Novartis' Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-5: Abbott's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-6: Biogen Idec' Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-7: Bayer's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-8: Eli Lilly's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-9: Bristol-Myers' Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-10: Novo Nordisk's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-11: Sanofi's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Figure 6-12: Allergan's Orphan Drug Sales as a Percentage of Total Prescription Drug Sales, United States
  • Table 6-1: Competitor Analysis, U.S. Orphan Drug Market, 2012
  • Figure 6-13: Competitor Analysis, U.S. Orphan Drug Market, 2012

CHAPTER SEVEN: ORPHAN DRUG INDUSTRY TRENDS

  • Table 7-1: The Global Population, 1980-2050
  • Figure 7-1: The Global Population, 1980-2050
  • Table 7-2: Global Population Trends Age 65+, 2000-2050
  • Figure 7-2: Global Population Trends Age 65+, 2000-2050
  • Table 7-3: The U.S. Population, 1980-2050
  • Figure 7-3: The U.S. Population, 1980-2050
  • Table 7-4: Percent U.S. Population Over Age 65 by Year, 2000-2050
  • Figure 7-4: United States Population Trend Age 65+, 2000-2050
  • Table 7-5: Average Life Expectancy in Years by Country 1980 and 2012
  • Figure 7-5: Average Life Expectancy in Years by Country 1980 and 2012
  • Table 7-6: Crude Birth Rate per 1,000 Persons 2000, 2005, 2010, Estimated 2012 and Projected 2020
  • Figure 7-6: Crude Birth Rate per 1,000 Persons 2000, 2005, 2010, Estimated 2012 and Projected 2020
  • Table 7-7: Total Healthcare Expenditures as a Percent of GDP by Country 1990, 2000 and 2010209
  • Figure 7-7: Total Healthcare Expenditures as a Percent of GDP by Country 1990, 2000 and 2010210
  • Table 7-8: Average Annual Growth of GDP by Country 2007-2011
  • Table 7-9: National Healthcare Expenditures in the United States 1960-2020
  • Table 7-10: National Health Expenditure Amounts, by Type of Expenditure: Calendar Years 2015- 2021

CHAPTER EIGHT: U.S. ORPHAN DRUG MARKET SIZE AND OPPORTUNITY

  • Table 8-1: The U.S. Orphan Drug Market 2007-2017
  • Figure 8-1: U.S. Orphan Drug Market
  • Figure 8-2: Distribution of Sales by Product Classification for Approved U.S. Orphan Drugs, 2012
  • Table 8-2: The U.S. Market for Orphan Drugs with Oncology Targets 2007-2017
  • Figure 8-3: The U.S. Market for Orphan Drugs with Oncology Targets 2007-2017
  • Figure 8-4: Distribution of Sales for Orphan Drugs with Oncology Targets by Supplier, 2012
  • Table 8-3: The U.S. Market for Orphan Drugs with Hematology Targets 2007-2017
  • Figure 8-5: The U.S. Market for Orphan Drugs with Hematology Targets 2007-2017
  • Figure 8-6: Distribution of Sales for Orphan Drugs with Hematology Targets by Supplier, 2012
  • Table 8-4: The U.S. Market for Orphan Drugs with Musculoskeletal Targets 2007-2017
  • Figure 8-7: The U.S. Market for Orphan Drugs with Musculoskeletal Targets 2007-2017
  • Figure 8-8: Distribution of Sales for Orphan Drugs with Musculoskeletal Targets by Supplier, 2012
  • Table 8-5: The U.S. Market for Orphan Drugs with Neurology Targets 2007-2017
  • Figure 8-9: The U.S. Market for Orphan Drugs with Neurology Targets 2007-2017
  • Figure 8-10: Distribution of Sales for Orphan Drugs with Neurology Targets by Supplier, 2012
  • Table 8-6: The U.S. Market for Orphan Drugs with Endocrinology Targets 2007-2017
  • Figure 8-11: The U.S. Market for Orphan Drugs with Endocrinology Targets 2007-2017
  • Figure 8-12: Distribution of Sales for Orphan Drugs with Endocrinology Targets by Supplier, 2012
  • Table 8-7: The U.S. Market for Orphan Drugs with Metabolic Targets 2007-2017
  • Figure 8-13: The U.S. Market for Orphan Drugs with Metabolic Targets 2007-2017
  • Figure 8-14: Distribution of Sales for Orphan Drugs with Metabolic Targets by Supplier, 2012
  • Table 8-8: The U.S. Market for Orphan Drugs with Other Disease Targets 2007-2017
  • Figure 8-15: The U.S. Market for Orphan Drugs with Other Disease Targets 2007-2017
  • Figure 8-16: Distribution of Sales for Orphan Drugs with Other Disease Targets by Supplier, 2012252

CHAPTER NINE: WORLD MARKET SUMMARY? ORPHAN DRUGS

  • Table 9-1: World Orphan Drug Market 2007-2017
  • Figure 9-1: World Orphan Drug Market .Share By Competitor
  • Table 9-2: World Orphan Drug Market: Distribution by United States and Rest of World, 2007- 2017
  • Figure 9-2: World Orphan Drug Market: Distribution by United States and Rest of World, 2007- 2017
  • Figure 9-3: Orphan Drugs: World Markets by Region, 2012

CORPORATE PROFILES

  • Figure 10-1: Orphan Drug Designations (FDA) by Company
  • Figure 10-2: Abbott's Financial Performance, 2010-2012
  • Figure 10-3: AbbVie's Financial Performance, 2010-2012
  • Figure 10-3: Actelion's Financial Performance, 2010-2012
  • Figure 10-4: Amgen's Financial Performance, 2010-2012
  • Figure 10-5: Astellas' Financial Performance, 2010-2012
  • Figure 10-6: AstraZeneca's Financial Performance, 2010-2012
  • Figure 10-7: Baxter's Financial Performance, 2010-2012
  • Figure 10-7: Bayer's Financial Performance, 2010-2012
  • Figure 10-8: Biogen's Financial Performance, 2010-2012
  • Figure 10-9: BioMarin's Financial Performance, 2010-2012
  • Figure 10-10: Boehringer Ingelheim's Financial Performance, 2010-2012
  • Figure 10-11: Bristol-Myers Squibb's Financial Performance, 2010-2012
  • Figure 10-12: Cangene's Financial Performance, 2010-2012
  • Figure 10-13: Celgene's Financial Performance, 2010-2012
  • Figure 10-14: CSL Limited's Financial Performance, 2010-2012
  • Figure 10-15: Depomed's Financial Performance, 2010-2012
  • Figure 10-16: Eisai Company's Financial Performance, 2010-2012
  • Figure 10-17: Eli Lilly's Financial Performance, 2010-2012
  • Figure 10-18: Merck KGaA's Financial Performance, 2010-2012
  • Figure 10-19: Exelixis' Financial Performance, 2010-2012
  • Figure 10-20: Gilead's Financial Performance, 2010-2012
  • Figure 10-21: GlaxoSmithKline's Financial Performance, 2010-2012
  • Figure 10-22: ImmunoGen's Financial Performance, 2010-2012
  • Figure 10-23: Insmeds's Financial Performance, 2010-2012
  • Figure 10-24: Johnson & Johnson's Financial Performance, 2010-2012
  • Figure 10-25: Daiichi Sankyo's Financial Performance, 2010-2012
  • Figure 10-26: Merck & Co.'s Financial Performance, 2010-2012
  • Figure 10-27: Takeda's Financial Performance, 2010-2012
  • Figure 10-28: Novartis' Financial Performance, 2010-2012
  • Figure 10-29: Novo Nordisk's Financial Performance, 2010-2012
  • Figure 10-30: NPS Pharmaceutical's Financial Performance, 2010-2012
  • Figure 10-31: Pfizer's Financial Performance, 2010-2012
  • Figure 10-32: Roche's Financial Performance, 2010-2012
  • Figure 10-33: Sanofi's Financial Performance, 2010-2012
  • Figure 10-34: Seattle Genetics' Financial Performance, 2010-2012
  • Figure 10-35: Shire's Financial Performance, 2010-2012
  • Figure 10-36: Soligenix' Financial Performance, 2010-2012
  • Figure 10-37: Spectrum's Financial Performance, 2010-2012
  • Figure 10-38: Swedish Orphan Biovitrum's Financial Performance, 2010-2012
  • Figure 10-39: TEVA's Financial Performance, 2010-2012
  • Figure 10-40: United Therapeutics' Financial Performance, 2010-2012
  • Figure 10-41: Valeant's Financial Performance, 2010-2012
  • Figure 10-42: XenoPort's Financial Performance, 2010-2012

REGULATORY AUTHORITIES AND ORGANIZATIONS

LIST OF COMPANIES

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