Global Markets for Orphan Drugs

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Global Markets for Orphan Drugs

Report price : $ 4 850
Publication date : September 2007

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 BCC report provides an in-depth look at orphan drugs that had sales of more than $200 million in 2006. Future growth prospects of these drugs as well as the therapeutic areas of these drugs in general have been analyzed based on the market dynamics and regulatory factors. Life stories of blockbuster orphan drugs are tracked and analyzed in detail, recognizing various factors that played key roles in their success. Regional analysis of the industry in terms of current and future trends of the orphan drug market in important regions has been presented in the study. The report also provides revenues of leading orphan products, identifies key market players with significant market shares, analyzes current and future strategies of those companies that will have an impact in the market, and, finally, studies the rare diseases that are being addressed by the industry and the broad therapeutic areas that these diseases represent and their future growth trends.

This report also 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.

INTRODUCTION STUDY GOAL AND OBJECTIVES REASONS FOR DOING THE STUDY INTENDED AUDIENCE SCOPE OF REPORT METHODOLOGY INFORMATION SOURCES AUTHOR'S CREDENTIALS RELATED REPORTS FROM BCC RESEARCH BCC ONLINE SERVICES DISCLAIMER

SUMMARY

Summary Table:

GLOBAL ORPHAN DRUGS DEMAND BY VALUE, THROUGH 2011 ($ MILLIONS)

Summary Figure:

GLOBAL ORPHAN DRUGS DEMAND BY VALUE, 2005-2011 ($ MILLIONS)

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

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)

ORPHAN DRUGS - A STRATEGIC CHOICE BY BIOTECH COMPANIES

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

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)

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

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)

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)

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

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)

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)

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)

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)

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 (%)

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)

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

TECHNOLOGY TRENDS ANALYSIS LEADING ORPHAN DRUGS AND RELATED PATENTS

Table 64 LEADING ORPHAN DRUGS AND THEIR RELEVANT U.S. PATENTS

Table 64 (CONTINUED)

TECHNOLOGY TRENDS ANALYSIS BASED ON ORPHAN DRUG APPROVALS

GENERAL TRENDS

Table 65 BIOLOGICS 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

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

LIST OF TABLES