[英文調查報告書] 罕見疾病藥物的成功案例與市場策略

INTRODUCTION

STUDY GOAL AND OBJECTIVES

This report provides an in-depth look at the life stories of drugs with orphan disease designation(s) that had sales in excess of $200 million in 2002. The report examines in detail orphan drugs that have become multimillion-dollar products, provides revenues for leading orphan products, presents current and future strategies of companies developing orphan products as well as diseases addressed by these products, describes current and possible future legislation affecting the development and marketing of orphan drugs in the U.S. and internationally, and examines the historical and current relationship between the biotech industry and the Orphan Drug Act as well as the future relationship of pharmacogenomics to the Act. Also included are profiles of companies with blockbuster orphan products and companies specializing in orphan products, with particular focus on their development and marketing strategies. Forecasts and trends are gleaned from industry sources as well as from considered assessment of emerging products and technologies.

REASONS FOR DOING THE STUDY

As of mid-2003, 221 orphan drugs had been developed that are indicated for the treatment of rare diseases affecting approximately 11 million patients. Between 1992 and 2002, orphan drugs accounted for 17% of all drugs and biologics approved for sale in the U.S. Some orphan products have sales in excess of $1 billion annually.

A drug may receive orphan designation as its first indication or it may be approved for an orphan disease after it has been marketed for a nonorphan indication. This report describes in detail the special advantages of orphan product designation, which include the following:

assistance from the FDA in various stages of product development
tax incentives for sponsors in the form of tax credits for clinical research; the tax credit may be claimed whether or not the product is approved by the FDA
grant funding to defray costs of qualified clinical testing expenses incurred in connection with the development of orphan products
seven years of marketing exclusivity after approval of an orphan drug product.

This report provides histories of drugs initially designated as orphan products that became blockbusters by treating only orphan diseases as well as those that subsequently received other approvals that turned them into blockbusters. The overall purpose of the report is to provide a better understanding of the characteristics of orphan drug designation and the commercial advantages associated with the development of orphan drugs. To this end, strategies of companies focusing on the development of orphan drugs as well as those that incorporate orphan products into a broader development pipeline are discussed.

CONTRIBUTIONS OF THE STUDY AND FOR WHOM

This report analyzes the advantages of orphan product development and traces the development, commercialization and strategy for receiving initial and subsequent orphan and nonorphan disease designations for products. The report provides information needed to understand the impact of orphan product development on the marketplace, both historically and in the future. This report is intended to be a tool for business planners, acquisitions specialists, licensing strategists, product managers, market research analysts, investor consultants, and anyone interested in the pharmaceuticals and biopharmaceuticals markets and the role of orphan drugs in these markets.

SCOPE AND FORMAT

This report focuses on marketed and in-development products with orphan disease indications. The Overview chapter defines the criteria for orphan drug designation, the history of the Orphan Drug Act and its influence on product development, and the advantages inherent in orphan drug designation.

The Products chapter provides detailed discussion of products with orphan drug designation that had sales in excess of $200 million in 2002, recently introduced orphan products with a sales potential of $200 million annually or more, and selected orphan products in development. Sales projections are provided for many of the 26 products analyzed in this section.

The Applications chapter provides an overview of specific orphan diseases for which products exist and the sales of leading orphan products for these diseases.

The chapter on Legislation and the Regulatory Environment outlines public and private agencies involved in the support and development of products for orphan diseases, outlines orphan drug legislation in other countries and how this legislation differs from the American Orphan Drug Act and discusses the controversial topics of orphan drugs that have annual treatment costs of hundreds of thousands of dollars a year and those orphan products that have multibillion dollar annual sales.

The Industry chapter provides an analysis of the relationship between the Orphan Drug Act and the biotechnology boom in the 1980s and the effect of pharmacogenomics on the development of orphan products in the future, outlines leading companies in terms of number and sales of marketed orphan drug products, provides strategies for success utilized by five companies focusing on the development and marketing of orphan products and assesses the impact of the Orphan Drug Act on public and private investment in drugs for rare diseases.

The Company Profiles chapter provides a close look at companies currently participating in the development and/or marketing of orphan drugs, including companies that specialize in the development of products for orphan diseases only.

METHODOLOGY

The base year for this study is 2003. Market figures are based on revenues at the manufacturers' level and are projected at 2003 dollar value (i.e., inflation is not computed into the projection figures).

Data to prepare this study was derived from information obtained from product managers, marketing strategists, research executives, government agency spokespersons and directors of organizations focused on specific orphan diseases. In addition, information also was obtained from others at pharmaceutical and biotechnology companies that have products for orphan diseases on the market or in development.

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 business literature was used as to obtain expert opinion on commercial potential and market sizes from industry professionals.

INTRODUCTION

STUDY GOAL AND OBJECTIVES
REASONS FOR DOING THE STUDY
CONTRIBUTIONS OF THE STUDY AND FOR WHOM
SCOPE AND FORMAT
METHODOLOGY
INFORMATION SOURCES
AUTHOR'S CREDENTIALS
RELATED BCC, INC. REPORTS
BCC ONLINE SERVICES

EXECUTIVE SUMMARY

Summary Table:
GLOBAL SALES OF DRUGS APPROVED FOR ORPHAN DISEASES WITH SALES EXCEEDING $200 MILLION, 2002 THROUGH 2008 ($ MILLIONS)
Summary Figure:
GLOBAL SALES OF DRUGS APPROVED FOR ORPHAN DISEASES WITH SALES EXCEEDING $200 MILLION, 2002-2008 ($ MILLIONS)

OVERVIEW

DEFINITION OF AN ORPHAN DRUG
THE INCIDENCE OF RARE DISEASES

Table 1 MEAN PATIENT POPULATION FOR DESIGNATED ORPHAN PRODUCTS AT THE TIME OF DESIGNATION, 1983-2008
- THE NEED FOR INCENTIVES TO DEVELOP DRUGS FOR RARE DISEASES
  - COSTS
  - EXCLUSIVITY
  - MARKET SIZE AND SHAREHOLDERS
  - PATENT PROTECTION
  - INCENTIVES
- HISTORY OF THE ORPHAN DRUG ACT
  - THE PURPOSE OF THE ORPHAN DRUG ACT
  - THE 1992 RESPONSE FROM THE FDA
- THE EFFECT OF 20 YEARS OF THE ORPHAN DRUG ACT
  - THE AVERAGE COST OF DEVELOPING AN ORPHAN DRUG
  - MARKETING ORPHAN DRUGS
  - PERCENTAGE OF PRESCRIPTIONS WRITTEN FOR ORPHAN PRODUCTS
  - BILLS PASSED IN 2002 INCREASE FUNDING AND ESTABLISH THE OFFICE OF RARE DISEASES
  - ORPHAN DRUGS DRAMATICALLY DECREASE MORTALITY IN RARE DISEASES
  - CONCERNS ABOUT THE ORPHAN DRUG ACT
- ORPHAN DRUG DESIGNATION
  - ADVANTAGES OF ORPHAN DRUG DESIGNATION
    - Creation of the Orphan Drug Product Board
    - Assistance from the FDA
    - Open Clinical Trials
    - Tax Credits
Table 2 FDA ORPHAN DRUG MARKETING APPROVALS, TAX CREDIT, PRE-TAX EXPENDITURES AND PRE-TAX EXPENDITURES PER APPROVED DRUG, 1983-1993 ($MILLIONS)
Table 2 (CONTINUED)
Table 3 FDA ORPHAN DRUG MARKETING APPROVALS, TAX CREDITS AND PRE-TAX EXPENDITURES: 1995-2000
- PROFITABILITY OF ORPHAN DRUGS
- DISINCENTIVES TO THE DEVELOPMENT OF ORPHAN DRUGS
  - CONTROLLED SUBSTANCE REGULATION
  - TORT LIABILITY AND INSURANCE
    - Tort Liability and Insurance (Continued)

PRODUCTS

ORPHAN DRUGS APPROVED AS OF THE END OF 2002
ORPHAN DRUG DESIGNATIONS SINCE 1983
NUMBER OF ORPHAN DRUGS APPROVED ANNUALLY SINCE 1983

Table 4 FDA ORPHAN DRUG MARKETING APPROVALS: 1983-2002
Table 4 (CONTINUED)
- PERCENTAGE OF ORPHAN DRUG APPROVALS IN OVERALL DRUG APPROVALS
Table 5 DRUGS AND BIOLOGICS APPROVED BY THE FDA FROM 1992 TO 2002: A COMPARISON OF PRODUCTS WITH AND WITHOUT ORPHAN DRUG STATUS
Table 5 (CONTINUED)
- ORPHAN DRUGS WITH SALES EXCEEDING $200 MILLION IN 2002
Table 6 GLOBAL ORPHAN DRUGS WITH SALES EXCEEDING $200 MILLION, 2002
Table 6 (CONTINUED)
Table 6 (CONTINUED)
Table 6 (CONTINUED)
Table 6 (CONTINUED)
- REGIONAL SALES OF DRUGS WITH ORPHAN DISEASE INDICATIONS
Table 7 REGIONAL SALES OF DRUGS WITH ORPHAN DRUGS DISEASE INDICATIONS, THROUGH 2008 ($ MILLIONS)
Figure 1 REGIONAL SALES OF DRUGS WITH ORPHAN DRUGS DISEASE INDICATIONS, 2003 AND 2008 (%)
Table 8 DRUGS WITH SALES EXCEEDING $200 MILLION APPROVED ONLY FOR ORPHAN INDICATIONS, 2002
Table 8 (CONTINUED)
- RECENTLY INTRODUCED ORPHAN DRUG PRODUCTS PROJECTED TO HAVE SALES EXCEEDING $200 MILLION ANNUALLY
  - ALDURAZYME™
    - Aldurazyme™ (Continued)
    - Potential Use in Hurler Disease
    - Lysosolutions™ Launched
    - Pricing and Cost of Aldurazyme
    - Sales Projections for Aldurazyme™
  - ONTAK™
  - REPLAGAL™ (ORIGINALLY APPROVED FOR ORPHAN DISEASE INDICATION)
    - Sales of Replagal™
  - VELCADE™
    - FDA Approval
    - Future Indications
    - Sales of Velcade™
  - ZEVALIN™
    - First Radioimmunotherapeutic Drug
    - Results of Trials
    - Additional Areas of Investigation
    - Patent Infringement Lawsuit Filed Against Potential Competitor
    - Sales of Zevalin™
- OTHER SELECTED RECENTLY APPROVED ORPHAN DRUGS
  - ALINIA™
  - ARALAST™
  - CAMPATH™/MABCAMPATH™
    - Campath™/Mabcampath™ (Continued)
  - ELITEK™
  - FABRAZYME
    - Projected Sales of Fabrazyme
  - ORFADIN™
  - REMODULIN™
  - SUBUTEX/SUBOXONE
  - SYNTHETIC PORCINE SECRETIN™
  - XYREM™
- SELECTED PRODUCTS IN DEVELOPMENT WITH ORPHAN DRUG DESIGNATION
  - AMD-3100™
    - AMD-3100™ (Continued)
  - ANTIGASTRIN IMMUNOGEN™ (6117DT)
  - BLYS™
  - CLOFARABINE™
  - DECITABINE™
  - HUMAN GAMMAGLOBULIN™
  - I2S™
  - MOLI1901™
  - THERALUX™ PHOTODYNAMIC THERAPY
  - VIRAZOLE™
  - VIRULIZIN™
  - ZAVESCA™
- VACCINES FOR ORPHAN DISEASES
- ORPHAN DRUG SALES BY TECHNOLOGY
Table 9 FORECAST OF LEADING BIOLOGICAL ORPHAN DRUG SALES, WORLDWIDE: THROUGH 2008 ($ MILLIONS)
Figure 2 FORECAST OF LEADING BIOLOGICAL ORPHAN DRUG SALES, WORLDWIDE: 2002-2008 ($ MILLIONS)
Table 10 FORECAST OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG SALES, WORLDWIDE: THROUGH 2008 ($ MILLIONS)
Figure 3 FORECAST OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG SALES, WORLDWIDE: 2002-2008 ($ MILLIONS)

APPLICATIONS

CANCER

Table 11 INCIDENCE, MORTALITY, AND SURVIVAL RATES FOR COMMON CANCERS: U.S., 2000
Table 11 (CONTINUED)
Table 12 CANCER PREVALENCE RATES: U.S., 2000
Table 13 APPROVED ORPHAN DRUGS TO TREAT CANCER AND CANCER-RELATED CONDITIONS
Table 13 (CONTINUED)
Table 13 (CONTINUED)
Table 13 (CONTINUED)
Table 14 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CANCER: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
- CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
  - ANEMIA
  - HYPERCALCEMIA
  - HYPERTENSION
  - HYPOTENSION
  - NEUTROPENIA
  - TACHYARRHYTHMIAS/TACHYCARDIA
  - THROMBOCYTHEMIA
  - THROMBOCYTOPENIA
  - ORPHAN PRODUCTS APPROVED TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
Table 15 APPROVED ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
Table 15 (CONTINUED)
Table 16 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Figure 4 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS: WORLDWIDE, 2002-2008 ($ MILLIONS)
- DERMATOLOGICAL DISEASES AND DISORDERS
  - ACNE ROSACEA
  - ERYTHEMA NODOSUM LEPROSUM
  - ORPHAN PRODUCTS APPROVED TO TREAT DERMATOLOGIC DISEASES
Table 17 APPROVED ORPHAN DRUGS TO TREAT DERMATOLOGICAL DISEASES
- GASTROINTESTINAL DISEASES
  - CHOLELITHIASIS
  - ESOPHAGEAL VARICES
  - ORPHAN PRODUCTS APPROVED TO TREAT GASTROINTESTINAL DISEASES
Table 18 APPROVED ORPHAN DRUGS TO TREAT GASTROINTESTINAL DISEASES
- GENETIC DISEASES AND DISORDERS
  - ALPHA-1 PROTEINASE DEFICIENCY
  - FABRY DISEASE
  - GAUCHER DISEASE
  - HEMOPHILIA
  - MUCOPOLYSACCHARIDOSIS I AND II
    - Hurler Disease
Table 19 GEOGRAPHIC DISTRIBUTION OF PATIENTS SUFFERING FROM HURLER DISEASE, 2003
Table 20 APPROVED ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS
Table 20 (CONTINUED)
Table 20 (CONTINUED)
Table 20 (CONTINUED)
Table 21 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Figure 5 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS: WORLDWIDE, 2002-2008 ($ 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 22 APPROVED ORPHAN DRUGS TO TREAT IMMUNE AND AUTOIMMUNE DISEASES/TRANSPLANTATION REJECTION
Table 23 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT IMMUNE DISEASES/TRANSPLANT REJECTION: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
- INFECTIOUS DISEASES
  - ACQUIRED IMMUNE DEFICIENCY SYNDROME
  - CRYPTOCOCCAL MENINGITIS
  - CRYPTOSPORIDIOSIS
  - CYTOMEGALOVIRUS RETINITIS
  - GIARDIASIS
  - HEMORRHAGIC FEVER
  - HYDATID DISEASE
  - KERATOCONJUNCTIVITIS
  - LEISHMANIASIS
  - LEPROSY
  - MALARIA
  - NEUROCYSTICERCOSIS
  - PNEUMOCYSTIC CARINII PNEUMONIA
  - RESPIRATORY SYNCYTIAL VIRUS
  - TUBERCULOSIS
  - ORPHAN PRODUCTS APPROVED TO TREAT INFECTIOUS DISEASES
Table 24 APPROVED ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES
Table 24 (CONTINUED)
Table 24 (CONTINUED)
Table 25 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
- INFLAMMATORY DISEASES
  - ANKYLOSING SPONDYLITIS
  - CROHN'S DISEASE
  - ORPHAN PRODUCTS APPROVED TO TREAT INFLAMMATORY DISEASES
Table 26 APPROVED ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES
Table 27 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
- NEUROLOGICAL DISEASES AND DISORDERS
  - BLEPHAROSPASM
  - DYSTONIA
  - EPILEPSY
  - LENNOX-GASTAUT SYNDROME
  - MULTIPLE SCLEROSIS
  - NARCOLEPSY/CATAPLEXY
  - STRABISMUS
  - ORPHAN PRODUCTS APPROVED TO TREAT NEUROLOGICAL DISEASES AND DISORDERS
Table 28 APPROVED ORPHAN DRUGS FOR NEUROLOGICAL DISEASES AND DISORDERS
Table 28 (CONTINUED)
Table 29 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Figure 6 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS: WORLDWIDE, 2002-2008 ($ MILLIONS)
- REPRODUCTIVE DISEASES AND DISORDERS
  - IMPAIRED SPERMATOGENESIS
  - PRECOCIOUS PUBERTY
  - ORPHAN PRODUCTS APPROVED TO TREAT REPRODUCTIVE DISEASES AND DISORDERS
Table 30 APPROVED ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS
Table 30 (CONTINUED)
Table 31 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Figure 7 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS: WORLDWIDE, 2002-2008 ($ MILLIONS)
- RESPIRATORY DISEASES
  - APNEA
  - HYALINE MEMBRANE DISEASE
  - MALIGNANT PLEURAL EFFUSION
  - PULMONARY HYPERTENSION
  - RESPIRATORY DISTRESS SYNDROME
  - ORPHAN PRODUCTS APPROVED TO TREAT RESPIRATORY DISEASES
Table 32 APPROVED ORPHAN DRUGS FOR RESPIRATORY DISEASES
- OTHER SELECTED RARE DISEASES AND DISORDERS FOR WHICH ORPHAN PRODUCTS ARE AVAILABLE
  - ACROMEGALY
  - CHRONIC GRANULOMATOUS DISEASE
  - CYSTINOSIS
  - HOMOCYSTINURIA
  - IMMUNOLOGIC (IDIOPATHIC) THROMBOCYTOPENIC PURPURA
  - MASTOCYTOSIS
  - PAGET'S DISEASE
  - ORPHAN PRODUCTS APPROVED TO TREAT MISCELLANEOUS DISEASES AND DISORDERS
Table 33 APPROVED ORPHAN DRUGS FOR MISCELLANEOUS INDICATIONS
Table 33 (CONTINUED)
Table 33 (CONTINUED)

LEGISLATION AND REGULATORY ENVIRONMENT

PUBLIC AGENCIES INVOLVED IN ORPHAN DRUG DEVELOPMENT IN THE U.S.
- PRESIDENT BUSH SIGNS THE "RARE DISEASES ACT OF 2002" AND "RARE DISEASES ORPHAN PRODUCT DEVELOPMENT ACT OF 2002"
- NATIONAL ORGANIZATION FOR RARE DISORDERS CALLS FOR DEVELOPMENT OF GENERIC BIOLOGICS
- CONCERN ABOUT ACCESS
- CONCERN ABOUT COST
- DEVELOPING A COMPETITIVE BIOLOGICS MARKET
- THE FEASIBILITY OF DEVELOPING GENERIC BIOLOGICS
  - The Feasibility of Developing Generic�c(Continued)
PRIVATE AGENCIES INVOLVED IN ORPHAN DRUG DEVELOPMENT IN THE U.S.
- BIG PHARMA
- NATIONAL ORGANIZATION FOR RARE DISORDERS
- BIOTECHNOLOGY INDUSTRY ORGANIZATION
PASSAGE OF ORPHAN DRUG LEGISLATION BY OTHER NATIONS
- RARE DISEASE TREATMENT-A GLOBAL PROBLEM AND A GLOBAL OPPORTUNITY
  - Legislation in the European Union

Table 34 COMPARISON OF NATIONAL MEASURES ON ORPHAN DRUGS IN THE E.U.
Table 35 DIFFERENCES IN ACCESS TO ORPHAN DRUGS IN VARIOUS EUROPEAN COUNTRIES
Table 35 (CONTINUED)
Table 36 COMPARISON OF ORPHAN DRUG LEGISLATION/PROVISIONS IN THE U.S., E.U., AUSTRALIA AND JAPAN
- ORPHAN VACCINES
  - ORPHAN VACCINES (CONTINUED)
  - PRICING OF VACCINES
  - PATENT PROTECTION FOR VACCINES
  - VACCINES FOR THIRD WORLD DISEASES
    - Development Strategy
    - Improving the Availability of Orphan Vaccines
- WHAT SHOULD BE DONE ABOUT ORPHAN DRUGS THAT MAKE TOO MUCH MONEY?
- �cAND ORPHAN DRUGS THAT COST TOO MUCH?
- �cAND ORPHAN DRUGS THAT COST TOO�c(CONTINUED)
- MEDICARE REDUCES FUNDING FOR RARE DISEASES

INDUSTRY

THE ORPHAN DRUG ACT GIVES BIRTH TO BIOTECH

Table 37 GROWTH IN BIOLOGIC ORPHAN PRODUCTS, 1983-2000 (%)
Table 38 ORPHAN DRUG STATUS OF THE TOP 10 BIOTECH DRUGS IN 2002
- COMPANIES WITH THE MOST APPROVED INDICATIONS FOR ORPHAN DRUGS
Table 39 MARKETERS OF ORPHAN DRUGS AND NUMBER OF APPROVED INDICATIONS (AS OF 5/15/03)
Table 40 NUMBER OF PRODUCTS APPROVED FOR THE TREATMENT OF ORPHAN DISEASES-BIG PHARMA VERSUS BIOTECH: MAY 2003
- LEADING COMPANIES IN SALES OF ORPHAN PRODUCTS
Table 41 SALES OF DRUGS WITH ORPHAN STATUS, BY COMPANY, 2002 ($ MILLIONS)
Table 41 (CONTINUED)
Table 42 SALES OF LEADING PRODUCTS APPROVED FOR THE TREATMENT OF ORPHAN DISEASES-BIG PHARMA VERSUS BIOTECH, 2002 ($ MILLIONS)
- STRATEGIES FOR SUCCESS IN MARKETING ORPHAN DRUGS-FIVE BUSINESS MODELS
  - THE LIPOSOME COMPANY
  - NOVAZYME (GENZYME)
  - Business Model Attracts Venture Capital
  - ORPHAN MEDICAL
  - SERONO
  - SIGMA-TAU PHARMACEUTICALS
- HOW THE ORPHAN DRUG ACT HAS AFFECTED INVESTMENT IN THE DEVELOPMENT OF PRODUCTS FOR RARE DISEASES
  - PUBLIC COMPANIES VERSUS PRIVATE COMPANIES
  - THE RELATIONSHIP BETWEEN VENTURE CAPITAL/PRIVATE EQUITY FIRMS AND THE ORPHAN DRUG ACT
Table 43 SELECTED VENTURE CAPITAL/PRIVATE EQUITY INVESTMENTS IN COMPANIES MARKETING ORPHAN DRUGS
- PHARMACOGENOMICS DRIVES THE ORPHAN DRUG MARKET OF THE FUTURE
  - PHARMACOGENOMICS IN DRUG DISCOVERY AND DEVELOPMENT
    - Three Areas of Benefit
  - APPLICATIONS OF PHARMACOGENOMICS
    - Selecting the Patient Subpopulation
    - Avoiding Adverse Effects
    - Resurrecting Failed Drugs
    - Developing Diagnostics
  - PERSONALIZED VERSUS OFF-THE-SHELF MEDICATIONS
    - A Flood of New Orphan Drugs?

COMPANY PROFILES

ALLERGAN, INC.
- BUSINESS OVERVIEW
- FINANCIAL INFORMATION
- ORPHAN DRUG PRODUCTS
  - Botox™

Table 44 BOTOX CLINICAL TRIALS
Table 45 ALLERGAN'S COLLABORATIVE AGREEMENTS
- AMGEN, INC.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN DRUG PRODUCTS
    - Epogen™ and Neupogen™
Table 46 AMGEN'S PRODUCT PIPELINE
- BIOGEN, INC.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN DRUG PRODUCTS
    - Avonex™ for Multiple Sclerosis
Table 47 BIOGEN'S PRODUCT PIPELINE
- GENENTECH, INC.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN DRUG PRODUCTS
    - Protropin™
    - Nutropin™
    - Rituxan™
    - Expanded Indications for Products with Orphan Drug Status
  - CORPORATE STRATEGY
Table 48 SELECTED PRODUCTS FROM WHICH GENENTECH RECEIVES ROYALTIES
- GENZYME GENERAL
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN DRUG PRODUCTS
    - Aldurazyme™
    - Cerezyme™
    - Fabrazyme™
Table 49 GENZYME'S PRODUCT PIPELINE
Table 49 (CONTINUED)
- IDEC PHARMACEUTICALS CORP.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN PRODUCTS
    - Rituxan™
    - Zevalin™
    - Rituxan™/Zevalin™ Combination Therapy
  - CORPORATE STRATEGY
    - Strategic Alliances
Table 50 IDEC'S COLLABORATIVE ARRANGEMENTS
- IMMUNEX CORP.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN DRUG PRODUCTS
    - Products in Development
Table 51 IMMUNEX'S PRODUCT PIPELINE
- MILLENNIUM PHARMACEUTICALS, INC.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN PRODUCTS
  - CORPORATE STRATEGY
    - Corporate Strategy (Continued)
    - Corporate Strategy (Continued)
- NOVARTIS
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - OPRHAN DRUG PRODUCTS
  - CORPORATE STRATEGY
- ORPHAN MEDICAL, INC.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - PRODUCTS
    - Antizol™ (Fomepizole) for Injection
    - Busulfex™ (busulfan) for Injection
    - Cystadane™ (betaine Anhydrous for Oral Solution)
    - Elliots B Solution™ (Buffered Intrathecal Electrolyte/Dextrose Injection)
    - Sucraid™ (sacrosidase) Oral Solution
    - Xyrem™
    - Antizol-Vet (Fomepizole) for Injection)
    - Product Development
  - CORPORATE STRATEGY
    - Corporate Strategy (Continued)
    - Corporate Strategy (Continued)
    - Alliances
    - Acquisitions
- PFIZER CORP.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN DRUG PRODUCTS
    - Ellence ™
    - Genotropin™
    - Product Pipeline
  - CORPORATE STRATEGY
- RARE DISEASE THERAPEUTICS, INC.
  - BUSINESS OVERVIEW
  - ORPHAN PRODUCTS
  - CORPORATE STRATEGY
- SCHERING-PLOUGH CORP.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN DRUG PRODUCTS
Table 52 SCHERING-PLOUGH'S PRODUCT PIPELINE
Table 52 (CONTINUED)
- TEVA PHARMACEUTICAL INDUSTRIES, LTD.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN DRUG PRODUCT
  - CORPORATE STRATEGY
- TRANSKARYOTIC THERAPIES, INC.
  - BUSINESS OVERVIEW
  - FINANCIAL INFORMATION
  - ORPHAN PRODUCTS
Table 53 TRANSKARYOTIC THERAPIES' PRODUCT PIPELINE
Table 53 (CONTINUED)

LIST OF TABLES

Summary Table: GLOBAL SALES OF DRUGS APPROVED FOR ORPHAN DISEASES WITH SALES EXCEEDING $200 MILLION, 2002 THROUGH 2008 ($ MILLIONS)
Table 1 MEAN PATIENT POPULATION FOR DESIGNATED ORPHAN PRODUCTS AT THE TIME OF DESIGNATION, 1983-2008
Table 2 FDA ORPHAN DRUG MARKETING APPROVALS, TAX CREDIT, PRE-TAX EXPENDITURES AND PRE-TAX EXPENDITURES PER APPROVED DRUG, 1983-1993 ($MILLIONS)
Table 3 FDA ORPHAN DRUG MARKETING APPROVALS, TAX CREDITS AND PRE-TAX EXPENDITURES: 1995-2000
Table 4 FDA ORPHAN DRUG MARKETING APPROVALS: 1983-2002
Table 5 DRUGS AND BIOLOGICS APPROVED BY THE FDA FROM 1992 TO 2002: A COMPARISON OF PRODUCTS WITH AND WITHOUT ORPHAN DRUG STATUS
Table 6 GLOBAL ORPHAN DRUGS WITH SALES EXCEEDING $200 MILLION, 2002
Table 7 REGIONAL SALES OF DRUGS WITH ORPHAN DRUGS DISEASE INDICATIONS, THROUGH 2008 ($ MILLIONS)
Table 8 DRUGS WITH SALES EXCEEDING $200 MILLION APPROVED ONLY FOR ORPHAN INDICATIONS, 2002
Table 9 FORECAST OF LEADING BIOLOGICAL ORPHAN DRUG SALES, WORLDWIDE: THROUGH 2008 ($ MILLIONS)
Table 10 FORECAST OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG SALES, WORLDWIDE: THROUGH 2008 ($ MILLIONS)
Table 11 INCIDENCE, MORTALITY, AND SURVIVAL RATES FOR COMMON CANCERS: U.S., 2000
Table 12 CANCER PREVALENCE RATES: U.S., 2000
Table 13 APPROVED ORPHAN DRUGS TO TREAT CANCER AND CANCER-RELATED CONDITIONS
Table 14 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CANCER: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Table 15 APPROVED ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS
Table 16 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Table 17 APPROVED ORPHAN DRUGS TO TREAT DERMATOLOGICAL DISEASES
Table 18 APPROVED ORPHAN DRUGS TO TREAT GASTROINTESTINAL DISEASES
Table 19 GEOGRAPHIC DISTRIBUTION OF PATIENTS SUFFERING FROM HURLER DISEASE, 2003
Table 20 APPROVED ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS
Table 21 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Table 22 APPROVED ORPHAN DRUGS TO TREAT IMMUNE AND AUTOIMMUNE DISEASES/TRANSPLANTATION REJECTION
Table 23 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT IMMUNE DISEASES/TRANSPLANT REJECTION: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Table 24 APPROVED ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES
Table 25 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFECTIOUS DISEASES: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Table 26 APPROVED ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES
Table 27 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT INFLAMMATORY DISEASES: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Table 28 APPROVED ORPHAN DRUGS FOR NEUROLOGICAL DISEASES AND DISORDERS
Table 29 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Table 30 APPROVED ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS
Table 31 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS: WORLDWIDE, THROUGH 2008 ($ MILLIONS)
Table 32 APPROVED ORPHAN DRUGS FOR RESPIRATORY DISEASES
Table 33 APPROVED ORPHAN DRUGS FOR MISCELLANEOUS INDICATIONS
Table 34 COMPARISON OF NATIONAL MEASURES ON ORPHAN DRUGS IN THE E.U.
Table 35 DIFFERENCES IN ACCESS TO ORPHAN DRUGS IN VARIOUS EUROPEAN COUNTRIES
Table 36 COMPARISON OF ORPHAN DRUG LEGISLATION/PROVISIONS IN THE U.S., E.U., AUSTRALIA AND JAPAN
Table 37 GROWTH IN BIOLOGIC ORPHAN PRODUCTS, 1983-2000 (%)
Table 38 ORPHAN DRUG STATUS OF THE TOP 10 BIOTECH DRUGS IN 2002
Table 39 MARKETERS OF ORPHAN DRUGS AND NUMBER OF APPROVED INDICATIONS (AS OF 5/15/03)
Table 40 NUMBER OF PRODUCTS APPROVED FOR THE TREATMENT OF ORPHAN DISEASES-BIG PHARMA VERSUS BIOTECH: MAY 2003
Table 41 SALES OF DRUGS WITH ORPHAN STATUS, BY COMPANY, 2002 ($ MILLIONS)
Table 42 SALES OF LEADING PRODUCTS APPROVED FOR THE TREATMENT OF ORPHAN DISEASES-BIG PHARMA VERSUS BIOTECH, 2002 ($ MILLIONS)
Table 43 SELECTED VENTURE CAPITAL/PRIVATE EQUITY INVESTMENTS IN COMPANIES MARKETING ORPHAN DRUGS
Table 44 BOTOX CLINICAL TRIALS
Table 45 ALLERGAN'S COLLABORATIVE AGREEMENTS
Table 46 AMGEN'S PRODUCT PIPELINE
Table 47 BIOGEN'S PRODUCT PIPELINE
Table 48 SELECTED PRODUCTS FROM WHICH GENENTECH RECEIVES ROYALTIES
Table 49 GENZYME'S PRODUCT PIPELINE
Table 50 IDEC'S COLLABORATIVE ARRANGEMENTS
Table 51 IMMUNEX'S PRODUCT PIPELINE
Table 52 SCHERING-PLOUGH'S PRODUCT PIPELINE
Table 53 TRANSKARYOTIC THERAPIES' PRODUCT PIPELINE

LIST OF FIGURES

Summary Figure: GLOBAL SALES OF DRUGS APPROVED FOR ORPHAN DISEASES WITH SALES EXCEEDING $200 MILLION, 2002-2008 ($ MILLIONS)
Figure 1 REGIONAL SALES OF DRUGS WITH ORPHAN DRUGS DISEASE INDICATIONS, 2003 AND 2008 (%)
Figure 2 FORECAST OF LEADING BIOLOGICAL ORPHAN DRUG SALES, WORLDWIDE: 2002-2008 ($ MILLIONS)
Figure 3 FORECAST OF LEADING MONOCLONAL ANTIBODY ORPHAN DRUG SALES, WORLDWIDE: 2002-2008 ($ MILLIONS)
Figure 4 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS: WORLDWIDE, 2002-2008 ($ MILLIONS)
Figure 5 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT GENETIC DISEASES AND DISORDERS: WORLDWIDE, 2002-2008 ($ MILLIONS)
Figure 6 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT NEUROLOGICAL DISEASES AND DISORDERS: WORLDWIDE, 2002-2008 ($ MILLIONS)
Figure 7 THE MARKET FOR LEADING ORPHAN DRUGS TO TREAT REPRODUCTIVE DISEASES AND DISORDERS: WORLDWIDE, 2002-2008 ($ MILLIONS)

罕見疾病藥物的成功案例與市場策略

Orphan Drugs: Success Stories and Market Strategies

INTRODUCTION

STUDY GOAL AND OBJECTIVES

REASONS FOR DOING THE STUDY

CONTRIBUTIONS OF THE STUDY AND FOR WHOM

SCOPE AND FORMAT

METHODOLOGY

INFORMATION SOURCES

TABLE OF CONTENTS

LIST OF TABLES

LIST OF FIGURES