全球孤兒藥市場 是由出版商BCC Research在2010年05月所出版的。
這份英文市場調查報告書包含205 Pages 價格從美金4850起跳。
本報告書提供了孤兒藥的的概要與2008年、2009年的數據資料，以及2014年之前的市場成長預測，並提供主要藥劑的詳細動態、與具有前瞻性的治療領域等介紹，另外還彙整了市場上主要企業的簡介與市佔率，內容概要摘記如下。
第1章 導論
第2章 摘要
第3章 概要
- 孤兒藥的定義
- 孤兒藥規範的歷史
- 孤兒藥研發初期的法律規範
- 孤兒藥的研發動機
- 研發動機的需求
- 美國FDA的核准（1983∼2009年）
- 孤兒藥應用領域的擴大
- 孤兒藥的收益性
- 市場佔有率
- 藥價設定缺乏法規標準
- 藥品的標示外使用
- 有效運用ODA効果的課題
- 市場獨佔性與專利保護
- 市場規模與主要企業 其他
第4章 全球的業界結構
- 全球及地區的孤兒藥銷售
- 孤兒藥市場上的企業分類
- 各類贊助商的動態
- 各類贊助商的孤兒藥收益
- 各家企業的孤兒藥認可
- 大型製藥公司與生物技術企業 其他
第5章 產品
- 美國FDA核准的孤兒藥（1983∼2009年）
- 1983年以後孤兒藥的指定與核准
- 2009年銷售額超過2億美金的藥劑
- 有前瞻性的孤兒藥之分析 其他
第6章 應用
- 癌症
- 白血病
- 淋巴瘤
- 多發性骨髓瘤
- 肢端肥大症
- 癌相關症狀 其他
第7章 法律規範
- 美國的法律規範
- RARE DISEASES ACT AND RARE DISEASES ORPHAN PRODUCT DEVELOPMENT ACT OF 2002
- 美國研發孤兒藥的動機
- 孤兒藥法帶給製藥公司與生物技術業界的影響
- 日本
- 澳洲
- 新加坡
- 其他國家與地區
- 法律規範比較
- 孤兒藥的高價問題
- 請求修改孤兒藥法規的意見
- 生物技術藥帶給孤兒藥領域的影響
第8章 技術的趨勢分析
- 專利分析
- 與孤兒藥相關的主要專利
- 基於孤兒藥核准的技術趨勢分析
- 技術趨勢分析的結論
第9章 企業簡介
詞彙
Abstract
THIS REPORT CONTAINS
- An overview of orphan drug criteria, the history of the Orphan Drug Act
and its influence and advantages inherent in orphan drug designation
- Global market projections, with data for 2008, estimates for 2009, and
forecasts for the compound annual growth rate (CAGR) through 2014
- Detailed discussion of successful orphan products and a discussion of
specific rare diseases for which orphan drugs exist
- Analyses of the most promising therapeutic areas in the industry and
leading orphan drugs in these therapeutic areas
- Profiles of key players in the market and their market shares
INTRODUCTION
STUDY GOAL AND OBJECTIVES
The orphan drug industry has come a long way since its implementation, leaving
many noticeable benchmarks along the way as the U.S. Orphan Drug Act (ODA),
which was implemented in 1983, entered its 26th year in 2009. The purpose of
this report is to analyze the orphan drug industry and provide market,
technology, and revenue forecasts for the period 2009 to 2014. The report also
analyzes the influence of the orphan drug industry in the development of the
biotechnology industry.
This BCC report provides an in-depth look at orphan drugs that had sales of
more than $200 million in 2009. Future growth prospects of these drugs as well
as the therapeutic areas of these drugs in general have been analyzed based on
the market dynamics and regulatory factors. Life stories of blockbuster orphan
drugs are tracked and analyzed in detail, recognizing various factors that
played key roles in their success. Regional analysis of the industry in terms
of current and future trends of the orphan drug market in important regions
has been presented. The report also provides revenues of leading orphan
products, identifies key market players with significant market shares,
analyzes current and future strategies of these companies that will have an
impact in the market, and, finally, analyzes the rare diseases that are being
addressed by the industry and the broad therapeutic areas that these diseases
represent and their future growth trends.
This report also analyzes the current and possible future legislation
affecting development and marketing of orphan drugs in the U.S. and other
countries and examines the influence of the Orphan Drug Act in the development
of the biotech industry. The impact of developments in pharmacogenomics in the
orphan drug industry is also analyzed. The rising influence of ODA in business
strategy development of pharmaceutical companies, which is driving the
emergence of innovative business strategies and approaches, is also analyzed
in detail. Issues such as the impact of market exclusivity in drug pricing,
affordability, and healthcare insurance are also analyzed. Finally, technology
trends analysis has been presented in the report, identifying how the most
prevalent technologies in the market have changed over the past decade with
the advent of biotech companies and future growth trends.
REASONS FOR DOING THE STUDY
BCC published comprehensive reports on the orphan drug industry in 2004, and
in 2007. The emerging trends identified in 2007 have become more established
during the past 3 years. Moreover, new trends are emerging as big pharma
realizes the failing of blockbuster business model. The role of biotechnology
industry in the pharmaceutical market has become more prominent, which is
reflected in the orphan drug sector as well.
The changing regulatory landscape of the industry and its effect in driving
the growth is obvious and has been pointed out in the earlier BCC report,
PHM038B, published in 2007. During the past 3 years, the role of regulators in
deciding the future of the sector has become more prominent. Globally,
awareness about rare diseases has increased, while the importance of ODA has
received rising recognition. Following the example of the U.S. Food and Drug
Administration' s (FDA' s) Orphan Drug Act, similar legislation - such as
Singapore' s Orphan Drug Exemption to the Medicines Act and Japan' s Orphan Drug
Amendment to the Pharmaceutical Affairs Law - has been developed by many
other countries. This has opened new markets for companies involved in orphan
drug development. The number of orphan drugs that have become blockbusters has
also gone up. In 2006, there were 19 orphan drugs with sales of more than $1
billion. In 2009, this increased to 27.
The change in the attitude of the pharmaceutical industry towards orphan drugs
is becoming more prominent. Business deals involving big pharmaceutical
companies and small companies with a pipeline of orphan drug designations have
increased lately, indicating the rising importance of orphan-drug-based
business strategies in the pharmaceutical industry. Such new trends are
changing the landscape of the entire pharmaceutical industry. This report
analyzes the current and emerging trends in the market. The report also
analyzes affordability issues. Rising prices of orphan drugs and the
reluctance of insurance agencies to cover orphan drugs that are priced
extremely high are two issues being taken seriously by governments and patient
support groups. Various business models are being suggested and being designed
to tackle these issues. This report analyzes these rising business models in
detail.
INTENDED AUDIENCE
This study provides insight on successful regulatory strategies that helped
orphan drugs become blockbusters. Valuable insights on various business models
that take advantage of ODA incentives are also provided. In addition, emerging
regulatory strategies in key markets for improving the affordability of orphan
drugs by patients are included. The key therapeutic categories that are
getting high attention from the developers and the rare indications within
these therapeutic areas that have benefited from ODA are identified. Business
strategies that helped big pharmaceutical companies as well as biotech
companies to enter the market as well as maintain their market shares have
been identified. These detailed analyses are targeted to help strategy
developers and decision makers of biotech and pharmaceutical companies. It
also gives insights into the incentives that have been attractive for
different segments of the industry. This is especially suitable for
legislators involved in government policy development programs. Technology
trend analysis gives an overview of successful technologies, which are helpful
for research managers in charge of project identification and development.
SCOPE OF REPORT
This report contains:
- An overview of orphan drug criteria, the history of the Orphan Drug Act
and its influence and advantages inherent in orphan drug designation and
future growth trends
- Detailed discussion of successful orphan products
- Global sales projections through 2014 for as many as 57 orphan drugs with
sales exceeding $200 million
- Key players in the market and their market shares
- Detailed analysis of blockbuster orphan drugs
- Analysis of the Orphan Drug Act and its influence in the growth of the
biotech industry
- The rising influence of pharmacogenomics in the orphan drug market
- A discussion of specific rare diseases for which orphan drugs exist
- Analysis of the most promising therapeutic areas in the industry and
leading orphan drugs in these therapeutic areas
- An outline of legislation and regulation, including public and private
agencies involved in support and development
- Technology trends analysis within the orphan drug market
- In-depth analysis of leading companies and their successful strategies
within the orphan drug market segment.
INFORMATION SOURCES AND METHODOLOGY
The base year of this study is 2009. The market size reflects the global
revenues of drugs with orphan drugs status in the United States that have
generated more than $200 million in revenues in 2009. Drugs with market
exclusivity active during the base year as well as those with expired market
exclusivities in the U.S. market are included. The sales represent total
revenues, which include the sales for rare diseases as well as other
indications. This approach is important since the objective of this report is
to identify the significance of ODA in the growth of the orphan drugs segment.
Regulatory incentives provided by ODA have helped to promote clinical trials
of existing drugs to identify their effectiveness for several rare indications
in addition to the emergence of companies with exclusive orphan-drug-based
business models.
Market figures are based on revenues at the manufacturer level and are
projected at 2009 dollar values (i.e., inflation is not computed into the
projection figures). Forecasts are mainly based on the projections of the
marketers. Growth of competing drugs, patient population, patent and market
exclusivity status, and life cycle management strategies adopted by marketers
have also been considered for estimating the future growth trends.
The information and data to prepare this study were derived from regulatory
agencies, trade literature, patient support groups, annual reports of
companies, and industry-related databases. The analysis presented are based on
an extensive survey of the pharmaceutical industry as well as a detailed
examination of published literature and reports obtained from regulatory
authorities, medical research institutions, and national and world health
organizations. Key information from the literature was used to obtain expert
opinion and insights on commercial potential and market sizes from industry
professionals, academic researchers, and government agency spokespersons.
Table of Contents
Chapter- 1: INTRODUCTION -- Complimentary 5
- STUDY GOAL AND OBJECTIVES 1
- REASONS FOR DOING THE STUDY 2
- INTENDED AUDIENCE 2
- SCOPE OF REPORT 3
- INFORMATION SOURCES AND METHODOLOGY 3
- AUTHOR' S CREDENTIALS 4
- RELATED REPORTS FROM BCC RESEARCH 4
- BCC ONLINE SERVICES 5
- DISCLAIMER 5
Chapter-2: SUMMARY 2
- SUMMARY 6
- SUMMARY TABLE GLOBAL ORPHAN DRUGS DEMAND BY VALUE, THROUGH 2014 ($
MILLIONS) 7
- SUMMARY FIGURE GLOBAL ORPHAN DRUGS DEMAND BY VALUE, 2008-2014 ($ MILLIONS)
7
Chapter-3: OVERVIEW 23
- DEFINITION OF AN ORPHAN DRUG 8
- HISTORY OF ORPHAN DRUG LEGISLATION 8
- FIRST LEGISLATION ON ORPHAN DRUGS 8
- Amendments to the ODA 8
- Bills Passed in 2002 Increased Funding and Established the Office of Rare
Diseases 9
- Orphan Drug Legislation - Global Status 9
- INCENTIVES FOR ORPHAN DRUG DEVELOPMENT 9
- THE NEED FOR INCENTIVES 10
- TABLE 1 NUMBER OF ORPHAN DRUG DESIGNATIONS AND APPROVALS PER YEAR BY U.S.
FDA, 1983-2009 10
- APPLICATIONS FOR ORPHAN DRUG DESIGNATIONS HAVE DOUBLED 10
- PROFITABILITY OF ORPHAN DRUGS 11
- MARKET EXCLUSIVITY 11
- NO REGULATORY RESTRICTIONS ON PRICING 12
- OFF-LABEL USE 12
- CHALLENGES FOR EFFECTIVE USE OF THE ODA 12
- MARKET EXCLUSIVITY VERSUS PATENT PROTECTION 13
- MARKET SIZE AND KEY PLAYERS 14
- TABLE 2 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKET AND THEIR MARKET SHARES
(%) 14
- THE ORPHAN DRUG ACT - A BONUS FOR BIOTECH 15
- BIG PHARMA IS ENTERING THE ORPHAN DRUGS SECTOR AGGRESSIVELY 15
- CONCERNS ABOUT THE ORPHAN DRUG ACT 16
- COST-EFFECTIVENESS 16
- PRICING OF ORPHAN DRUGS 17
- TABLE 3 SOME EXAMPLES OF HIGHLY EXPENSIVE ORPHAN DRUGS AND THEIR PRICES 17
- TABLE 3 (CONTINUED) 18
- HIGH-PRICED ORPHAN DRUGS LEADING TO PRICE CONTROL MEASURES BY GOVERNMENTS
19
- TABLE 4 SOME OF THE RECENT CONTROVERSIES RELATED TO ORPHAN DRUGS PRICING 20
- ACCESS TO ORPHAN DRUGS AND REIMBURSEMENT - CURRENT PRACTICES 21
- ORPHAN DRUG ACCESS AND REIMBURSEMENT SCENARIO IN THE U.S. 21
- ACCESS AND REIMBURSEMENT SCENARIO IN EUROPE 21
- TABLE 5 ORPHAN DRUG ACCESS AND REIMBURSEMENT PRACTICES IN EUROPEAN
COUNTRIES 22
- TABLE 5 (CONTINUED) 23
- TABLE 5 (CONTINUED) 24
- CALL FOR UNIFICATION OF ORPHAN DRUG ACCESS ACROSS EUROPE 24
- INNOVATIVE APPROACHES OF PRICING AND REIMBURSEMENT 25
- TABLE 6 EMERGING TRENDS IN PRICING AND REIMBURSEMENT OF HIGH-PRICED DRUGS
25
- TABLE 6 (CONTINUED) 26
- HOW EFFECTIVE ODA IS IN PROMOTING RESEARCH ON RARE DISEASES? 26
- ORPHAN DRUGS-BASED BUSINESS MODELS - CASE STUDIES 27
- A NOT-FOR-PROFIT COMPANY 27
- A DISEASE FOUNDATION OPERATING AS A VIRTUAL COMPANY 28
- TABLE 7 PARTNERSHIP DEALS SIGNED RECENTLY BETWEEN CFFT AND PHARMACEUTICAL
INDUSTRY 28
- TABLE 7 (CONTINUED) 29
- A FOR-PROFIT COMPANY FOCUSED ON RARE DISEASES 29
- A SPECIALTY PHARMACY FOCUSING ON ULTRA- ORPHAN DRUGS 29
- A Specialty Pharmacy ...(Continued) 30
Chapter-4: WORLDWIDE INDUSTRY STRUCTURE 11
- GLOBAL AND REGIONAL SALES OF ORPHAN DRUGS 31
- TABLE 8 GLOBAL PROJECTED SALES OF ORPHAN DRUGS BY REGION, THROUGH 2014
($ MILLIONS) 32
- FIGURE 1 GLOBAL PROJECTED SALES OF ORPHAN DRUGS BY REGION, 2008-2014 ($
MILLIONS) 32
- CATEGORIES OF ORPHAN DRUG MARKET PLAYERS 32
- INDUSTRY TRENDS BY SPONSOR TYPES 33
- ANALYSIS OF ORPHAN DRUG APPROVALS BY SPONSOR CATEGORIES 33
- FIGURE 2 ANALYSIS OF ORPHAN DRUG APPROVALS BY SPONSOR TYPES, 1983-2009 (%)
34
- ORPHAN DRUG APPROVALS BY COMPANIES 35
- TABLE 9 COMPANIES WITH MORE THAN FIVE ORPHAN DRUG APPROVALS, 1983-2009 35
- TABLE 9 (CONTINUED) 36
- ORPHAN DRUG REVENUES BY SPONSOR TYPES 36
- BIG PHARMA VERSUS BIOTECH COMPANIES 36
- TABLE 10 SALES OF LEADING ORPHAN DRUGS - BIG PHARMA VERSUS BIOTECH
COMPANIES, 2009 ($ MILLIONS) 37
- TABLE 10 (CONTINUED) 38
- TABLE 10 (CONTINUED) 39
- ORPHAN DRUGS - NOW A STRATEGIC CHOICE FOR THE PHARMA INDUSTRY 39
- LEADING PLAYERS IN THE GLOBAL ORPHAN DRUG MARKET 40
- TABLE 11 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKET AND THEIR REVENUES
FROM ORPHAN DRUGS, 2009 41
Chapter-5: PRODUCTS 46
- ORPHAN DRUGS APPROVED BY THE U.S. FDA (1983 - 2009) 42
- ORPHAN DRUG DESIGNATIONS AND APPROVALS SINCE 1983 42
- TABLE 12 NUMBER OF ORPHAN DESIGNATIONS AND APPROVALS BY U.S. FDA,
1983-2009 42
- TABLE 12 (CONTINUED) 43
- TABLE 13 ORPHAN DRUG MARKETING APPROVALS BY U.S. FDA PER YEAR, 1983-2009 43
- TABLE 13 (CONTINUED) 44
- TABLE 13 (CONTINUED) 45
- ORPHAN DRUGS WITH SALES EXCEEDING $200 MILLION IN 2009 45
- TABLE 14 ORPHAN DRUGS WITH GLOBAL SALES EXCEEDING $200 MILLION, 2009 45
- TABLE 14 (CONTINUED) 46
- TABLE 14 (CONTINUED) 47
- DETAILED ANALYSIS OF SUCCESSFUL AND PROMISING ORPHAN DRUGS 47
- ALIMTA 47
- Additional indications 48
- Future Growth Trends 48
- TABLE 15 GLOBAL SALES OF ALIMTA, 2004 - 2009 ($ MILLIONS) 49
- AVASTIN 49
- Key Indications 49
- TABLE 16 KEY INDICATIONS FOR AVASTIN GRANTED BY VARIOUS REGULATORY
AGENCIES 49
- TABLE 16 (CONTINUED) 50
- Future Growth Trends 50
- AVONEX 50
- Additional Approvals Gained 50
- Market Strategies and Future Growth Trends 51
- TABLE 17 GLOBAL SALES OF AVONEX, 2000-2009 ($ MILLIONS) 52
- BETASERON 52
- Additional Approvals Gained 52
- Marketing Strategies and Future Growth Trends 53
- TABLE 18 GLOBAL SALES OF BETASERON, 2000-2009 ($ MILLIONS) 53
- BOTOX 54
- Marketing Strategies and Future Growth Trends 54
- TABLE 19 GLOBAL SALES OF BOTOX FOR THERAPEUTIC AND COSMETIC USAGES,
2002-2009 ($ MILLIONS) 55
- COPAXONE 55
- Marketing Strategies 55
- TABLE 20 GLOBAL SALES OF COPAXONE, 2002-2009 ($ MILLIONS) 56
- ENBREL 56
- Additional Indications 56
- Market Strategies 57
- TABLE 21 U.S. AND INTERNATIONAL SALES OF ENBREL, 2003-2009 ($ MILLIONS) 57
- EPOGEN 58
- Recent Threats to Market Share 58
- TABLE 22 GLOBAL SALES OF EPOGEN, 2000-2009 ($ MILLIONS) 59
- ERBITUX 59
- Additional Indications 60
- Market Strategies and Future Growth Trends 60
- TABLE 23 GLOBAL SALES OF ERBITUX, 2004-2009 ($ MILLIONS) 61
- EVISTA 61
- TABLE 24 GLOBAL SALES OF EVISTA, 2003-2009 ($ MILLIONS) 62
- GLEEVEC 62
- Approval for Multiple Indications 62
- Market Strategy and Future Growth Trends 63
- TABLE 25 GLOBAL SALES OF GLEEVEC, 2001-2009 ($ MILLIONS) 63
- HUMIRA 63
- TABLE 26 GLOBAL SALES OF HUMIRA, 2004 - 2009 ($ MILLIONS) 64
- INTRON A/PEGINTRON 64
- Market Strategy and Future Growth Trends 65
- TABLE 27 GLOBAL SALES OF INTRON A/PEGINTRON, 2004-2009 ($ MILLIONS) 65
- KOGENATE 65
- TABLE 28 GLOBAL SALES OF KOGENATE, 2001-2009 ($ MILLIONS) 66
- NEUPOGEN/NEULASTA 66
- Additional Indications 66
- Marketing Strategies 67
- TABLE 29 GLOBAL SALES OF NEUPOGEN/NEULASTA, 2002-2009 ($ MILLIONS) 67
- NOVOSEVEN 67
- TABLE 30 GLOBAL SALES OF NOVOSEVEN, 2000-2009 ($ MILLIONS) 68
- PROGRAF 68
- Additional Indications 68
- Future Prospects 69
- PROVIGIL 69
- REMICADE 70
- TABLE 31 GLOBAL SALES OF REMICADE 2001-2009 ($ MILLIONS) 70
- REVLIMID 70
- TABLE 32 GLOBAL SALES OF REVLIMID, 2006-2009 ($ MILLIONS) 71
- RITUXAN 71
- Additional Indications 71
- Characteristics of Market 72
- TABLE 33 GLOBAL SALES OF RITUXAN, 2000-2009 ($ MILLIONS) 72
- SANDOSTATIN LAR 72
- TEMODAR 73
- TABLE 34 GLOBAL SALES OF TEMODAR, 2003-2009 ($ MILLIONS) 73
- TOPAMAX 73
- Additional Indications 74
- Characteristics of the Market and Future Prospects 74
- TABLE 35 GLOBAL SALES OF TOPAMAX, 2001-2009 ($ MILLIONS) 74
- TRACLEER 75
- TABLE 36 GLOBAL SALES OF TRACLEER, 2002-2009 ($ MILLIONS) 75
- VELCADE 75
- TABLE 37 GLOBAL SALES OF VELCADE, 2006-2009 ($ MILLIONS) 76
- ZOMETA 76
- Additional Indications 76
- Market Characteristics 77
- TABLE 38 U.S. AND GLOBAL SALES OF ZOMETA, 2002 - 2009 ($ MILLIONS) 77
- ORPHAN DRUG SALES BY TECHNOLOGY 77
- ORPHAN BIOLOGICS 77
- Global Sales and Forecasts of Orphan Biologics 78
- TABLE 39 SALES AND FORECASTS OF LEADING BIOLOGIC ORPHAN DRUGS, WORLDWIDE,
THROUGH 2014 ($ MILLIONS) 78
- FIGURE 3 SALES AND FORECASTS OF LEADING BIOLOGIC ORPHAN DRUGS, WORLDWIDE,
2008-2014 ($ MILLIONS) 79
- SUBCLASSES OF ORPHAN BIOLOGICS 79
- TABLE 40 SALES AND FORECASTS OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG
SALES, WORLDWIDE, THROUGH 2014 ($ MILLIONS) 80
- FIGURE 4 SALES AND FORECASTS OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG
WORLDWIDE, 2008-2014 ($ MILLIONS) 80
- TABLE 41 GLOBAL SALES AND FORECASTS OF LEADING INTERFERONS WITH ORPHAN
DRUG STATUS, THROUGH 2014 ($ MILLIONS) 81
- FIGURE 5 GLOBAL SALES AND FORECASTS OF LEADING INTERFERONS WITH ORPHAN
STATUS, 2008-2014 ($ MILLIONS) 81
- TABLE 42 GLOBAL SALES AND FORECASTS OF LEADING GROWTH HORMONES WITH ORPHAN
DRUG STATUS, THROUGH 2014 ($ MILLIONS) 82
- FIGURE 6 GLOBAL SALES AND FORECASTS OF LEADING GROWTH HORMONES WITH ORPHAN
DRUG STATUS, 2008-2014 ($ MILLIONS) 83
- TABLE 43 GLOBAL SALES AND FORECASTS OF LEADING PLASMA PRODUCT ORPHAN DRUG
SALES, THROUGH 2014 ($ MILLIONS) 83
- FIGURE 7 FORECAST OF LEADING PLASMA PRODUCT ORPHAN DRUG SALES, 2008-2014
($ MILLIONS) 84
- NONBIOLOGIC ORPHAN DRUGS 84
- TABLE 44 GLOBAL SALES AND FORECASTS OF LEADING NONBIOLOGIC ORPHAN DRUGS,
THROUGH 2014 ($ MILLIONS) 85
- FIGURE 8 GLOBAL SALES AND FORECASTS FOR NONBIOLOGIC ORPHAN DRUGS,
2008-2014 ($ MILLIONS) 86
- FIGURE 9 ORPHAN DRUGS MARKET BY TECHNOLOGY TYPE, 2009 AND 2014 (%) 86
- FIGURE 9 (CONTINUED) 87
Chapter-6: APPLICATIONS 66
- CANCER 88
- LEUKEMIAS 88
- Acute Lymphocytic/Lymphoblastic Leukemia 89
- Acute Myelogenous/Myelocytic Leukemia 89
- Chronic Lymphocytic Leukemia 89
- Chronic Myelocytic/Myelogenous Leukemia 90
- LYMPHOMAS 90
- Hodgkin' s Lymphoma 90
- Non-Hodgkin' s Lymphoma 91
- MULTIPLE MYELOMA 91
- SOLID TUMORS 91
- ACROMEGALY 92
- CANCER-RELATED CONDITIONS 92
- TABLE 45 ORPHAN DRUGS APPROVED BY U.S. FDA TO TREAT CANCER AND
CANCER-RELATED CONDITIONS 93
- TABLE 45 (CONTINUED) 94
- TABLE 45 (CONTINUED) 95
- TABLE 45 (CONTINUED) 96
- TABLE 45 (CONTINUED) 97
- TABLE 45 (CONTINUED) 98
- TABLE 45 (CONTINUED) 99
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT CANCER AND CANCER-RELATED
CONDITIONS 99
- TABLE 46 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT CANCER, THROUGH
2014 ($ MILLIONS) 100
- FIGURE 10 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT CANCER AND
CANCER RELATED CONDITIONS, 2008-2014 ($ MILLIONS) 101
- CARDIOVASCULAR DISEASES AND BLOOD DISORDERS 101
- ANEMIA 101
- HYPERCALCEMIA 102
- HYPERTENSION 102
- HYPOTENSION 102
- NEUTROPENIA 102
- TACHYARRHYTHMIAS/TACHYCARDIA 103
- THROMBOCYTHEMIA 103
- THROMBOCYTOPENIA 103
- ORPHAN DRUGS APPROVED TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
103
- TABLE 47 APPROVED ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD
DISORDERS 104
- TABLE 47 (CONTINUED) 105
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT CARDIOVASCULAR DISEASES AND
BLOOD DISORDERS: WORLDWIDE, THROUGH 2014 106
- TABLE 48 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR
DISEASES AND BLOOD DISORDERS, THROUGH 2014 ($ MILLIONS) 106
- FIGURE 11 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR
DISEASES AND BLOOD DISORDERS, 2008-2014 ($ MILLIONS) 106
- GASTROINTESTINAL DISEASES 107
- CHOLELITHIASIS 107
- ESOPHAGEAL VARICES 107
- ORPHAN PRODUCTS APPROVED TO TREAT GASTROINTESTINAL DISEASES 107
- TABLE 49 APPROVED ORPHAN DRUGS TO TREAT GASTROINTESTINAL DISEASES 108
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT GASTROINTESTINAL DISEASES 108
- GENETIC DISEASES AND DISORDERS 108
- ALPHA-1 PROTEINASE DEFICIENCY 109
- FABRY DISEASE 109
- GAUCHER' S DISEASE 110
- HEMOPHILIA 110
- MUCOPOLYSACCHARIDOSIS 111
- TYROSINEMIA 111
- ORPHAN PRODUCTS APPROVED TO TREAT GENETIC DISEASES AND DISORDERS 112
- TABLE 50 APPROVED ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS 112
- TABLE 50 (CONTINUED) 113
- TABLE 50 (CONTINUED) 114
- TABLE 50 (CONTINUED) 115
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT GENETIC DISEASES AND DISORDERS
115
- TABLE 51 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES
AND DISORDERS, THROUGH 2014 ($ MILLIONS) 115
- FIGURE 12 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES
AND DISORDERS, 2008-2014 ($ MILLIONS) 116
- IMMUNE DISEASES 116
- COMMON VARIABLE IMMUNODEFICIENCY 116
- INTERSTITIAL CYSTITIS 117
- JUVENILE RHEUMATOID ARTHRITIS 117
- ORGAN TRANSPLANT REJECTION 118
- ORPHAN PRODUCTS APPROVED TO TREAT IMMUNE AND AUTOIMMUNE DISEASES AND
TRANSPLANT REJECTION 118
- TABLE 52 APPROVED ORPHAN DRUGS TO TREAT IMMUNE AND AUTOIMMUNE
DISEASES/TRANSPLANT REJECTION 118
- TABLE 52 (CONTINUED) 119
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT IMMUNE AND AUTOIMMUNE DISEASES
AND TRANSPLANT REJECTION 119
- TABLE 53 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN THE IMMUNE DISEASES
SECTOR, THROUGH 2014 ($ MILLIONS) 119
- FIGURE 13 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN THE IMMUNE DISEASES
SECTOR, 2008-2014 ($ MILLIONS) 120
- INFECTIOUS DISEASES 120
- ACQUIRED IMMUNE DEFICIENCY SYNDROME RELATED HEALTH CONDITIONS 120
- CRYPTOCOCCAL MENINGITIS 121
- CRYPTOSPORIDIOSIS 121
- CYTOMEGALOVIRUS RETINITIS 121
- GIARDIASIS 122
- HYDATID DISEASE 122
- KERATOCONJUNCTIVITIS 122
- LEISHMANIASIS 122
- LEPROSY 123
- MALARIA 123
- NEUROCYSTICERCOSIS 124
- PNEUMOCYSTIC CARINII PNEUMONIA 124
- RESPIRATORY SYNCYTIAL VIRUS 124
- TUBERCULOSIS 124
- ORPHAN PRODUCTS APPROVED TO TREAT INFECTIOUS DISEASES 125
- TABLE 54 APPROVED ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES 125
- TABLE 54 (CONTINUED) 126
- TABLE 54 (CONTINUED) 127
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT INFECTIOUS DISEASES 128
- TABLE 55 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFECTIOUS
DISEASES, THROUGH 2014 ($ MILLIONS) 128
- FIGURE 14 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFECTIOUS
DISEASES, 2008-2014 ($ MILLIONS) 128
- INFLAMMATORY DISEASES 129
- ANKYLOSING SPONDYLITIS 129
- CROHN' S DISEASE 129
- ORPHAN PRODUCTS APPROVED TO TREAT INFLAMMATORY DISEASES 129
- TABLE 56 APPROVED ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES 130
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT INFLAMMATORY DISEASES 130
- TABLE 57 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFLAMMATORY
DISEASES, THROUGH 2014 ($ MILLIONS) 131
- FIGURE 15 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFLAMMATORY
DISEASES, 2008-2014 ($ MILLIONS) 131
- NEUROLOGICAL DISEASES AND DISORDERS 131
- BLEPHAROSPASM 132
- DYSTONIA 132
- LENNOX-GASTAUT SYNDROME 132
- MULTIPLE SCLEROSIS 133
- NARCOLEPSY/CATAPLEXY 133
- STRABISMUS 133
- ORPHAN PRODUCTS APPROVED TO TREAT NEUROLOGICAL DISEASES AND DISORDERS 134
- TABLE 58 APPROVED ORPHAN DRUGS FOR NEUROLOGICAL DISEASES AND DISORDERS 134
- TABLE 58 (CONTINUED) 135
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT NEUROLOGICAL DISEASES AND
DISORDERS 135
- TABLE 59 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL
DISEASES AND DISORDERS, THROUGH 2014 ($ MILLIONS) 136
- FIGURE 16 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL
DISEASES AND DISORDERS, 2008-2014 ($ MILLIONS) 136
- REPRODUCTIVE DISEASES AND DISORDERS 137
- AMENORRHEA 137
- PRECOCIOUS PUBERTY 137
- IMPAIRED SPERMATOGENESIS 138
- ORPHAN PRODUCTS APPROVED TO TREAT REPRODUCTIVE DISEASES 138
- TABLE 60 APPROVED ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND
DISORDERS 138
- TABLE 60 (CONTINUED) 139
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT REPRODUCTIVE DISEASES AND
DISORDERS 139
- TABLE 61 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT: REPRODUCTIVE
DISEASES AND DISORDERS, THROUGH 2014 ($ MILLIONS) 139
- FIGURE 17 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT REPRODUCTIVE
DISEASES AND DISORDERS, 2008-2014 ($ MILLIONS) 140
- RESPIRATORY DISEASES 140
- HYALINE MEMBRANE 140
- MALIGNANT PLEURAL EFFUSION 141
- PULMONARY HYPERTENSION 141
- ORPHAN PRODUCTS APPROVED TO TREAT RESPIRATORY DISEASES 141
- TABLE 62 APPROVED ORPHAN DRUGS FOR RESPIRATORY DISEASES 142
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT RESPIRATORY DISEASES 143
- TABLE 63 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT: RESPIRATORY
DISEASES, THROUGH 2014 ($ MILLIONS) 143
- FIGURE 18 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT RESPIRATORY
DISEASES, 2008-2014 ($ MILLIONS) 143
- OTHER SELECTED RARE DISEASES AND DISORDERS FOR WHICH ORPHAN PRODUCTS ARE
AVAILABLE 144
- CHRONIC GRANULOMATOUS DISEASE 144
- CYSTINOSIS 144
- HOMOCYSTINURIA 144
- MASTOCYTOSIS 145
- PAGET' S DISEASE 145
- ORPHAN PRODUCTS APPROVED TO TREAT MISCELLANEOUS DISEASES AND DISORDERS 145
- TABLE 64 APPROVED ORPHAN DRUGS FOR MISCELLANEOUS INDICATIONS 146
- TABLE 64 (CONTINUED) 147
- TABLE 64 (CONTINUED) 148
- TABLE 64 (CONTINUED) 149
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT MISCELLANEOUS DISEASES AND
DISORDERS 149
- TABLE 65 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT: MISCELLANEOUS
DISEASES AND DISORDERS, THROUGH 2014 ($ MILLIONS) 150
- FIGURE 19 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT MISCELLANEOUS
DISEASES AND DISORDERS, 2008-2014 ($ MILLIONS) 150
- THERAPEUTIC AREAS WITH THE HIGHEST REVENUES FROM ORPHAN DRUGS IN 2009 AND
THEIR FUTURE GROWTH PROSPECTS 150
- TABLE 66 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN KEY THERAPEUTIC AREAS,
THROUGH 2014 ($ MILLIONS) 151
- FIGURE 20 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN KEY THERAPEUTIC AREAS,
2009 AND 2014 (%) 152
- FIGURE 20 (CONTINUED) 153
Chapter-7: LEGISLATION AND REGULATORY ENVIRONMENT 29
- LEGISLATION IN THE U.S. 154
- RARE DISEASES ACT AND RARE DISEASES ORPHAN PRODUCT DEVELOPMENT ACT OF 2002
155
- INCENTIVES FOR ORPHAN DRUG DEVELOPMENT IN THE U.S. 155
- Market Exclusivity for 7 Years Following Market Approval 155
- Tax Credits - 50% of the Cost of Clinical Trials Undertaken in the U.S.
155
- FDA Grants for Clinical Trials 156
- Technical and Administrative Assistance with the Identification and
Development of Orphan Products 156
- Exemption from Prescription Drug User Fee Act (PDUFA) - Waiver of
Application Fee 156
- Fast-track Approvals 156
- IMPACT OF THE ORPHAN DRUG ACT IN THE PHARMA AND BIOTECH INDUSTRIES 156
- FIGURE 21 NUMBER OF ORPHAN DRUG DESIGNATIONS IN THE U.S., 1983-2009 157
- FIGURE 21 (CONTINUED) 158
- FIGURE 22 ORPHAN DRUG APPROVALS IN THE U.S. (1983-2009) 158
- WAYS TO CIRCUMVENT MARKET EXCLUSIVITY 159
- SOME IMPORTANT PUBLIC AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND
PROMOTION IN THE U.S. 159
- TABLE 67 PUBLIC/NONPROFIT AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND
PROMOTION IN THE U.S. 159
- TABLE 67 (CONTINUED) 160
- IMPROVING ACCESS TO CLINICAL TRIALS ACT OF 2009 160
- LEGISLATION - EUROPEAN UNION 160
- TABLE 68 LIST OF ORPHAN DRUGS APPROVED BY EU, 2000-2009 161
- TABLE 68 (CONTINUED) 162
- TABLE 68 (CONTINUED) 163
- INCENTIVES FOR ORPHAN DRUG DEVELOPMENT IN THE EU 164
- 10 Years of Post-approval Market Exclusivity 164
- Protocol Assistance 164
- Access to a Centralized Community Procedure for Marketing Authorization
with a 50% Reduction in the Fee 164
- Other Fee Reductions 165
- Development Grants 165
- ORPHAN DESIGNATION PROCESS IN EU 165
- MAIN PUBLIC AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN
THE EU 165
- TABLE 69 MAIN PUBLIC/NONPROFIT AGENCIES INVOLVED IN ORPHAN DRUG REGULATION
AND PROMOTION IN THE EU 166
- AVAILABILITY OF ORPHAN DRUGS IN THE MEMBER STATES OF THE EU 167
- TABLE 70 COUNTRY-WISE SUMMARY OF NATIONAL MEASURES RELATED TO ORPHAN DRUG
LEGISLATION IN EU 167
- TABLE 70 (CONTINUED) 168
- TABLE 70 (CONTINUED) 169
- TABLE 70 (CONTINUED) 170
- INITIATIVES BY EMEA TO SPEED UP APPROVAL PROCESS 170
- CONVERGENCE OF REGULATORY PRACTICES OF EMEA AND FDA FOR ORPHAN DRUG
APPROVALS - AN EMERGING TREND 170
- LEGISLATION - JAPAN 171
- LEGISLATION - AUSTRALIA 172
- LEGISLATION - SINGAPORE 173
- LEGISLATION STATUS - OTHER COUNTRIES/REGIONS 174
- TAIWAN 174
- KOREA 174
- HONG KONG 175
- CANADA 175
- SWITZERLAND 175
- ISRAEL 176
- BRAZIL 176
- LEGISLATION COMPARISON - SUMMARY 176
- TABLE 71 COMPARISON OF ORPHAN DRUG LEGISLATION IN THE U.S., EU, AUSTRALIA,
AND JAPAN 177
- TABLE 71 (CONTINUED) 178
- CALL FOR MODIFICATIONS OF ORPHAN DRUG LEGISLATION 178
- CORRELATION BETWEEN MARKET EXCLUSIVITY AND THE HIGH PRICE OF ORPHAN DRUGS
179
- TABLE 72 ORPHAN DRUGS APPROVED FOR PULMONARY ARTERIAL HYPERTENSION (PAH)
IN THE U.S. AND EU 180
- IMPACT OF BIOSIMILARS ON THE ORPHAN DRUGS SECTOR 181
- FACTORS LEADING TO THE DELAY IN IMPLEMENTING REGULATIONS ON BIOGENERICS IN
THE U.S. 182
Chapter-8: TECHNOLOGY TRENDS ANALYSIS 8
- PATENT ANALYSIS 183
- LEADING ORPHAN DRUGS AND RELATED PATENTS 183
- TABLE 73 LEADING ORPHAN DRUGS AND THEIR RELEVANT U.S. PATENTS 184
- TABLE 73 (CONTINUED) 185
- TABLE 73 (CONTINUED) 186
- TECHNOLOGY TRENDS ANALYSIS BASED ON ORPHAN DRUG APPROVALS 186
- GENERAL TRENDS 187
- TABLE 74 PERCENTAGE OF BIOLOGICS IN ORPHAN DRUG APPROVALS, 1983-2009 187
- FIGURE 23 ORPHAN DRUG APPROVAL TRENDS: BIOLOGICS VERSUS NONBIOLOGICS 188
- TRENDS BY PRODUCT TYPES 188
- FIGURE 24 ORPHAN DRUG APPROVALS BY PRODUCT TYPES, 2009 (%) 189
- CONCLUSIONS OF TECHNICAL TRENDS ANALYSIS 190
Chapter-9: COMPANY PROFILES 12
- ABBOTT LABORATORIES 191
- ACTELION PHARMACEUTICALS 192
- AMGEN, INC. 192
- BAYER AG 193
- TABLE 75 ORPHAN DRUG PORTFOLIO OF BAYER - MARKETED AND BEING DEVELOPED
193
- TABLE 75 (CONTINUED) 194
- BIOGEN IDEC, INC. 194
- CELGENE CORPORATION 195
- ELI LILLY AND COMPANY 195
- TABLE 76 ORPHAN DRUGS MARKETED AND BEING DEVELOPED BY ELI LILLY 196
- GENZYME CORPORATION 196
- F. HOFFMANN-LA ROCHE, LTD. 197
- TABLE 77 ORPHAN DRUG DEVELOPMENT PROGRAMS BY ROCHE 198
- JOHNSON & JOHNSON 198
- TABLE 78 ORPHAN DRUG DEVELOPMENT PROGRAMS OF J&J 199
- NOVARTIS INTERNATIONAL AG 199
- MERCK & CO, INC. 200
- TABLE 79 ORPHAN DRUG DEVELOPMENT PROGRAMS BY MERCK 201
- PFIZER, INC. 201
- TABLE 80 ORPHAN DRUG DEVELOPMENT PROGRAMS BY PFIZER 202
GLOSSARY 3
- TABLE 81 GLOSSARY 203
- TABLE 81 (CONTINUED) 204
- TABLE 81 (CONTINUED) 205
|
