NEW YORK, Aug. 10 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:
Global Markets for Orphan Drugs
http://www.reportlinker.com/p096587/Global-Markets-for-Orphan-Drugs.html
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
CHAPTER ONE: INTRODUCTION
STUDY GOAL AND OBJECTIVES
REASONS FOR DOING THE STUDY
INTENDED AUDIENCE
SCOPE OF REPORT
INFORMATION SOURCES AND METHODOLOGY
AUTHOR'S CREDENTIALS
RELATED REPORTS FROM BCC RESEARCH
BCC ONLINE SERVICES
DISCLAIMER
CHAPTER TWO: SUMMARY
SUMMARY
SUMMARY TABLE GLOBAL ORPHAN DRUGS DEMAND BY VALUE, THROUGH 2014 ($ MILLIONS)
SUMMARY FIGURE GLOBAL ORPHAN DRUGS DEMAND BY VALUE, 2008-2014 ($ MILLIONS)
CHAPTER THREE: 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, 1983-2009
APPLICATIONS FOR ORPHAN DRUG DESIGNATIONS HAVE DOUBLED
PROFITABILITY OF ORPHAN DRUGS
MARKET EXCLUSIVITY
NO REGULATORY RESTRICTIONS ON PRICING
OFF-LABEL USE
CHALLENGES FOR EFFECTIVE USE OF THE ODA
MARKET EXCLUSIVITY VERSUS PATENT PROTECTION
MARKET SIZE AND KEY PLAYERS
TABLE 2 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKET AND THEIR MARKET SHARES (%)
THE ORPHAN DRUG ACT — A BONUS FOR BIOTECH
BIG PHARMA IS ENTERING THE ORPHAN DRUGS SECTOR AGGRESSIVELY
CONCERNS ABOUT THE ORPHAN DRUG ACT
COST-EFFECTIVENESS
PRICING OF ORPHAN DRUGS
TABLE 3 SOME EXAMPLES OF HIGHLY EXPENSIVE ORPHAN DRUGS AND THEIR PRICES
HIGH-PRICED ORPHAN DRUGS LEADING TO PRICE CONTROL MEASURES BY GOVERNMENTS
TABLE 4 SOME OF THE RECENT CONTROVERSIES RELATED TO ORPHAN DRUGS PRICING
ACCESS TO ORPHAN DRUGS AND REIMBURSEMENT — CURRENT PRACTICES
ORPHAN DRUG ACCESS AND REIMBURSEMENT SCENARIO IN THE U.S.
ACCESS AND REIMBURSEMENT SCENARIO IN EUROPE
TABLE 5 ORPHAN DRUG ACCESS AND REIMBURSEMENT PRACTICES IN EUROPEAN COUNTRIES
CALL FOR UNIFICATION OF ORPHAN DRUG ACCESS ACROSS EUROPE
INNOVATIVE APPROACHES OF PRICING AND REIMBURSEMENT
TABLE 6 EMERGING TRENDS IN PRICING AND REIMBURSEMENT OF HIGH-PRICED DRUGS
HOW EFFECTIVE ODA IS IN PROMOTING RESEARCH ON RARE DISEASES?
ORPHAN DRUGS-BASED BUSINESS MODELS — CASE STUDIES
A NOT-FOR-PROFIT COMPANY
A DISEASE FOUNDATION OPERATING AS A VIRTUAL COMPANY
TABLE 7 PARTNERSHIP DEALS SIGNED RECENTLY BETWEEN CFFT AND PHARMACEUTICAL INDUSTRY
A FOR-PROFIT COMPANY FOCUSED ON RARE DISEASES
A SPECIALTY PHARMACY FOCUSING ON ULTRAORPHAN DRUGS
CHAPTER FOUR: WORLDWIDE INDUSTRY STRUCTURE
GLOBAL AND REGIONAL SALES OF ORPHAN DRUGS
TABLE 8 GLOBAL PROJECTED SALES OF ORPHAN DRUGS BY REGION, THROUGH 2014 ($ MILLIONS)
FIGURE 1 GLOBAL PROJECTED SALES OF ORPHAN DRUGS BY REGION, 2008-2014 ($ 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, 1983-2009 (%)
ORPHAN DRUG APPROVALS BY COMPANIES
TABLE 9 COMPANIES WITH MORE THAN FIVE ORPHAN DRUG APPROVALS, 1983-2009
ORPHAN DRUG REVENUES BY SPONSOR TYPES
BIG PHARMA VERSUS BIOTECH COMPANIES
TABLE 10 SALES OF LEADING ORPHAN DRUGS — BIG PHARMA VERSUS BIOTECH COMPANIES, 2009 ($ MILLIONS)
ORPHAN DRUGS — NOW A STRATEGIC CHOICE FOR THE PHARMA INDUSTRY
LEADING PLAYERS IN THE GLOBAL ORPHAN DRUG MARKET
TABLE 11 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKET AND THEIR REVENUES FROM ORPHAN DRUGS, 2009
CHAPTER FIVE: PRODUCTS
ORPHAN DRUGS APPROVED BY THE U.S. FDA (1983 - 2009)
ORPHAN DRUG DESIGNATIONS AND APPROVALS SINCE 1983
TABLE 12 NUMBER OF ORPHAN DESIGNATIONS AND APPROVALS BY U.S. FDA, 1983-2009
TABLE 13 ORPHAN DRUG MARKETING APPROVALS BY U.S. FDA PER YEAR, 1983-2009
ORPHAN DRUGS WITH SALES EXCEEDING $200 MILLION IN 2009
TABLE 14 ORPHAN DRUGS WITH GLOBAL SALES EXCEEDING $200 MILLION, 2009
DETAILED ANALYSIS OF SUCCESSFUL AND PROMISING ORPHAN DRUGS
ALIMTA
Additional indications
Future Growth Trends
TABLE 15 GLOBAL SALES OF ALIMTA, 2004-2009 ($ MILLIONS)
AVASTIN
Key Indications
TABLE 16 KEY INDICATIONS FOR AVASTIN GRANTED BY VARIOUS REGULATORY AGENCIES
Future Growth Trends
AVONEX
Additional Approvals Gained
Market Strategies and Future Growth Trends
TABLE 17 GLOBAL SALES OF AVONEX, 2000-2009 ($ MILLIONS)
BETASERON
Additional Approvals Gained
Marketing Strategies and Future Growth Trends
TABLE 18 GLOBAL SALES OF BETASERON, 2000-2009 ($ MILLIONS)
BOTOX
Marketing Strategies and Future Growth Trends
TABLE 19 GLOBAL SALES OF BOTOX FOR THERAPEUTIC AND COSMETIC USAGES, 2002-2009 ($ MILLIONS)
COPAXONE
Marketing Strategies
TABLE 20 GLOBAL SALES OF COPAXONE, 2002-2009 ($ MILLIONS)
ENBREL
Additional Indications
Market Strategies
TABLE 21 U.S. AND INTERNATIONAL SALES OF ENBREL, 2003-2009 ($ MILLIONS)
EPOGEN
Recent Threats to Market Share
TABLE 22 GLOBAL SALES OF EPOGEN, 2000-2009 ($ MILLIONS)
ERBITUX
Additional Indications
Market Strategies and Future Growth Trends
TABLE 23 GLOBAL SALES OF ERBITUX, 2004-2009 ($ MILLIONS)
EVISTA
TABLE 24 GLOBAL SALES OF EVISTA, 2003-2009 ($ MILLIONS)
GLEEVEC
Approval for Multiple Indications
Market Strategy and Future Growth Trends
TABLE 25 GLOBAL SALES OF GLEEVEC, 2001-2009 ($ MILLIONS)
HUMIRA
TABLE 26 GLOBAL SALES OF HUMIRA, 2004-2009 ($ MILLIONS)
INTRON A/PEGINTRON
Market Strategy and Future Growth Trends
TABLE 27 GLOBAL SALES OF INTRON A/PEGINTRON, 2004-2009 ($ MILLIONS)
KOGENATE
TABLE 28 GLOBAL SALES OF KOGENATE, 2001-2009 ($ MILLIONS)
NEUPOGEN/NEULASTA
Additional Indications
Marketing Strategies
TABLE 29 GLOBAL SALES OF NEUPOGEN/NEULASTA, 2002-2009 ($ MILLIONS)
NOVOSEVEN
TABLE 30 GLOBAL SALES OF NOVOSEVEN, 2000-2009 ($ MILLIONS)
PROGRAF
Additional Indications
Future Prospects
PROVIGIL
REMICADE
TABLE 31 GLOBAL SALES OF REMICADE 2001-2009 ($ MILLIONS)
REVLIMID
TABLE 32 GLOBAL SALES OF REVLIMID, 2006-2009 ($ MILLIONS)
RITUXAN
Additional Indications
Characteristics of Market
TABLE 33 GLOBAL SALES OF RITUXAN, 2000-2009 ($ MILLIONS)
SANDOSTATIN LAR
TEMODAR
TABLE 34 GLOBAL SALES OF TEMODAR, 2003-2009 ($ MILLIONS)
TOPAMAX
Additional Indications
Characteristics of the Market and Future Prospects
TABLE 35 GLOBAL SALES OF TOPAMAX, 2001-2009 ($ MILLIONS)
TRACLEER
TABLE 36 GLOBAL SALES OF TRACLEER, 2002-2009 ($ MILLIONS)
VELCADE
TABLE 37 GLOBAL SALES OF VELCADE, 2006-2009 ($ MILLIONS)
ZOMETA
Additional Indications
Market Characteristics
TABLE 38 U.S. AND GLOBAL SALES OF ZOMETA, 2002-2009 ($ MILLIONS)
ORPHAN DRUG SALES BY TECHNOLOGY
ORPHAN BIOLOGICS
Global Sales and Forecasts of Orphan Biologics
TABLE 39 SALES AND FORECASTS OF LEADING BIOLOGIC ORPHAN DRUGS, WORLDWIDE, THROUGH 2014 ($ MILLIONS)
FIGURE 3 SALES AND FORECASTS OF LEADING BIOLOGIC ORPHAN DRUGS, WORLDWIDE, 2008-2014 ($ MILLIONS)
SUBCLASSES OF ORPHAN BIOLOGICS
TABLE 40 SALES AND FORECASTS OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG SALES, WORLDWIDE, THROUGH 2014 ($ MILLIONS)
FIGURE 4 SALES AND FORECASTS OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG WORLDWIDE, 2008-2014 ($ MILLIONS)
TABLE 41 GLOBAL SALES AND FORECASTS OF LEADING INTERFERONS WITH ORPHAN DRUG STATUS, THROUGH 2014 ($ MILLIONS)
FIGURE 5 GLOBAL SALES AND FORECASTS OF LEADING INTERFERONS WITH ORPHAN STATUS, 2008-2014 ($ MILLIONS)
TABLE 42 GLOBAL SALES AND FORECASTS OF LEADING GROWTH HORMONES WITH ORPHAN DRUG STATUS, THROUGH 2014 ($ MILLIONS)
FIGURE 6 GLOBAL SALES AND FORECASTS OF LEADING GROWTH HORMONES WITH ORPHAN DRUG STATUS, 2008-2014 ($ MILLIONS)
TABLE 43 GLOBAL SALES AND FORECASTS OF LEADING PLASMA PRODUCT ORPHAN DRUG SALES, THROUGH 2014 ($ MILLIONS)
FIGURE 7 FORECAST OF LEADING PLASMA PRODUCT ORPHAN DRUG SALES, 2008-2014 ($ MILLIONS)
NONBIOLOGIC ORPHAN DRUGS
TABLE 44 GLOBAL SALES AND FORECASTS OF LEADING NONBIOLOGIC ORPHAN DRUGS, THROUGH 2014 ($ MILLIONS)
FIGURE 8 GLOBAL SALES AND FORECASTS FOR NONBIOLOGIC ORPHAN DRUGS, 2008-2014 ($ MILLIONS)
FIGURE 9 ORPHAN DRUGS MARKET BY TECHNOLOGY TYPE, 2009 AND 2014 (%)
CHAPTER SIX: 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
ACROMEGALY
CANCER-RELATED CONDITIONS
TABLE 45 ORPHAN DRUGS APPROVED BY U.S. FDA TO TREAT CANCER AND CANCER-RELATED CONDITIONS
SALES OF LEADING ORPHAN PRODUCTS TO TREAT CANCER AND CANCER-RELATED CONDITIONS
TABLE 46 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT CANCER, THROUGH 2014 ($ MILLIONS)
FIGURE 10 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT CANCER AND CANCER RELATED CONDITIONS, 2008-2014 ($ 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 47 APPROVED ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
SALES OF LEADING ORPHAN PRODUCTS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS: WORLDWIDE, THROUGH 2014
TABLE 48 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS, THROUGH 2014 ($ MILLIONS)
FIGURE 11 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS, 2008-2014 ($ MILLIONS)
GASTROINTESTINAL DISEASES
CHOLELITHIASIS
ESOPHAGEAL VARICES
ORPHAN PRODUCTS APPROVED TO TREAT GASTROINTESTINAL DISEASES
TABLE 49 APPROVED ORPHAN DRUGS TO TREAT GASTROINTESTINAL DISEASES
SALES OF LEADING ORPHAN PRODUCTS TO TREAT GASTROINTESTINAL DISEASES
GENETIC DISEASES AND DISORDERS
ALPHA-1 PROTEINASE DEFICIENCY
FABRY DISEASE
GAUCHER'S DISEASE
HEMOPHILIA
MUCOPOLYSACCHARIDOSIS
TYROSINEMIA
ORPHAN PRODUCTS APPROVED TO TREAT GENETIC DISEASES AND DISORDERS
TABLE 50 APPROVED ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS
SALES OF LEADING ORPHAN PRODUCTS TO TREAT GENETIC DISEASES AND DISORDERS
TABLE 51 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS, THROUGH 2014 ($ MILLIONS)
FIGURE 12 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS, 2008-2014 ($ 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 52 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 53 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN THE IMMUNE DISEASES SECTOR, THROUGH 2014 ($ MILLIONS)
FIGURE 13 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN THE IMMUNE DISEASES SECTOR, 2008-2014 ($ MILLIONS)
INFECTIOUS DISEASES
ACQUIRED IMMUNE DEFICIENCY SYNDROME RELATED HEALTH CONDITIONS
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 54 APPROVED ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES
SALES OF LEADING ORPHAN PRODUCTS TO TREAT INFECTIOUS DISEASES
TABLE 55 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES, THROUGH 2014 ($ MILLIONS)
FIGURE 14 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES, 2008-2014 ($ MILLIONS)
INFLAMMATORY DISEASES
ANKYLOSING SPONDYLITIS
CROHN'S DISEASE
ORPHAN PRODUCTS APPROVED TO TREAT INFLAMMATORY DISEASES
TABLE 56 APPROVED ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES
SALES OF LEADING ORPHAN PRODUCTS TO TREAT INFLAMMATORY DISEASES
TABLE 57 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES, THROUGH 2014 ($ MILLIONS)
FIGURE 15 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES, 2008-2014 ($ 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 58 APPROVED ORPHAN DRUGS FOR NEUROLOGICAL DISEASES AND DISORDERS
SALES OF LEADING ORPHAN PRODUCTS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS
TABLE 59 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS, THROUGH 2014 ($ MILLIONS)
FIGURE 16 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS, 2008-2014 ($ MILLIONS)
REPRODUCTIVE DISEASES AND DISORDERS
AMENORRHEA
PRECOCIOUS PUBERTY
IMPAIRED SPERMATOGENESIS
ORPHAN PRODUCTS APPROVED TO TREAT REPRODUCTIVE DISEASES
TABLE 60 APPROVED ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS
SALES OF LEADING ORPHAN PRODUCTS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS
TABLE 61 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT: REPRODUCTIVE DISEASES AND DISORDERS, THROUGH 2014 ($ MILLIONS)
FIGURE 17 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS, 2008-2014 ($ MILLIONS)
RESPIRATORY DISEASES
HYALINE MEMBRANE
MALIGNANT PLEURAL EFFUSION
PULMONARY HYPERTENSION
ORPHAN PRODUCTS APPROVED TO TREAT RESPIRATORY DISEASES
TABLE 62 APPROVED ORPHAN DRUGS FOR RESPIRATORY DISEASES
SALES OF LEADING ORPHAN PRODUCTS TO TREAT RESPIRATORY DISEASES
TABLE 63 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT: RESPIRATORY DISEASES, THROUGH 2014 ($ MILLIONS)
FIGURE 18 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT RESPIRATORY DISEASES, 2008-2014 ($ MILLIONS)
OTHER SELECTED RARE DISEASES AND DISORDERS FOR WHICH ORPHAN PRODUCTS ARE AVAILABLE
CHRONIC GRANULOMATOUS DISEASE
CYSTINOSIS
HOMOCYSTINURIA
MASTOCYTOSIS
PAGET'S DISEASE
ORPHAN PRODUCTS APPROVED TO TREAT MISCELLANEOUS DISEASES AND DISORDERS
TABLE 64 APPROVED ORPHAN DRUGS FOR MISCELLANEOUS INDICATIONS
SALES OF LEADING ORPHAN PRODUCTS TO TREAT MISCELLANEOUS DISEASES AND DISORDERS
TABLE 65 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT: MISCELLANEOUS DISEASES AND DISORDERS, THROUGH 2014 ($ MILLIONS)
FIGURE 19 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO TREAT MISCELLANEOUS DISEASES AND DISORDERS, 2008-2014 ($ MILLIONS)
THERAPEUTIC AREAS WITH THE HIGHEST REVENUES FROM ORPHAN DRUGS IN 2009 AND THEIR FUTURE GROWTH PROSPECTS
TABLE 66 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN KEY THERAPEUTIC AREAS, THROUGH 2014 ($ MILLIONS)
FIGURE 20 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN KEY THERAPEUTIC AREAS, 2009 AND 2014 (%)
CHAPTER SEVEN: LEGISLATION AND REGULATORY ENVIRONMENT
LEGISLATION IN THE U.S.
RARE DISEASES ACT AND RARE DISEASES ORPHAN PRODUCT DEVELOPMENT ACT OF 2002
INCENTIVES FOR ORPHAN DRUG DEVELOPMENT IN THE U.S.
Market Exclusivity for 7 Years Following Market Approval
Tax Credits — 50% of the Cost of Clinical Trials Undertaken in the U.S.
FDA Grants for Clinical Trials
Technical and Administrative Assistance with the Identification and Development of Orphan Products
Exemption from Prescription Drug User Fee Act (PDUFA) — Waiver of Application Fee
Fast-track Approvals
IMPACT OF THE ORPHAN DRUG ACT IN THE PHARMA AND BIOTECH INDUSTRIES
FIGURE 21 NUMBER OF ORPHAN DRUG DESIGNATIONS IN THE U.S., 1983-2009
FIGURE 22 ORPHAN DRUG APPROVALS IN THE U.S. (1983-2009)
WAYS TO CIRCUMVENT MARKET EXCLUSIVITY
SOME IMPORTANT PUBLIC AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN THE U.S.
TABLE 67 PUBLIC/NONPROFIT AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN THE U.S.
IMPROVING ACCESS TO CLINICAL TRIALS ACT OF 2009
LEGISLATION — EUROPEAN UNION
TABLE 68 LIST OF ORPHAN DRUGS APPROVED BY EU, 2000-2009
EU
10 Years of Post-approval Market Exclusivity
Protocol Assistance
Access to a Centralized Community Procedure for Marketing Authorization with a 50% Reduction in the Fee
Other Fee Reductions
Development Grants
ORPHAN DESIGNATION PROCESS IN EU
MAIN PUBLIC AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN THE EU
TABLE 69 MAIN PUBLIC/NONPROFIT AGENCIES INVOLVED IN ORPHAN DRUG REGULATION AND PROMOTION IN THE EU
AVAILABILITY OF ORPHAN DRUGS IN THE MEMBER STATES OF THE EU
TABLE 70 COUNTRY-WISE SUMMARY OF NATIONAL MEASURES RELATED TO ORPHAN DRUG LEGISLATION IN EU
INITIATIVES BY EMEA TO SPEED UP APPROVAL PROCESS
CONVERGENCE OF REGULATORY PRACTICES OF EMEA AND FDA FOR ORPHAN DRUG APPROVALS - AN EMERGING TREND
LEGISLATION — JAPAN
LEGISLATION — AUSTRALIA
LEGISLATION — SINGAPORE
LEGISLATION STATUS — OTHER COUNTRIES/REGIONS
TAIWAN
KOREA
HONG KONG
CANADA
SWITZERLAND
ISRAEL
BRAZIL
LEGISLATION COMPARISON — SUMMARY
TABLE 71 COMPARISON OF ORPHAN DRUG LEGISLATION IN THE U.S., EU, AUSTRALIA, AND JAPAN
CALL FOR MODIFICATIONS OF ORPHAN DRUG LEGISLATION
CORRELATION BETWEEN MARKET EXCLUSIVITY AND THE HIGH PRICE OF ORPHAN DRUGS
TABLE 72 ORPHAN DRUGS APPROVED FOR PULMONARY ARTERIAL HYPERTENSION (PAH) IN THE U.S. AND EU
IMPACT OF BIOSIMILARS ON THE ORPHAN DRUGS SECTOR
FACTORS LEADING TO THE DELAY IN IMPLEMENTING REGULATIONS ON BIOGENERICS IN THE U.S.
CHAPTER EIGHT: TECHNOLOGY TRENDS ANALYSIS
PATENT ANALYSIS
LEADING ORPHAN DRUGS AND RELATED PATENTS
TABLE 73 LEADING ORPHAN DRUGS AND THEIR RELEVANT U.S. PATENTS
TECHNOLOGY TRENDS ANALYSIS BASED ON ORPHAN DRUG APPROVALS
GENERAL TRENDS
TABLE 74 PERCENTAGE OF BIOLOGICS IN ORPHAN DRUG APPROVALS, 1983-2009
FIGURE 23 ORPHAN DRUG APPROVAL TRENDS: BIOLOGICS VERSUS NONBIOLOGICS
TRENDS BY PRODUCT TYPES
FIGURE 24 ORPHAN DRUG APPROVALS BY PRODUCT TYPES, 2009 (%)
CONCLUSIONS OF TECHNICAL TRENDS ANALYSIS
CHAPTER NINE: COMPANY PROFILES
ABBOTT LABORATORIES
ACTELION PHARMACEUTICALS
AMGEN, INC.
BAYER AG
TABLE 75 ORPHAN DRUG PORTFOLIO OF BAYER - MARKETED AND BEING DEVELOPED
BIOGEN IDEC, INC.
CELGENE CORPORATION
ELI LILLY AND COMPANY
TABLE 76 ORPHAN DRUGS MARKETED AND BEING DEVELOPED BY ELI LILLY
GENZYME CORPORATION
F. HOFFMANN-LA ROCHE, LTD.
TABLE 77 ORPHAN DRUG DEVELOPMENT PROGRAMS BY ROCHE
JOHNSON & JOHNSON
TABLE 78 ORPHAN DRUG DEVELOPMENT PROGRAMS OF J&J
NOVARTIS INTERNATIONAL AG
MERCK & CO, INC.
TABLE 79 ORPHAN DRUG DEVELOPMENT PROGRAMS BY MERCK
PFIZER, INC.
TABLE 80 ORPHAN DRUG DEVELOPMENT PROGRAMS BY PFIZER
GLOSSARY
TABLE 81 GLOSSARY
LIST OF TABLES
SUMMARY TABLE GLOBAL ORPHAN DRUGS DEMAND BY VALUE,
THROUGH 2014 ($ MILLIONS)
TABLE 1 NUMBER OF ORPHAN DRUG DESIGNATIONS AND
APPROVALS PER YEAR BY U.S. FDA, 1983-2009
TABLE 2 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKET AND
THEIR MARKET SHARES (%)
TABLE 3 SOME EXAMPLES OF HIGHLY EXPENSIVE ORPHAN DRUGS
AND THEIR PRICES
TABLE 4 SOME OF THE RECENT CONTROVERSIES RELATED TO
ORPHAN DRUGS PRICING
TABLE 5 ORPHAN DRUG ACCESS AND REIMBURSEMENT PRACTICES
IN EUROPEAN COUNTRIES
TABLE 6 EMERGING TRENDS IN PRICING AND REIMBURSEMENT OF
HIGH-PRICED DRUGS
TABLE 7 PARTNERSHIP DEALS SIGNED RECENTLY BETWEEN CFFT
AND PHARMACEUTICAL INDUSTRY
TABLE 8 GLOBAL PROJECTED SALES OF ORPHAN DRUGS BY
REGION, THROUGH 2014 ($ MILLIONS)
TABLE 9 COMPANIES WITH MORE THAN FIVE ORPHAN DRUG
APPROVALS, 1983-2009
TABLE 10 SALES OF LEADING ORPHAN DRUGS — BIG PHARMA
VERSUS BIOTECH COMPANIES, 2009 ($ MILLIONS)
TABLE 11 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKET AND
THEIR REVENUES FROM ORPHAN DRUGS, 2009
TABLE 12 NUMBER OF ORPHAN DESIGNATIONS AND APPROVALS
BY U.S. FDA, 1983-2009
TABLE 13 ORPHAN DRUG MARKETING APPROVALS BY U.S. FDA PER
YEAR, 1983-2009
TABLE 14 ORPHAN DRUGS WITH GLOBAL SALES EXCEEDING $200
MILLION, 2009
TABLE 15 GLOBAL SALES OF ALIMTA, 2004-2009 ($ MILLIONS)
TABLE 16 KEY INDICATIONS FOR AVASTIN GRANTED BY VARIOUS
REGULATORY AGENCIES
TABLE 17 GLOBAL SALES OF AVONEX, 2000-2009 ($ MILLIONS)
TABLE 18 GLOBAL SALES OF BETASERON, 2000-2009 ($ MILLIONS)
TABLE 19 GLOBAL SALES OF BOTOX FOR THERAPEUTIC AND
COSMETIC USAGES, 2002-2009 ($ MILLIONS)
TABLE 20 GLOBAL SALES OF COPAXONE, 2002-2009 ($ MILLIONS)
TABLE 21 U.S. AND INTERNATIONAL SALES OF ENBREL, 2003-2009 ($
MILLIONS)
TABLE 22 GLOBAL SALES OF EPOGEN, 2000-2009 ($ MILLIONS)
TABLE 23 GLOBAL SALES OF ERBITUX, 2004-2009 ($ MILLIONS)
TABLE 24 GLOBAL SALES OF EVISTA, 2003-2009 ($ MILLIONS)
TABLE 25 GLOBAL SALES OF GLEEVEC, 2001-2009 ($ MILLIONS)
TABLE 26 GLOBAL SALES OF HUMIRA, 2004-2009 ($ MILLIONS)
TABLE 27 GLOBAL SALES OF INTRON A/PEGINTRON, 2004-2009 ($
MILLIONS)
TABLE 28 GLOBAL SALES OF KOGENATE, 2001-2009 ($ MILLIONS)
TABLE 29 GLOBAL SALES OF NEUPOGEN/NEULASTA, 2002-2009 ($
MILLIONS)
TABLE 30 GLOBAL SALES OF NOVOSEVEN, 2000-2009 ($ MILLIONS)
TABLE 31 GLOBAL SALES OF REMICADE 2001-2009 ($ MILLIONS)
TABLE 32 GLOBAL SALES OF REVLIMID, 2006-2009 ($ MILLIONS)
TABLE 33 GLOBAL SALES OF RITUXAN, 2000-2009 ($ MILLIONS)
TABLE 34 GLOBAL SALES OF TEMODAR, 2003-2009 ($ MILLIONS)
TABLE 35 GLOBAL SALES OF TOPAMAX, 2001-2009 ($ MILLIONS)
TABLE 36 GLOBAL SALES OF TRACLEER, 2002-2009 ($ MILLIONS)
TABLE 37 GLOBAL SALES OF VELCADE, 2006-2009 ($ MILLIONS)
TABLE 38 U.S. AND GLOBAL SALES OF ZOMETA, 2002-2009 ($
MILLIONS)
TABLE 39 SALES AND FORECASTS OF LEADING BIOLOGIC ORPHAN
DRUGS, WORLDWIDE, THROUGH 2014 ($ MILLIONS)
TABLE 40 SALES AND FORECASTS OF LEADING MONOCLONAL
ANTIBODY ORPHAN DRUG SALES, WORLDWIDE, THROUGH 2014 ($
MILLIONS)
TABLE 41 GLOBAL SALES AND FORECASTS OF LEADING
INTERFERONS WITH ORPHAN DRUG STATUS, THROUGH 2014 ($
MILLIONS)
TABLE 42 GLOBAL SALES AND FORECASTS OF LEADING GROWTH
HORMONES WITH ORPHAN DRUG STATUS, THROUGH 2014 ($
MILLIONS)
TABLE 43 GLOBAL SALES AND FORECASTS OF LEADING PLASMA
PRODUCT ORPHAN DRUG SALES, THROUGH 2014 ($ MILLIONS)
TABLE 44 GLOBAL SALES AND FORECASTS OF LEADING
NONBIOLOGIC ORPHAN DRUGS, THROUGH 2014 ($ MILLIONS)
TABLE 45 ORPHAN DRUGS APPROVED BY U.S. FDA TO TREAT
CANCER AND CANCER-RELATED CONDITIONS
TABLE 46 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT CANCER, THROUGH 2014 ($ MILLIONS)
TABLE 47 APPROVED ORPHAN DRUGS TO TREAT CARDIOVASCULAR
DISEASES AND BLOOD DISORDERS
TABLE 48 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS,
THROUGH 2014 ($ MILLIONS)
TABLE 49 APPROVED ORPHAN DRUGS TO TREAT
GASTROINTESTINAL DISEASES
TABLE 50 APPROVED ORPHAN DRUGS TO TREAT GENETIC
DISEASES AND DISORDERS
TABLE 51 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT GENETIC DISEASES AND DISORDERS, THROUGH 2014 ($
MILLIONS)
TABLE 52 APPROVED ORPHAN DRUGS TO TREAT IMMUNE AND
AUTOIMMUNE DISEASES/TRANSPLANT REJECTION
TABLE 53 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN THE
IMMUNE DISEASES SECTOR, THROUGH 2014 ($ MILLIONS)
TABLE 54 APPROVED ORPHAN DRUGS TO TREAT
INFECTIOUS DISEASES
TABLE 55 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT INFECTIOUS DISEASES, THROUGH 2014 ($ MILLIONS)
TABLE 56 APPROVED ORPHAN DRUGS TO TREAT INFLAMMATORY
DISEASES
TABLE 57 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT INFLAMMATORY DISEASES, THROUGH 2014 ($ MILLIONS)
TABLE 58 APPROVED ORPHAN DRUGS FOR NEUROLOGICAL
DISEASES AND DISORDERS
TABLE 59 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT NEUROLOGICAL DISEASES AND DISORDERS, THROUGH
2014 ($ MILLIONS)
TABLE 60 APPROVED ORPHAN DRUGS TO TREAT REPRODUCTIVE
DISEASES AND DISORDERS
TABLE 61 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT: REPRODUCTIVE DISEASES AND DISORDERS, THROUGH
2014 ($ MILLIONS)
TABLE 62 APPROVED ORPHAN DRUGS FOR RESPIRATORY DISEASES .... 142
TABLE 63 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT: RESPIRATORY DISEASES, THROUGH 2014 ($ MILLIONS)
TABLE 64 APPROVED ORPHAN DRUGS FOR
MISCELLANEOUS INDICATIONS
TABLE 65 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT: MISCELLANEOUS DISEASES AND DISORDERS, THROUGH
2014 ($ MILLIONS)
TABLE 66 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN KEY
THERAPEUTIC AREAS, THROUGH 2014 ($ MILLIONS)
TABLE 67 PUBLIC/NONPROFIT AGENCIES INVOLVED IN ORPHAN
DRUG REGULATION AND PROMOTION IN THE U.S.
TABLE 68 LIST OF ORPHAN DRUGS APPROVED BY EU, 2000-2009
TABLE 69 MAIN PUBLIC/NONPROFIT AGENCIES INVOLVED IN
ORPHAN DRUG REGULATION AND PROMOTION IN THE EU
TABLE 70 COUNTRY-WISE SUMMARY OF NATIONAL MEASURES
RELATED TO ORPHAN DRUG LEGISLATION IN EU
TABLE 71 COMPARISON OF ORPHAN DRUG LEGISLATION IN THE
U.S., EU, AUSTRALIA, AND JAPAN
TABLE 72 ORPHAN DRUGS APPROVED FOR PULMONARY
ARTERIAL HYPERTENSION (PAH) IN THE U.S. AND EU
TABLE 73 LEADING ORPHAN DRUGS AND THEIR RELEVANT U.S.
PATENTS
TABLE 74 PERCENTAGE OF BIOLOGICS IN ORPHAN DRUG
APPROVALS, 1983-2009
TABLE 75 ORPHAN DRUG PORTFOLIO OF BAYER - MARKETED AND
BEING DEVELOPED
TABLE 76 ORPHAN DRUGS MARKETED AND BEING DEVELOPED BY
ELI LILLY
TABLE 77 ORPHAN DRUG DEVELOPMENT PROGRAMS BY ROCHE
TABLE 78 ORPHAN DRUG DEVELOPMENT PROGRAMS OF J&J
TABLE 79 ORPHAN DRUG DEVELOPMENT PROGRAMS BY MERCK
TABLE 80 ORPHAN DRUG DEVELOPMENT PROGRAMS BY PFIZER
TABLE 81 GLOSSARY
LIST OF FIGURES
SUMMARY FIGURE GLOBAL ORPHAN DRUGS DEMAND BY VALUE,
2008-2014 ($ MILLIONS)
FIGURE 1 GLOBAL PROJECTED SALES OF ORPHAN DRUGS BY
REGION, 2008-2014 ($ MILLIONS)
FIGURE 2 ANALYSIS OF ORPHAN DRUG APPROVALS BY SPONSOR
TYPES, 1983-2009 (%)
FIGURE 3 SALES AND FORECASTS OF LEADING BIOLOGIC ORPHAN
DRUGS, WORLDWIDE, 2008-2014 ($ MILLIONS)
FIGURE 4 SALES AND FORECASTS OF LEADING MONOCLONAL
ANTIBODY ORPHAN DRUG WORLDWIDE, 2008-2014 ($ MILLIONS)
FIGURE 5 GLOBAL SALES AND FORECASTS OF LEADING
INTERFERONS WITH ORPHAN STATUS, 2008-2014 ($ MILLIONS)
FIGURE 6 GLOBAL SALES AND FORECASTS OF LEADING GROWTH
HORMONES WITH ORPHAN DRUG STATUS, 2008-2014 ($ MILLIONS)
FIGURE 7 FORECAST OF LEADING PLASMA PRODUCT ORPHAN
DRUG SALES, 2008-2014 ($ MILLIONS)
FIGURE 8 GLOBAL SALES AND FORECASTS FOR NONBIOLOGIC
ORPHAN DRUGS, 2008-2014 ($ MILLIONS)
FIGURE 9 ORPHAN DRUGS MARKET BY TECHNOLOGY TYPE, 2009
AND 2014 (%)
FIGURE 10 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT CANCER AND CANCER RELATED CONDITIONS, 2008-2014 ($
MILLIONS)
FIGURE 11 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS,
2008-2014 ($ MILLIONS)
FIGURE 12 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT GENETIC DISEASES AND DISORDERS, 2008-2014 ($
MILLIONS)
FIGURE 13 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN THE
IMMUNE DISEASES SECTOR, 2008-2014 ($ MILLIONS)
FIGURE 14 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT INFECTIOUS DISEASES, 2008-2014 ($ MILLIONS)
FIGURE 15 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT INFLAMMATORY DISEASES, 2008-2014 ($ MILLIONS)
FIGURE 16 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT NEUROLOGICAL DISEASES AND DISORDERS, 2008-2014 ($
MILLIONS)
FIGURE 17 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT REPRODUCTIVE DISEASES AND DISORDERS, 2008-2014 ($
MILLIONS)
FIGURE 18 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT RESPIRATORY DISEASES, 2008-2014 ($ MILLIONS)
FIGURE 19 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO
TREAT MISCELLANEOUS DISEASES AND DISORDERS, 2008-2014 ($
MILLIONS)
FIGURE 20 GLOBAL MARKET FOR LEADING ORPHAN DRUGS IN KEY
THERAPEUTIC AREAS, 2009 AND 2014 (%)
FIGURE 21 NUMBER OF ORPHAN DRUG DESIGNATIONS IN THE U.S.,
1983-2009
FIGURE 22 ORPHAN DRUG APPROVALS IN THE U.S. (1983-2009)
FIGURE 23 ORPHAN DRUG APPROVAL TRENDS: BIOLOGICS
VERSUS NONBIOLOGICS
FIGURE 24 ORPHAN DRUG APPROVALS BY PRODUCT TYPES, 2009 (%)
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Nicolas Bombourg |
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