市場調査報告書：オーファンドラッグ

Abstract

INTRODUCTION

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, which was implemented in 1983, entered its 24th year in 2007. The purpose of this report is to analyze the orphan drug industry and provide market, technology, and revenue forecasts for the period 2006 to 2011. The report also analyzes the influence of the orphan drug industry in the development of the biotechnology industry.

This report also reviews 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. 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.

SCOPE OF STUDY

This report contains:

An overview of orphan drug criteria, the history of Orphan Drug Act and its influence and advantages inherent in orphan drug designation and future growth trends
Detailed discussion of successful orphan products and a discussion of specific rare diseases for which orphan drugs exist
Analysis of most promising therapeutic areas in the industry and leading orphan dugs in these therapeutic areas
Global sales projections through 2011 for as many as 50 orphan drugs with sales exceeding $200 million
Profiles of key players in the market and their market shares
Technology trends analysis within orphan drugs market

METHODOLOGY

The base year of this study is 2006. Market figures are based on revenues at the manufacturer level and are projected at 2006 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 lifecycle management strategies adopted by marketers have also been considered for estimating the future growth trends.

Data to prepare this study were derived from regulatory agencies, trade literature, patient support groups, and annual reports of companies and industry-related databases. In addition, discussion with product managers of pharmaceutical companies, research executives, academic researchers, government agency spokespersons, and directors of organizations focused on specific rare diseases have been conducted for gaining insights about the market, strategies, and future trends.

INFORMATION SOURCES

The information and analysis presented in this report 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, pharmaceutical trade associations, and national and world health organizations. Key information from the literature was used to obtain expert opinion on commercial potential and market sizes from industry professionals.

ANALYST CREDENTIALS

Syamala Ariyanchira was involved in chemical and pharmaceutical research for 7 years in India in companies, such as Chembiotech Research International, as research scientist and ICI as head of the Analytical Department. She has also worked in an academic research institute, and guided students in their Masters and Ph.D. programs at the University Malaya, Malaysia. For the past 4 years, she has been actively involved in industry analysis. She has worked as program manager with Frost & Sullivan, where she conducted many global market analyses of specialized market segments and several due diligence studies for multinational clients. She has also conducted technology foresight studies for government and private investors in the biotechnology sector. She has a Ph.D. in Chemistry from the Indian Institute of Science and post-doctoral experience in biophysics and molecular biology from Bose Institute, India.

Chapter 1 - SUMMARY
- Summary Table: GLOBAL ORPHAN DRUGS DEMAND BY VALUE, THROUGH 2011 ($ MILLIONS)
- Summary Figure: GLOBAL ORPHAN DRUGS DEMAND BY VALUE, 2005-2011 ($ MILLIONS)
Chapter 2 - OVERVIEW
- DEFINITION OF AN ORPHAN DRUG
- HISTORY OF ORPHAN DRUG LEGISLATION
  - FIRST LEGISLATION ON ORPHAN DRUGS
    - Amendments to the ODA
    - Bills Passed in 2002 Increased Funding and Established the Office of Rare Diseases
    - Orphan Drug Legislation-Global Status
  - INCENTIVES FOR ORPHAN DRUG DEVELOPMENT
  - THE NEED FOR INCENTIVES
- Table 1 NUMBER OF ORPHAN DRUG DESIGNATIONS AND APPROVALS PER YEAR BY U.S. FDA, 1983-2006
  - CONCERNS ABOUT THE ORPHAN DRUG ACT
  - COST-EFFECTIVENESS
  - MARKET EXCLUSIVITY VERSUS PATENT PROTECTION
  - MARKET SIZE AND KEY PLAYERS
- Table 2 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKET AND THEIR MARKET SHARES (%)
  - CHALLENGES FOR EFFECTIVE USAGE OF THE ORPHAN DRUG ACT
  - THE ORPHAN DRUG ACT-A BONUS FOR BIOTECH
  - PROFITABILITY OF ORPHAN DRUGS
    - MARKET EXCLUSIVITY
    - NO REGULATORY RESTRICTIONS ON PRICING
    - OFF-LABEL USE
Chapter 3 - WORLDWIDE INDUSTRY STRUCTURE
- GLOBAL AND REGIONAL SALES OF ORPHAN DRUGS
- Table 3 REGIONAL SALES OF ORPHAN DRUGS, THROUGH 2011 ($ MILLIONS)
- Figure 1 REGIONAL SALES AND FORECASTS FOR ORPHAN DRUGS, 2005-2011 ($ MILLIONS)
  - CATEGORIES OF ORPHAN DRUG MARKET PLAYERS
  - INDUSTRY TRENDS BY SPONSOR TYPES
    - ANALYSIS OF ORPHAN DRUG APPROVALS BY SPONSOR CATEGORIES
- Figure 2 ANALYSIS OF ORPHAN DRUG APPROVALS BY SPONSOR TYPES (%)
  - ORPHAN DRUG DESIGNATIONS AND APPROVALS BY COMPANIES
- Table 4 COMPANIES WITH MORE THAN 15 ORPHAN DRUG DESIGNATIONS, 1983-2006
- Table 5 COMPANIES WITH MORE THAN FIVE ORPHAN DRUG APPROVALS, 1983-2006
  - ORPHAN DRUG REVENUES BY SPONSOR TYPES
    - BIG PHARMA VERSUS BIOTECH COMPANIES
- Table 6 SALES OF LEADING ORPHAN DRUGS-BIG PHARMA VERSUS BIOTECH COMPANIES, 2006
- Table 6 (CONTINUED)
- Table 6 (CONTINUED)
  - ORPHAN DRUGS - A STRATEGIC CHOICE BY BIOTECH COMPANIES
  - LEADING PLAYERS IN THE GLOBAL ORPHAN DRUG MARKET
- Table 7 LEADING COMPANIES IN THE ORPHAN DRUG MARKET AND THEIR REVENUES FROM ORPHAN DRUGS
  - PARTNERSHIP STRATEGIES IN THE ORPHAN DRUG MARKET
- Table 8 ANALYSIS OF PARTNERSHIP STRATEGIES IN THE CASE OF LEADING ORPHAN DRUGS
- Table 8 (CONTINUED)
  - ORPHANS TO BLOCKBUSTERS-KEY GROWTH FACTORS
    - MULTIPLE ORPHAN INDICATIONS
- Table 9 BLOCKBUSTER ORPHAN DRUGS WITH APPROVALS FOR MULTIPLE INDICATIONS
- Table 9 (CONTINUED)
  - OFF-LABEL USAGES/ APPROVAL FOR NON-ORPHAN INDICATIONS
  - LACK OF COMPETITION FROM BIOGENERICS
- Table 10 BIOLOGIC BLOCKBUSTERS AND THEIR MARKET EXCLUSIVITY STATUS, 2006
- Table 10 (CONTINUED)
  - SYNTHETIC PHARMACEUTICALS AS BLOCKBUSTERS
  - PHARMACOGENOMICS AND ITS POTENTIAL IMPACT ON THE ORPHAN DRUG ACT
Chapter 4 - PRODUCTS
- ORPHAN DRUGS APPROVED BY THE U.S. FDA (1983-2006)
- ORPHAN DRUG DESIGNATIONS AND APPROVALS SINCE 1983
- Table 11 NUMBER OF ORPHAN DESIGNATIONS AND APPROVALS BY U.S. FDA 1983-2006
- Table 11 (CONTINUED)
- Table 12 DETAILS OF ORPHAN DRUG MARKETING APPROVALS BY U.S. FDA, 1983-2006 (TOTAL NO. OF ORPHAN DRUG APPROVALS EACH YEAR
- Table 12 (CONTINUED)
  - PERCENTAGE OF ORPHAN DRUG APPROVALS IN OVERALL DRUG APPROVALS BY THE U.S. FDA IN THE PAST DECADE
- Table 13 DRUGS AND BIOLOGICS APPROVED BY THE FDA FROM 1990 TO 2006, A COMPARISON OF PRODUCTS WITH AND WITHOUT ORPHAN DRUG STATUS
  - ORPHAN DRUGS WITH SALES EXCEEDING $200 MILLION IN 2006
- Table 14 ORPHAN DRUGS WITH GLOBAL SALES EXCEEDING $200 MILLION, 2006
- Table 14 (CONTINUED)
  - DETAILED ANALYSIS OF SUCCESSFULL AND PROMISING ORPHAN DRUGS
    - AVONEX
      - Additional Approvals Gained
      - Marketing Strategies
      - Sales of Avonex
- Table 15 GLOBAL SALES OF AVONEX, 2000-2006 ($ MILLIONS)
  - BETASERON
    - Additional Approvals Gained
    - Marketing Strategies
    - Sales of Betaseron
- Table 16 GLOBAL SALES OF BETASERON, 2000-2006 ($ MILLIONS)
  - BOTOX
    - Additional Approvals Gained
    - Marketing Strategies
    - Sales of Botox
- Table 17 GLOBAL SALES OF BOTOX FOR THERAPEUTIC AND COSMETIC USAGES, 2002-2006 ($ MILLIONS)
  - CEREZYME/ CEREDASE
    - Additional Approvals and Marketing Strategies
    - Sales of Cerezyme
- Table 18 GLOBAL SALES OF CEREZYME, 2000-2006 ($ MILLIONS)
  - COPAXONE
    - Marketing Strategies
    - Sales of Copaxone
- Table 19 GLOBAL SALES OF COPAXONE, 2002-2006 ($ MILLIONS)
  - ENBREL
    - Additional Indications
    - Market Strategies
    - Sales of Enbrel
- Table 20 U.S. AND INTERNATIONAL SALES OF ENBREL, 2003-2006 ($ MILLIONS)
  - EPOGEN
    - Recent Threats to Market Share
    - Sales of Epogen
- Table 21 GLOBAL SALES OF EPOGEN, 2000-2006 ($ MILLIONS)
  - ERBITUX
    - Additional Indications
    - Market Strategies and Threats
    - Sales of Erbitux
- Table 22 GLOBAL SALES OF ERBITUX, 2004-2006 ($ MILLIONS)
  - GLEEVEC
    - Approval for Multiple Indications
    - Market Strategy
    - Sales of Gleevec
- Table 23 GLOBAL SALES OF GLEEVEC, 2001-2006 ($ MILLIONS)
  - INTRON A/PEGINTRON
    - Market Strategy
    - Sales of Intron A/PegIntron
- Table 24 GLOBAL SALES OF INTRON A/PEGINTRON, 2004-2006 ($ MILLIONS)
  - KOGENATE
    - Sales of Kogenate
- Table 25 GLOBAL SALES OF KOGENATE, 2001-2006 ($ MILLIONS)
  - LAMICTAL
    - Sales of Lamictal
- Table 26 GLOBAL SALES OF LAMICTAL, 2001-2006 ($ MILLIONS)
  - NEUPOGEN/ NEULASTA
    - Additional Indications
    - Marketing Strategies
    - Sales of Neupogen/ Neulasta
- Table 27 GLOBAL SALES OF NEUPOGEN/NEULASTA, 2001-2006 ($ MILLIONS)
  - NOVOSEVEN
    - Sales of NovoSeven
- Table 28 GLOBAL SALES OF NOVOSEVEN, 2000-2006 ($ MILLIONS)
  - PROGRAF
    - Additional Indications
    - Future Prospects
    - Sales of Prograf
  - REMICADE
    - Additional Approvals
    - Characteristics of Target Market
    - Sales of Remicade
- Table 29 GLOBAL SALES OF REMICADE, 2001-2006 ($ MILLIONS)
  - RITUXAN
    - Additional Indications
    - Characteristics of Market
    - Sales of Rituxan
- Table 30 GLOBAL SALES OF RITUXAN, 2000-2006 ($ MILLIONS)
  - TOPAMAX
    - Additional Indications
    - Sales of Topamax
- Table 31 GLOBAL SALES OF TOPAMAX, 2001-2006 ($ MILLIONS)
  - ZOMETA
    - Additional indications
    - Market Characteristics
    - Sales of Zometa
- Table 32 U.S. AND GLOBAL SALES OF ZOMETA, 2002-2006 ($ MILLIONS)
  - ORPHAN VACCINES
  - ORPHAN DRUG SALES BY TECHNOLOGY
    - ORPHAN BIOLOGICS
      - Global Sales and Forecasts of Orphan Biologics
- Table 33 SALES AND FORECASTS OF LEADING BIOLOGIC ORPHAN DRUGS, WORLDWIDE: THROUGH 2011 ($ MILLIONS)
- Figure 3 FORECASTS OF LEADING BIOLOGICAL ORPHAN DRUG SALES, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - SUB-CLASSES OF ORPHAN BIOLOGICS
- Table 34 SALES AND FORECASTS OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG SALES, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 4 FORECAST OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG SALES, WORLDWIDE, 2005-2011 ($ MILLIONS)
- Table 35 GLOBAL SALES AND FORECASTS OF LEADING INTERFERONS/ INTERLEUKINS WITH ORPHAN DRUG STATUS, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 5 GLOBAL SALES AND FORECASTS OF LEADING INTERFERONS/ INTERLEUKINS WITH ORPHAN STATUS, WORLDWIDE, 2005-2011 ($ MILLIONS)
- Table 36 GLOBAL SALES AND FORECASTS OF LEADING GROWTH HORMONES WITH ORPHAN DRUG STATUS, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 6 GLOBAL SALES AND FORECASTS OF LEADING GROWTH HORMONES WITH ORPHAN DRUG STATUS, WORLDWIDE, 2005-2011 ($ MILLIONS)
- Table 37 GLOBAL SALES AND FORECASTS OF LEADING PLASMA PRODUCT ORPHAN DRUG SALES, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 7 FORECAST OF LEADING PLASMA PRODUCT ORPHAN DRUG SALES, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - NON-BIOLOGIC ORPHAN DRUGS
- Table 38 GLOBAL SALES AND FORECASTS OF LEADING NON-BIOLOGIC ORPHAN DRUGS, WORLDWIDE, THROUGH 2011($ MILLIONS)
- Figure 8 GLOBAL SALES AND FORECASTS FOR NON-BIOLOGIC ORPHAN DUGS, WORLDWIDE, 2005-2011 ($ MILLIONS)
Chapter 5 - APPLICATIONS
- CANCER
  - LEUKEMIAS
    - Acute Lymphocytic/ Lymphoblastic Leukemia
    - Acute Myelogenous/ Myelocytic Leukemia
    - Chronic Lymphocytic Leukemia
    - Chronic Myelocytic/ Myelogenous Leukemia
  - LYMPHOMAS
    - Hodgkin' s Lymphoma
    - Non-Hodgkin' s Lymphoma
  - MULTIPLE MYELOMA
  - SOLID TUMORS
  - CANCER-RELATED CONDITIONS
- Table 39 ORPHAN DRUGS APPROVED BY U.S. FDA TO TREAT CANCER AND CANCER-RELATED CONDITIONS
- Table 39 (CONTINUED)
- Table 39 (CONTINUED)
- Table 39 (CONTINUED)
- Table 39 (CONTINUED)
- Table 39 (CONTINUED)
- Table 39 (CONTINUED)
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT CANCER AND CANCER-RELATED CONDITIONS
- Table 40 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CANCER: WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 9 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CANCER AND CANCER RELATED CONDITIONS, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
    - ANEMIA
    - HYPERCALCEMIA
    - HYPERTENSION
    - HYPOTENSION
    - NEUTROPENIA
    - TACHYARRHYTHMIAS/TACHYCARDIA
    - THROMBOCYTHEMIA
    - THROMBOCYTOPENIA
    - ORPHAN DRUGS APPROVED TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
- Table 41 APPROVED ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
- Table 41 (CONTINUED)
- Table 41 (CONTINUED)
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS: WORLDWIDE, THROUGH 2011
- Table 42 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS, THROUGH 2011 ($ MILLIONS)
- Figure 10 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - GASTROINTESTINAL DISEASES
    - CHOLELITHIASIS
    - ESOPHAGEAL VARICES
    - ORPHAN PRODUCTS APPROVED TO TREAT GASTROINTESTINAL DISEASES
- Table 43 APPROVED ORPHAN DRUGS TO TREAT GASTROINTESTINAL DISEASES
- Table 43 (CONTINUED)
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT GASTROINTESTINAL DISEASES
- GENETIC DISEASES AND DISORDERS
  - ALPHA-1 PROTEINASE DEFICIENCY
  - FABRY DISEASE
  - GAUCHER DISEASE
  - HEMOPHILIA
  - MUCOPOLYSACCHARIDOSIS
  - TYROSINEMIA
  - ORPHAN PRODUCTS APPROVED TO TREAT GENETIC DISEASES AND DISORDERS
- Table 44 APPROVED ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS
- Table 44 (CONTINUED)
- Table 44 (CONTINUED)
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT GENETIC DISEASES AND DISORDERS
- Table 45 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS, WORLDWIDE, 2005-2011 ($ MILLIONS)
- Figure 11 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - IMMUNE DISEASES
    - COMMON VARIABLE IMMUNODEFICIENCY
    - INTERSTITIAL CYSTITIS
    - JUVENILE RHEUMATOID ARTHRITIS
    - ORGAN TRANSPLANT REJECTION
    - ORPHAN PRODUCTS APPROVED TO TREAT IMMUNE AND AUTOIMMUNE DISEASES AND TRANSPLANT REJECTION
- Table 46 APPROVED ORPHAN DRUGS TO TREAT IMMUNE AND AUTOIMMUNE DISEASES/ TRANSPLANT REJECTION
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT IMMUNE AND AUTOIMMUNE DISEASES AND TRANSPLANT REJECTION
- Table 47 THE MARKET FOR LEADING ORPHAN DRUGS IN THE IMMUNE DISEASES SECTOR, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 12 THE MARKET FOR LEADING ORPHAN DRUGS IN THE IMMUNE DISEASES SECTOR, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - INFECTIOUS DISEASES
    - ACQUIRED IMMUNE DEFICIENCY SYNDROME
    - CRYPTOCOCCAL MENINGITIS
    - CRYPTOSPORIDIOSIS
    - CYTOMEGALOVIRUS RETINITIS
    - GIARDIASIS
    - HYDATID DISEASE
    - KERATOCONJUNCTIVITIS
    - LEISHMANIASIS
    - LEPROSY
    - MALARIA
    - NEUROCYSTICERCOSIS
    - PNEUMOCYSTIC CARINII PNEUMONIA
    - RESPIRATORY SYNCYTIAL VIRUS
    - TUBERCULOSIS
    - ORPHAN PRODUCTS APPROVED TO TREAT INFECTIOUS DISEASES
- Table 48 APPROVED ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES
- Table 48 (CONTINUED)
- Table 48 (CONTINUED)
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT INFECTIOUS DISEASES
- Table 49 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 13 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - INFLAMMATORY DISEASES
    - ANKYLOSING SPONDYLITIS
    - CROHN' S DISEASE
    - ORPHAN PRODUCTS APPROVED TO TREAT INFLAMMATORY DISEASES
- Table 50 APPROVED ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT INFLAMMATORY DISEASES
- Table 51 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 14 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - NEUROLOGICAL DISEASES AND DISORDERS
    - BLEPHAROSPASM
    - DYSTONIA
    - LENNOX-GASTAUT SYNDROME
    - MULTIPLE SCLEROSIS
    - NARCOLEPSY/CATAPLEXY
    - STRABISMUS
    - ORPHAN PRODUCTS APPROVED TO TREAT NEUROLOGICAL DISEASES AND DISORDERS
- Table 52 APPROVED ORPHAN DRUGS FOR NEUROLOGICAL DISEASES AND DISORDERS
- Table 52 (CONTINUED)
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS
- Table 53 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 15 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - REPRODUCTIVE DISEASES AND DISORDERS
  - AMENORRHEA
  - PRECOCIOUS PUBERTY
  - IMPAIRED SPERMATOGENESIS
  - ORPHAN PRODUCTS APPROVED TO TREAT REPRODUCTIVE DISEASES
- Table 54 APPROVED ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS
- Table 55 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT, REPRODUC-TIVE DISEASES AND DISORDERS, WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 16 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - RESPIRATORY DISEASES
    - HYALINE MEMBRANE
    - MALIGNANT PLEURAL EFFUSION
    - PULMONARY HYPERTENSION
    - ORPHAN PRODUCTS APPROVED TO TREAT RESPIRATORY DISEASES
Table 56 APPROVED ORPHAN DRUGS FOR RESPIRATORY DISEASES
- SALES OF LEADING ORPHAN PRODUCTS TO TREAT RESPIRATORY DISEASES
- OTHER SELECTED RARE DISEASES AND DISORDERS FOR WHICH ORPHAN PRODUCTS ARE AVAILABLE
  - ACROMEGALY
  - CHRONIC GRANULOMATOUS DISEASE
  - CYSTINOSIS
  - HOMOCYSTINURIA
  - MASTOCYTOSIS
  - PAGET' S DISEASE
  - ORPHAN PRODUCTS APPROVED TO TREAT MISCELLANEOUS DISEASES AND DISORDERS
- Table 57 APPROVED ORPHAN DRUGS FOR MISCELLANEOUS INDICATIONS
- Table 57 (CONTINUED)
- Table 57 (CONTINUED)
- Table 57 (CONTINUED)
  - SALES OF LEADING ORPHAN PRODUCTS TO TREAT MISCELLANEOUS DISEASES AND DISORDERS
- Table 58 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT, MISCELLANEOUS DISEASES AND DISORDERS WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 17 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT MISCELLANEOUS DISEASES AND DISORDERS, WORLDWIDE, 2005-2011 ($ MILLIONS)
  - THERAPEUTIC AREAS WITH THE HIGHEST REVENUES FROM ORPHAN DRUGS IN 2006 AND THEIR FUTURE GROWTH PROSPECTS
- Table 59 THE MARKET FOR LEADING ORPHAN DRUGS IN KEY THERAPEUTIC AREAS WORLDWIDE, THROUGH 2011 ($ MILLIONS)
- Figure 18 LEADING ORPHAN DRUG SALES WORLDWIDE AND FORECASTS BY THERAPEUTIC AREAS, 2006 AND 2011 (%)
Chapter 6 - LEGISLATION AND REGULATORY ENVIRONMENT
- LEGISLATION IN THE U.S.
  - RARE DISEASES ACT OF 200" AND RARE DISEASES ORPHAN PRODUCT DEVELOPMENT ACT OF 2002
  - INCENTIVES FOR ORPHAN DRUG DEVELOPMENT IN THE U.S.
    - Technical and Administrative Assistance with the Identification and Development of Orphan Products
    - Availability of FDA Grants to Cover Clinical trial Expenses
    - Exemption from Prescription Drug User Fee Act (PDUFA)-Waiver of Application Fee
    - Tax Credits-50% of the Cost of Clinical Trials Undertaken in the U.S.
    - Market Exclusivity for 7 Years Following Market Approval
  - IMPACT OF THE ORPHAN DRUG ACT IN THE PHARMA AND BIOTECH INDUSTRIES
  - WAYS TO CIRCUMVENT MARKET EXCLUSIVITY
  - SOME IMPORTANT PUBLIC AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN THE U.S.
- Table 60 PUBLIC/NON-PROFIT AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN THE U.S.
  - LEGISLATION-EUROPEAN UNION
    - INCENTIVES FOR ORPHAN DRUG DEVELOPMENT IN THE EU
      - Free Protocol Assistance
      - Access to a Centralized Community Procedure for Marketing Authorization with a 50% Reduction in the Fee
      - 10 Years of Post-approval Market Exclusivity
    - MAIN PUBLIC AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN THE EU
- Table 61 MAIN PUBLIC/NON-PROFIT AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN THE EU
  - AVAILABILITY OF ORPHAN DRUGS IN THE MEMBER STATES OF THE EU
- Table 62 SUMMARY OF NATIONAL MEASURES ON ORPHAN DRUGS IN EU
- Table 62 (CONTINUED)
- Table 62 (CONTINUED)
- Table 62 (CONTINUED)
  - LEGISLATION-JAPAN
  - LEGISLATION-AUSTRALIA
  - LEGISLATION-SINGAPORE
  - LEGISLATION-OTHER COUNTRIES/ REGIONS
    - TAIWAN
    - KOREA
    - HONG KONG
  - LEGISLATION COMPARISON-SUMMARY
- Table 63 COMPARISON OF ORPHAN DRUG LEGISLATION IN THE U.S., EU, AUSTRALIA AND JAPAN
- Table 63 (CONTINUED)
  - CONCERN ABOUT THE HIGH PRICE OF ORPHAN DRUGS
  - CALL FOR MODIFICATIONS OF ORPHAN DRUG LEGISLATION
  - CORRELATION BETWEEN MARKET EXCLUSIVITY AND THE HIGH PRICE OF ORPHAN DRUGS
  - IMPACT OF BIOGENERICS ON THE ORPHAN DRUGS SECTOR
    - FACTORS LEADING TO THE DELAY IN IMPLEMENTING REGULATIONS ON BIOGENERICS
Chapter 7 - TECHNOLOGY TRENDS ANALYSIS
- PATENT ANALYSIS
- LEADING ORPHAN DRUGS AND RELATED PATENTS
- Table 64 LEADING ORPHAN DRUGS AND THEIR RELEVANT U.S. PATENTS
- Table 64 (CONTINUED)
- Table 64 (CONTINUED)
  - TECHNOLOGY TRENDS ANALYSIS BASED ON ORPHAN DRUG APPROVALS
    - GENERAL TRENDS
- Table 65BIOLOGICS IN ORPHAN DRUG APPROVALS, 1983-2006
- Figure 19 ORPHAN DRUG APPROVAL TRENDS, BIOLOGICS VERSUS NON-BIOLOGICS, 1983-2006
  - TRENDS BY PRODUCT TYPES
- Figure 20 ORPHAN DRUG APPROVALS BY PRODUCT TYPES, 2006 (%)
  - CONCLUSIONS OF TECHNICAL TRENDS ANALYSIS
Chapter 8 - COMPANY PROFILES
- AMGEN, INC.
- BAYER AG
- BIOGEN IDEC, INC.
- CELGENE CORPORATION
- GENENTECH, INC.
- GENZYME CORPORATION
- HOFFMANN-LA ROCHE, INC.
- JOHNSON & JOHNSON
- NOVARTIS INTERNATIONAL AG
- NOVO NOVODISK A/S
- SCHERING-PLOUGH CORPORATION
- SIGMA-TAU PHARMACEUTICALS, INC.
- SWEDISH ORPHAN INTERNATIONAL L AB

世界のオーファンドラッグ市場

Global Markets for Orphan Drugs

Abstract

INTRODUCTION

SCOPE OF STUDY

METHODOLOGY

INFORMATION SOURCES

ANALYST CREDENTIALS

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