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Reportlinker Adds Global Markets for Orphan Drugs

Wednesday, August 11, 2010 Drug News J E 4
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NEW YORK, Aug. 10 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

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 (%)

To order this report:

Pharmaceutical Industry: Global Markets for Orphan Drugs

Pharmaceutical Business News

More Market Research Report

Check our Company Profile, SWOT and Revenue Analysis!

-- 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

SOURCE Reportlinker
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