Global Markets for Orphan Drugs

Table of Contents

• 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