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1、,MARKET RESEARCH REPORT,GLOBAL MARKETS FOR,ORPHAN DRUGS,PHM038,Syamala Ariyanchira,Project Analyst,BCC Research,35 Walnut Street,Wellesley,MA 02481,Phone:866-285-7215(sales),Web Address:,E-mail:,ISBN:1-59623-605-1,ii,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,ABOUT BCC,BCC Research is

2、 a leading information resource producing high-quality market research reports,newsletters,and conferences.BCCs information products explore major market,economic,scientific,and technological developments for business leaders in industrial,pharmaceutical,and hightechnology organizations.Industry ana

3、lysis and market forecasts for advanced materials,high-techsystems and components,nanotechnology and novel processing methods are at the forefront of thecompanys expertise.For more than 35 years,BCCs market analysis has provided businesses withthe insight needed to make intelligent and strategic bus

4、iness decisions.BCC Research is a unit ofEli Research,which is based in Durham,N.C.CONDITIONS OF PURCHASE,The purchase of the bound or electronic copy of this report constitutes a contractual agreementbetween BCC Research and the purchaser.This report is intended for use solely by the person whomade

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8、ot covered in detail in this report.Please contact BCC regarding theterms and conditions of such services.,DISCLAIMER,This publication provides informative material of a professional nature.It does not constitutemanagerial,legal or accounting advice,nor should it serve as a corporate policy guide,la

9、boratorymanual or an endorsement of any product.This information is intended to be as accurate as possible atthe time of publication,but BCC Inc.assumes no responsibility for any losses or damages that mightresult because of reliance on this material.,Copyright 2010 by BCC Research Wellesley,MA 0248

10、1,Reproduction of any material in this report is strictly forbidden without express permission of thePublisher.,To receive a complete catalog of BCC studies,please visit our Web site at,JUNE 2010,Copyright BCC Research,Wellesley,MA USA,Web:,iii,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTEN

11、TS,CHAPTER ONE:INTRODUCTION.1STUDY GOAL AND OBJECTIVES.1REASONS FOR DOING THE STUDY.2INTENDED AUDIENCE.2SCOPE OF REPORT.3INFORMATION SOURCES AND METHODOLOGY.3AUTHORS CREDENTIALS.4RELATED REPORTS FROM BCC RESEARCH.4BCC ONLINE SERVICES.5DISCLAIMER.5CHAPTER TWO:SUMMARY.6SUMMARY.6SUMMARY TABLE GLOBAL OR

12、PHAN DRUGS DEMAND BY VALUE,THROUGH 2014($MILLIONS).7SUMMARY FIGURE GLOBAL ORPHAN DRUGS DEMAND BY VALUE,2008-2014($MILLIONS).7CHAPTER THREE:OVERVIEW.8DEFINITION OF AN ORPHAN DRUG.8HISTORY OF ORPHAN DRUG LEGISLATION.8FIRST LEGISLATION ON ORPHAN DRUGS.8Amendments to the ODA.8Bills Passed in 2002 Increa

13、sed Funding and Establishedthe Office of Rare Diseases.9Orphan Drug Legislation Global Status.9INCENTIVES FOR ORPHAN DRUG DEVELOPMENT.9THE NEED FOR INCENTIVES.10,TABLE 1 NUMBER OF ORPHAN DRUG DESIGNATIONS AND,APPROVALS PER YEAR BY U.S.FDA,1983-2009.10,APPLICATIONS FOR ORPHAN DRUG DESIGNATIONS HAVE,D

14、OUBLED.10PROFITABILITY OF ORPHAN DRUGS.11MARKET EXCLUSIVITY.11NO REGULATORY RESTRICTIONS ON PRICING.12OFF-LABEL USE.12CHALLENGES FOR EFFECTIVE USE OF THE ODA.12MARKET EXCLUSIVITY VERSUS PATENT PROTECTION.13MARKET SIZE AND KEY PLAYERS.14,TABLE 2 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKET,AND THEIR MA

15、RKET SHARES(%).14,Copyright BCC Research,Wellesley,MA USA,Web:,iv,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,THE ORPHAN DRUG ACT A BONUS FOR BIOTECH.15BIG PHARMA IS ENTERING THE ORPHAN DRUGS SECTOR,AGGRESSIVELY.15CONCERNS ABOUT THE ORPHAN DRUG ACT.16COST-EFFECTIVENESS.16PRICING OF ORP

16、HAN DRUGS.17,TABLE 3 SOME EXAMPLES OF HIGHLY EXPENSIVE ORPHAN,DRUGS AND THEIR PRICES.17TABLE 3(CONTINUED).18,HIGH-PRICED ORPHAN DRUGS LEADING TO PRICE,CONTROL MEASURES BY GOVERNMENTS.19TABLE 4 SOME OF THE RECENT CONTROVERSIES RELATED TOORPHAN DRUGS PRICING.20,ACCESS TO ORPHAN DRUGS AND REIMBURSEMENT

17、,CURRENT PRACTICES.21,ORPHAN DRUG ACCESS AND REIMBURSEMENT,SCENARIO IN THE U.S.21ACCESS AND REIMBURSEMENT SCENARIO IN EUROPE.21,TABLE 5 ORPHAN DRUG ACCESS AND REIMBURSEMENT,PRACTICES IN EUROPEAN COUNTRIES.22TABLE 5(CONTINUED).23TABLE 5(CONTINUED).24,CALL FOR UNIFICATION OF ORPHAN DRUG ACCESS,ACROSS

18、EUROPE.24,INNOVATIVE APPROACHES OF PRICING AND,REIMBURSEMENT.25TABLE 6 EMERGING TRENDS IN PRICING AND REIMBURSEMENTOF HIGH-PRICED DRUGS.25TABLE 6(CONTINUED).26,HOW EFFECTIVE ODA IS IN PROMOTING RESEARCH ON RAREDISEASES?.26ORPHAN DRUGS-BASED BUSINESS MODELS CASE STUDIES.27A NOT-FOR-PROFIT COMPANY.27A

19、 DISEASE FOUNDATION OPERATING AS A VIRTUAL,COMPANY.28TABLE 7 PARTNERSHIP DEALS SIGNED RECENTLY BETWEEN,CFFT AND PHARMACEUTICAL INDUSTRY.28TABLE 7(CONTINUED).29,A FOR-PROFIT COMPANY FOCUSED ON RARE DISEASES.29A SPECIALTY PHARMACY FOCUSING ON ULTRA-,ORPHAN DRUGS.29,Copyright BCC Research,Wellesley,MA

20、USA,Web:,v,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,A Specialty Pharmacy(Continued).30CHAPTER FOUR:WORLDWIDE INDUSTRY STRUCTURE.30GLOBAL AND REGIONAL SALES OF ORPHAN DRUGS.31,TABLE 8 GLOBAL PROJECTED SALES OF ORPHAN DRUGS BY,REGION,THROUGH 2014($MILLIONS).32,FIGURE 1 GLOBAL PROJECTE

21、D SALES OF ORPHAN DRUGS BYREGION,2008-2014($MILLIONS).32,CATEGORIES OF ORPHAN DRUG MARKET PLAYERS.32INDUSTRY TRENDS BY SPONSOR TYPES.33,ANALYSIS OF ORPHAN DRUG APPROVALS BY SPONSORCATEGORIES.33FIGURE 2 ANALYSIS OF ORPHAN DRUG APPROVALS BY SPONSORTYPES,1983-2009(%).34,ORPHAN DRUG APPROVALS BY COMPANI

22、ES.35TABLE 9 COMPANIES WITH MORE THAN FIVE ORPHAN DRUG,APPROVALS,1983-2009.35TABLE 9(CONTINUED).36ORPHAN DRUG REVENUES BY SPONSOR TYPES.36BIG PHARMA VERSUS BIOTECH COMPANIES.36TABLE 10 SALES OF LEADING ORPHAN DRUGS BIG PHARMAVERSUS BIOTECH COMPANIES,2009($MILLIONS).37TABLE 10(CONTINUED).38TABLE 10(C

23、ONTINUED).39,ORPHAN DRUGS NOW A STRATEGIC CHOICE FOR THE,PHARMA INDUSTRY.39LEADING PLAYERS IN THE GLOBAL ORPHAN DRUG MARKET.40,TABLE 11 TOP 10 COMPANIES IN THE ORPHAN DRUG MARKETAND THEIR REVENUES FROM ORPHAN DRUGS,2009.41CHAPTER FIVE:PRODUCTS.42ORPHAN DRUGS APPROVED BY THE U.S.FDA(1983 2009).42ORPH

24、AN DRUG DESIGNATIONS AND APPROVALS SINCE 1983.42TABLE 12 NUMBER OF ORPHAN DESIGNATIONS AND APPROVALSBY U.S.FDA,1983-2009.42TABLE 12(CONTINUED).43TABLE 13 ORPHAN DRUG MARKETING APPROVALS BY U.S.FDAPER YEAR,1983-2009.43TABLE 13(CONTINUED).44TABLE 13(CONTINUED).45,Copyright BCC Research,Wellesley,MA US

25、A,Web:,vi,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,ORPHAN DRUGS WITH SALES EXCEEDING$200 MILLION IN,2009.45TABLE 14 ORPHAN DRUGS WITH GLOBAL SALES EXCEEDING$200MILLION,2009.45TABLE 14(CONTINUED).46TABLE 14(CONTINUED).47,DETAILED ANALYSIS OF SUCCESSFUL AND PROMISING,ORPHAN DRUGS.47AL

26、IMTA.47,Additional indications.48Future Growth Trends.48TABLE 15 GLOBAL SALES OF ALIMTA,20042009($MILLIONS).49,AVASTIN.49,Key Indications.49,TABLE 16 KEY INDICATIONS FOR AVASTIN GRANTED BY VARIOUSREGULATORY AGENCIES.49TABLE 16(CONTINUED).50,Future Growth Trends.50AVONEX.50,Additional Approvals Gaine

27、d.50Market Strategies and Future Growth Trends.51TABLE 17 GLOBAL SALES OF AVONEX,2000-2009($MILLIONS).52,BETASERON.52,Additional Approvals Gained.52Marketing Strategies and Future Growth Trends.53TABLE 18 GLOBAL SALES OF BETASERON,2000-2009($MILLIONS).53,BOTOX.54,Marketing Strategies and Future Grow

28、th Trends.54TABLE 19 GLOBAL SALES OF BOTOX FOR THERAPEUTIC ANDCOSMETIC USAGES,2002-2009($MILLIONS).55,COPAXONE.55,Marketing Strategies.55TABLE 20 GLOBAL SALES OF COPAXONE,2002-2009($MILLIONS).56,ENBREL.56,Additional Indications.56Market Strategies.57,TABLE 21 U.S.AND INTERNATIONAL SALES OF ENBREL,20

29、03-2009($MILLIONS).57EPOGEN.58,Recent Threats to Market Share.58TABLE 22 GLOBAL SALES OF EPOGEN,2000-2009($MILLIONS).59,Copyright BCC Research,Wellesley,MA USA,Web:,vii,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,ERBITUX.59,Additional Indications.60Market Strategies and Future Growth T

30、rends.60TABLE 23 GLOBAL SALES OF ERBITUX,2004-2009($MILLIONS).61,EVISTA.61TABLE 24 GLOBAL SALES OF EVISTA,2003-2009($MILLIONS).62,GLEEVEC.62,Approval for Multiple Indications.62Market Strategy and Future Growth Trends.63TABLE 25 GLOBAL SALES OF GLEEVEC,2001-2009($MILLIONS).63,HUMIRA.63TABLE 26 GLOBA

31、L SALES OF HUMIRA,20042009($MILLIONS).64,INTRON A/PEGINTRON.64,Market Strategy and Future Growth Trends.65,TABLE 27 GLOBAL SALES OF INTRON A/PEGINTRON,2004-2009($MILLIONS).65,KOGENATE.65TABLE 28 GLOBAL SALES OF KOGENATE,2001-2009($MILLIONS).66,NEUPOGEN/NEULASTA.66,Additional Indications.66Marketing

32、Strategies.67,TABLE 29 GLOBAL SALES OF NEUPOGEN/NEULASTA,2002-2009($MILLIONS).67,NOVOSEVEN.67,TABLE 30 GLOBAL SALES OF NOVOSEVEN,2000-2009($,MILLIONS).68,PROGRAF.68,Additional Indications.68Future Prospects.69PROVIGIL.69REMICADE.70TABLE 31 GLOBAL SALES OF REMICADE 2001-2009($MILLIONS).70,REVLIMID.70

33、TABLE 32 GLOBAL SALES OF REVLIMID,2006-2009($MILLIONS).71,RITUXAN.71,Additional Indications.71Characteristics of Market.72TABLE 33 GLOBAL SALES OF RITUXAN,2000-2009($MILLIONS).72,SANDOSTATIN LAR.72TEMODAR.73TABLE 34 GLOBAL SALES OF TEMODAR,2003-2009($MILLIONS).73,Copyright BCC Research,Wellesley,MA

34、USA,Web:,viii,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,TOPAMAX.73,Additional Indications.74Characteristics of the Market and Future Prospects.74TABLE 35 GLOBAL SALES OF TOPAMAX,2001-2009($MILLIONS).74,TRACLEER.75TABLE 36 GLOBAL SALES OF TRACLEER,2002-2009($MILLIONS).75,VELCADE.75TAB

35、LE 37 GLOBAL SALES OF VELCADE,2006-2009($MILLIONS).76,ZOMETA.76,Additional Indications.76Market Characteristics.77TABLE 38 U.S.AND GLOBAL SALES OF ZOMETA,20022009($,MILLIONS).77ORPHAN DRUG SALES BY TECHNOLOGY.77ORPHAN BIOLOGICS.77,Global Sales and Forecasts of Orphan Biologics.78TABLE 39 SALES AND F

36、ORECASTS OF LEADING BIOLOGIC,ORPHAN DRUGS,WORLDWIDE,THROUGH 2014($MILLIONS).78,FIGURE 3 SALES AND FORECASTS OF LEADING BIOLOGIC,ORPHAN DRUGS,WORLDWIDE,2008-2014($MILLIONS).79,SUBCLASSES OF ORPHAN BIOLOGICS.79TABLE 40 SALES AND FORECASTS OF LEADING MONOCLONALANTIBODY ORPHAN DRUG SALES,WORLDWIDE,THROU

37、GH 2014($MILLIONS).80,FIGURE 4 SALES AND FORECASTS OF LEADING MONOCLONALANTIBODY ORPHAN DRUG WORLDWIDE,2008-2014($MILLIONS).80,TABLE 41 GLOBAL SALES AND FORECASTS OF LEADING,INTERFERONS WITH ORPHAN DRUG STATUS,THROUGH 2014($MILLIONS).81,FIGURE 5 GLOBAL SALES AND FORECASTS OF LEADING,INTERFERONS WITH

38、 ORPHAN STATUS,2008-2014($MILLIONS).81TABLE 42 GLOBAL SALES AND FORECASTS OF LEADING GROWTHHORMONES WITH ORPHAN DRUG STATUS,THROUGH 2014($,MILLIONS).82FIGURE 6 GLOBAL SALES AND FORECASTS OF LEADING GROWTH,HORMONES WITH ORPHAN DRUG STATUS,2008-2014($,MILLIONS).83TABLE 43 GLOBAL SALES AND FORECASTS OF

39、 LEADING PLASMAPRODUCT ORPHAN DRUG SALES,THROUGH 2014($MILLIONS).83,FIGURE 7 FORECAST OF LEADING PLASMA PRODUCT ORPHANDRUG SALES,2008-2014($MILLIONS).84,Copyright BCC Research,Wellesley,MA USA,Web:,ix,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,NONBIOLOGIC ORPHAN DRUGS.84,TABLE 44 GLOB

40、AL SALES AND FORECASTS OF LEADING,NONBIOLOGIC ORPHAN DRUGS,THROUGH 2014($MILLIONS).85FIGURE 8 GLOBAL SALES AND FORECASTS FOR NONBIOLOGICORPHAN DRUGS,2008-2014($MILLIONS).86FIGURE 9 ORPHAN DRUGS MARKET BY TECHNOLOGY TYPE,2009AND 2014(%).86FIGURE 9(CONTINUED).87CHAPTER SIX:APPLICATIONS.88CANCER.88LEUK

41、EMIAS.88,Acute Lymphocytic/Lymphoblastic Leukemia.89Acute Myelogenous/Myelocytic Leukemia.89Chronic Lymphocytic Leukemia.89Chronic Myelocytic/Myelogenous Leukemia.90LYMPHOMAS.90,Hodgkins Lymphoma.90Non-Hodgkins Lymphoma.91MULTIPLE MYELOMA.91SOLID TUMORS.91ACROMEGALY.92CANCER-RELATED CONDITIONS.92,TA

42、BLE 45 ORPHAN DRUGS APPROVED BY U.S.FDA TO TREAT,CANCER AND CANCER-RELATED CONDITIONS.93TABLE 45(CONTINUED).94TABLE 45(CONTINUED).95TABLE 45(CONTINUED).96TABLE 45(CONTINUED).97TABLE 45(CONTINUED).98TABLE 45(CONTINUED).99,SALES OF LEADING ORPHAN PRODUCTS TO TREAT,CANCER AND CANCER-RELATED CONDITIONS.

43、99TABLE 46 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TOTREAT CANCER,THROUGH 2014($MILLIONS).100FIGURE 10 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TOTREAT CANCER AND CANCER RELATED CONDITIONS,2008-2014($MILLIONS).101CARDIOVASCULAR DISEASES AND BLOOD DISORDERS.101ANEMIA.101HYPERCALCEMIA.102,Copyright BCC R

44、esearch,Wellesley,MA USA,Web:,x,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,HYPERTENSION.102HYPOTENSION.102NEUTROPENIA.102TACHYARRHYTHMIAS/TACHYCARDIA.103THROMBOCYTHEMIA.103THROMBOCYTOPENIA.103ORPHAN DRUGS APPROVED TO TREAT,CARDIOVASCULAR DISEASES AND BLOOD DISORDERS.103,TABLE 47 APPRO

45、VED ORPHAN DRUGS TO TREAT,CARDIOVASCULAR DISEASES AND BLOOD DISORDERS.104TABLE 47(CONTINUED).105,SALES OF LEADING ORPHAN PRODUCTS TO TREAT,CARDIOVASCULAR DISEASES AND BLOOD,DISORDERS:WORLDWIDE,THROUGH 2014.106TABLE 48 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TOTREAT CARDIOVASCULAR DISEASES AND BLOOD D

46、ISORDERS,THROUGH 2014($MILLIONS).106FIGURE 11 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TOTREAT CARDIOVASCULAR DISEASES AND BLOOD DISORDERS,2008-2014($MILLIONS).106GASTROINTESTINAL DISEASES.107CHOLELITHIASIS.107ESOPHAGEAL VARICES.107ORPHAN PRODUCTS APPROVED TO TREAT,GASTROINTESTINAL DISEASES.107,TABLE

47、49 APPROVED ORPHAN DRUGS TO TREAT,GASTROINTESTINAL DISEASES.108,SALES OF LEADING ORPHAN PRODUCTS TO TREAT,GASTROINTESTINAL DISEASES.108GENETIC DISEASES AND DISORDERS.108ALPHA-1 PROTEINASE DEFICIENCY.109FABRY DISEASE.109GAUCHERS DISEASE.110HEMOPHILIA.110MUCOPOLYSACCHARIDOSIS.111TYROSINEMIA.111ORPHAN

48、PRODUCTS APPROVED TO TREAT GENETIC,DISEASES AND DISORDERS.112TABLE 50 APPROVED ORPHAN DRUGS TO TREAT GENETIC,DISEASES AND DISORDERS.112TABLE 50(CONTINUED).113,Copyright BCC Research,Wellesley,MA USA,Web:,xi,PHM038C Global Markets for Orphan Drugs,TABLE OF CONTENTS,TABLE 50(CONTINUED).114TABLE 50(CON

49、TINUED).115,SALES OF LEADING ORPHAN PRODUCTS TO TREAT,GENETIC DISEASES AND DISORDERS.115TABLE 51 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TOTREAT GENETIC DISEASES AND DISORDERS,THROUGH 2014($MILLIONS).115FIGURE 12 GLOBAL MARKET FOR LEADING ORPHAN DRUGS TO,TREAT GENETIC DISEASES AND DISORDERS,2008-2014

50、($,MILLIONS).116IMMUNE DISEASES.116COMMON VARIABLE IMMUNODEFICIENCY.116INTERSTITIAL CYSTITIS.117JUVENILE RHEUMATOID ARTHRITIS.117ORGAN TRANSPLANT REJECTION.118ORPHAN PRODUCTS APPROVED TO TREAT IMMUNE AND,AUTOIMMUNE DISEASES AND TRANSPLANT,REJECTION.118TABLE 52 APPROVED ORPHAN DRUGS TO TREAT IMMUNE A