CHAPTER FOUR: CLINICAL TRIAL COST AND FUNDING... 30

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1 CHAPTER ONE: INTRODUCTION... 1 STUDY GOALS AND OBJECTIVES... 1 REASONS FOR DOING THE STUDY... 1 INTENDED AUDIENCE... 2 METHODOLOGY... 2 ANALYST CREDENTIALS... 2 SCOPE OF REPORT... 2 INFORMATION SOURCES... 3 RELATED BCC REPORTS... 3 BCC ONLINE SERVICES... 3 DISCLAIMER... 4 CHAPTER TWO: SUMMARY... 5 SUMMARY... 5 SUMMARY TABLE U.S. FORECAST OF SPONSORED CLINICAL TRIALS MARKET, THROUGH SUMMARY FIGURE U.S. FORECAST OF SPONSORED CLINICAL TRIALS MARKET, ($ BILLIONS)... 7 CHAPTER THREE: OVERVIEW... 8 TYPES OF CLINICAL TRIALS... 8 CLINICAL TRIAL PHASES... 8 WHO SPONSORS CLINICAL TRIALS?... 8 TABLE 1 U.S. FORECAST OF SPONSORED CLINICAL TRIALS, THROUGH FIGURE 1 U.S. SPONSORED CLINICAL TRIALS MARKET, ($ BILLIONS)... 9 FIGURE 2 DRUG DEVELOPMENT TIME LINE TABLE 2 PHASES OF THE CLINICAL TRIAL PHASE I PHASE II PHASE III PHASE IV/POST-MARKETING TRIALS FACTORS AFFECTING STUDY SIZE PHYSICIAN AND PATIENT RECRUITMENT U.S. GOVERNMENT REGULATIONS PHARMACEUTICALS AND BIOLOGICS TABLE 3 U.S. LAWS AFFECTING DRUG APPROVAL TIMES Additional U.S. Laws Affecting Clinical Trials Health Insurance Portability and Accountability Act of 1996 (HIPAA) Best Pharmaceuticals for Children Act (BPCA),

2 TABLE 4 TOTAL APPROVED ACTIVE MOIETIES WITH NO MARKETING PROTECTION FOR WHICH FDA HAS ISSUED A WRITTEN REQUEST FOOD AND DRUG ADMINISTRATION (FDA) Background Center for Biologics Evaluation and Research (CBER) CBER and Bioterrorism Center for Drug Evaluation and Research (CDER) CDER and Bioterrorism Critical Path Initiative (CPI) MEDICAL DEVICES Center for Devices and Radiologic Health (CDRH) Articles Subject to Medical Device Amendments Classification of Devices (General Section 513) Class I General Controls Class II Special Controls Class III Expedited Review FIGURE 3 NEW DRUG DEVELOPMENT TABLE 5 CDER APPROVAL TIMES FOR PRIORITY AND STANDARD NDAS AND BLAS, CALENDAR YEARS, Expedited Development and Review TABLE 6 FIRST CYCLE APPROVAL RATE FOR OVERALL STUDY COHORT BY YEAR, Subpart E Accelerated Approval (Subpart H) Priority Review Fast Track TABLE 7 NUMBER AND TOTAL APPROVAL TIME (IN MONTHS) OF APPROVED NDAS AND BLAS, BY FISCAL YEAR OF SUBMISSION AND BY REVIEW PROCEDURE Pediatrics U.S. LITIGATION AND CLINICAL TRIALS TABLE 8 INSURANCE COMPANIES OFFERING CLINICAL TRIAL LIABILITY INSURANCE INVESTIGATIONAL REVIEW BOARD (IRB) EUROPEAN REGULATIONS JAPANESE REGULATIONS GLOBAL REGULATORY STANDARDS INTERNATIONAL CONFERENCE OF HARMONIZATION (ICH) International Conference of Harmonization (Continued) CHAPTER FOUR: CLINICAL TRIAL COST AND FUNDING... 30

3 MANAGING CLINICAL TRIALS CONTRACT RESEARCH ORGANIZATIONS (CROS) CLINICAL TRIAL RESEARCH THERAPEUTIC AREAS FIGURE 4 PHRMA MEMBER R&D FUNDING BY THERAPEUTIC CATEGORY, 2010 (% SHARE) TABLE 9 BEST-SELLING PHARMACEUTICAL DRUGS WORLDWIDE, 2009 ($ BILLIONS) TRENDS IN CLINICAL TRIALS ELECTRONIC DATA CAPTURE (EDC) CLINICAL TRIAL COSTS FACTORS INFLUENCING CLINICAL TRIAL COSTS Factors Increasing Clinical Trial Costs Factors Increasing Clinical (Continued) Factors Decreasing Clinical Trial Costs RECENT AND FUTURE TRENDS IN CLINICAL TRIAL PROCESSES RESEARCH AND DEVELOPMENT BUDGETS TABLE 10 INVESTMENT IN R&D AS A PERCENT OF SALES, PHARMACEUTICAL INDUSTRY, THROUGH 2014 (%) TABLE 11 INVESTMENT IN R&D AS A PERCENT OF SALES, PHARMACEUTICAL INDUSTRY, THROUGH 2014, PHRMA MEMBERS, (%) FUNDING CLINICAL TRIALS TABLE 12 TOTAL R&D SPENDING BY SOURCE, THROUGH 2014 ($ BILLIONS) TABLE 13 ESTIMATES OF NIH RESEARCH FUNDING FOR TOP 50 CONDITIONS AND DISEASE CATEGORIES, RESEARCH/DISEASE AREAS, THROUGH 2010 ($ MILLIONS/ROUNDED) BIOTECH/PHARMACEUTICAL COMPANIES TABLE 14 U.S. MARKET FORECAST OF REGISTERED CLINICAL TRIALS SPONSORED BY INDUSTRY BY PHASE, THROUGH 2014 (RECRUITING SUBJECTS) NATIONAL INSTITUTES OF HEALTH AND OTHER FEDERAL AGENCIES OF THE FEDERAL GOVERNMENT TABLE 15 U.S. MARKET FORECAST OF REGISTERED CLINICAL TRIALS SPONSORED BY FEDERAL GOVERNMENT BY PHASE, THROUGH 2014 (RECRUITING SUBJECTS) PRIVATE ORGANIZATIONS, UNIVERSITIES, AND SPECIALTY GROUPS TABLE 16 U.S. MARKET FORECAST OF REGISTERED CLINICAL TRIALS SPONSORED BY UNIVERSITIES OR OTHER ORGANIZATIONS BY PHASE, THROUGH 2014 (RECRUITING SUBJECTS)... 44

4 TABLE 17 TOP 25 FOUNDATIONS AWARDING U.S.-FOCUSED GRANTS FOR GLOBAL HEALTH, RANKED BY NUMBER OF GRANTS AND DOLLAR AMOUNT, Private Organizations, Universities (Continued) CHAPTER FIVE: CLINICAL TRIAL PIPELINES AND TECHNOLOGY TRENDS DRUGS BY SYSTEM CARDIOVASCULAR PhRMA Facts PhRMA Facts (Continued) TABLE 18 SELECTED CARDIOVASCULAR THERAPEUTICS IN DEVELOPMENT, CANCER PhRMA Facts PhRMA Facts (Continued) TABLE 19 SELECTED ANTICANCER CRUGS IN DEVELOPMENT, CENTRAL NERVOUS SYSTEM PhRMA Facts TABLE 20 SELECTED CENTRAL NERVOUS SYSTEM THERAPEUTICS IN DEVELOPMENT, INFECTION INFLAMMATION RESPIRATORY TABLE 21 CDER PRIORITY DRUG AND BIOLOGIC APPROVALS: CALENDAR YEAR TABLE 21 (CONTINUED) DEVICES BIOMEMS STENTS IMPLANTS TABLE 22 MEDICAL DEVICES CLEARED OR APPROVED BY FDA IN TABLE 22 (CONTINUED) TABLE 23 TOP MEDICAL DEVICE COMPANIES, IN TERMS OF 2009 REVENUES ($ MILLIONS) BIOTECHNOLOGY VERSUS PHARMACEUTICAL BIOLOGICS TABLE 24 SELECTED BIOLOGICS IN DEVELOPMENT, TABLE 25 BIOTECHNOLOGY CANDIDATES IN DEVELOPMENT BY MODALITY, TABLE 26 BIOTECHNOLOGY CANDIDATES IN DEVELOPMENT BY INDICATION, TECHNOLOGIES... 62

5 TABLE 27 NUMBER OF CLINICAL TRIALS USING NEW TECHNOLOGIES, THROUGH TABLE 28 NEW TECHNOLOGIES AND PROJECTED EFFECT ON CLINICAL TRIALS ANTISENSE APOPTOSIS BIOCHIPS/MICROARRAYS BIOINFORMATICS BIOPROCESSORS CELLS AND CELL SIGNALING CELL THERAPY (I.E., STEM CELLS, TISSUE REPAIR) CHEMICAL GENOMICS CYTOMICS ELECTRONIC DATA CAPTURE AND E-SUBMISSION GENE THERAPY GENOMICS NOVEL DRUG DELIVERY SYSTEMS PROTEOMICS Four-part Definition of Proteomics THERANOSTICS TRANSGENICS NANOTECHNOLOGY NATURAL SOURCES BIOMARKERS CONSORTIUM NIH Public-Private Partnerships Policy Statement FACTORS INFLUENCING DRUG DEVELOPMENT FIGURE 5 FACTORS INFLUENCING DRUG DEVELOPMENT EPIDEMIOLOGICAL TRENDS LEGISLATION NEW TECHNOLOGIES BIOTERRORISM CHAPTER SIX: CONTRACT RESEARCH ORGANIZATIONS AND GLOBALIZATION CONTRACT RESEARCH ORGANIZATIONS AND GLOBALIZATION CONTRACT RESEARCH ORGANIZATIONS (CONTINUED) ASSOCIATION OF CLINICAL RESEARCH ORGANIZATIONS FACTS ASSOCIATION OF CLINICAL RESEARCH (CONTINUED) TABLE 29 TOP TEN CROS BY REVENUES, GLOBALIZATION TABLE 30 CLINICAL TRIALS WITH OPEN ENROLLMENT BY REGION, JULY OUTSOURCING CLINICAL TRIALS TO FOREIGN COUNTRIES... 78

6 TABLE 31 CLINICAL TRIALS RECRUITING SUBJECTS IN TARGETED COUNTRIES, JULY TABLE 31 (CONTINUED) TABLE 32 AVERAGE COST PER PATIENT FOR TRIALS IN THE U.S., CANADA, AND WESTERN EUROPE FOR ALL THERAPEUTIC AREAS, THROUGH 2014 ($) Advantages Costs TABLE 33 AVERAGE COST PER PATIENT FOR TRIALS IN REDUCED COST COUNTRIES, THROUGH 2014 ($) Efficiency Disadvantages Sociopolitical Issues BRAZIL Patent Protection Pharmaceutical and Clinical Trial Registration Clinical Trials TABLE 34 BRAZILIAN DOMESTIC PHARMACEUTICAL COMPANIES CHINA Patent Protection Pharmaceutical and Clinical Trial Registration Clinical Trials Leading Chinese Pharmaceutical Companies Beijing Double-Crane Pharmaceutical (DCPC) Northeast General Pharmaceutical Sanjiu Medical and Pharmaceutical Co., Ltd C & O Pharmaceutical Technology Holdings Ltd Shijiazhuang Pharma Group Wuxi Pharmatech Harbin Pharmaceutical Group Co INDIA Advantages Disadvantages Key Points for Doing Clinical Studies in India TABLE 35 INDIAN DOMESTIC PHARMACEUTICAL PRODUCERS TABLE 36 INDIA S DOMESTIC PHARMACEUTICAL MARKET (12 MONTHS ENDED JANUARY 2009) TABLE 37 TOP FIVE BIOTECHNOLOGY COMPANIES IN INDIA ROMANIA Patent Protection RUSSIA Patent Protection TABLE 38 LEADING RUSSIAN PHARMACEUTICAL COMPANIES CHAPTER SEVEN: BIOTECHNOLOGY COMPANIES... 89

7 ACTELION PHARMACEUTICALS, LTD TABLE 39 CLINICAL PIPELINE AS OF 12/31/ ALEXION PHARMACEUTICALS, INC TABLE 40 CLINICAL PIPELINE AS OF 12/31/ ALLERGAN, INC TABLE 41 CLINICAL PIPELINE AS OF 12/31/ TABLE 41 (CONTINUED) ALSERES PHARMACEUTICALS, INC TABLE 42 CLINICAL PIPELINE AS OF 12/31/ AMGEN TABLE 43 CLINICAL PIPELINE AS OF 12/31/ TABLE 43 (CONTINUED) ARQULE TABLE 44 CLINICAL PIPELINE AS OF 12/31/ ARRAY BIOPHARMA TABLE 45 CLINICAL PIPELINE AS OF 12/31/ AVANIR PHARMACEUTICALS TABLE 46 CLINICAL PIPELINE AS OF 12/31/ BAXTER BIOSCIENCE TABLE 47 SAMPLE OF BAXTER S CLINICAL TRIALS IN PROGRESS, AUGUST TABLE 47 (CONTINUED) TABLE 47 (CONTINUED) BIOCON TABLE 48 CLINICAL PIPELINE AS OF 12/31/ BIOGEN IDEC TABLE 49 CLINICAL PIPELINE AS OF 12/31/ TABLE 49 (CONTINUED) CELGENE CORP TABLE 50 CLINICAL PIPELINE AS OF 12/31/ CELL THRAPEUTICS TABLE 51 CLINICAL PIPELINE AS OF 12/31/ CELLDEX THERAPEUTICS TABLE 52 CLINICAL PIPELINE AS OF 12/31/ CEPHALON, INC TABLE 53 CLINICAL PIPELINE AS OF 12/31/ CSL BEHRING (CSL, LTD) TABLE 54 CLINICAL PIPELINE AS OF 12/31/ CYTOKINETICS TABLE 55 CLINICAL PIPELINE AS OF 12/31/ DENDREON TABLE 56 CLINICAL PIPELINE AS OF 12/31/ DYNAVAX TECHNOLOGIES TABLE 57 CLINICAL PIPELINE AS OF 12/31/

8 ELAN TABLE 58 CLINICAL PIPELINE AS OF 12/31/ ENZON PHARMACEUTICALS TABLE 59 CLINICAL PIPELINE AS OF 12/31/ EPICEPT CORPORATION TABLE 60 CLINICAL PIPELINE AS OF 12/31/ GENENTECH TABLE 61 CLINICAL PIPELINE AS OF 12/31/ TABLE 61 (CONTINUED) GENZYME TABLE 62 CLINICAL PIPELINE AS OF 12/31/ GILEAD TABLE 63 CLINICAL PIPELINE AS OF 12/31/ GTC BIOTHERAPEUTICS, INC TABLE 64 CLINICAL PIPELINE AS OF 12/31/ GTX TABLE 65 CLINICAL PIPELINE AS OF 12/31/ HUMAN GENOME SCIENCES TABLE 66 CLINICAL PIPELINE AS OF 12/31/ IDERA PHARMACEUTICALS, INC TABLE 67 CLINICAL PIPELINE AS OF 12/31/ IMMUNOGEN, INC TABLE 68 CLINICAL PIPELINE AS OF 12/31/ IMMUNOMEDICS, INC TABLE 69 CLINICAL PIPELINE AS OF 12/31/ LORUS THERAPEUTICS INC TABLE 70 CLINICAL PIPELINE AS OF 12/31/ MAXYGEN TABLE 71 CLINICAL PIPELINE AS OF 12/31/ MYRIAD PHARMACEUTICALS, INC TABLE 72 CLINICAL PIPELINE AS OF 12/31/ NOVO NORDISK A/S TABLE 73 CLINICAL PIPELINE AS OF 12/31/ NPS PHARMACEUTICALS, INC TABLE 74 CLINICAL PIPELINE AS OF 12/31/ REPLIGEN CORPORATION TABLE 75 CLINICAL PIPELINE AS OF 12/31/ SANGAMO BIOSCIENCES, INC TABLE 76 CLINICAL PIPELINE AS OF 12/31/ SEATTLE GENETICS, INC TABLE 77 CLINICAL PIPELINE AS OF 12/31/ TALECRIS BIOTHERAPEUTICS, INC TABLE 78 CLINICAL PIPELINE AS OF 12/31/ UCB SA

9 TABLE 79 CLINICAL PIPELINE AS OF 12/31/ URIGEN PHARMACEUTICALS, INC TABLE 80 CLINICAL PIPELINE AS OF 12/31/ VERTEX PHARMACEUTICALS INCORPORATED TABLE 81 CLINICAL PIPELINE AS OF 12/31/ VICAL, INCORPORATED TABLE 82 CLINICAL PIPELINE AS OF 12/31/ XOMA, LTD TABLE 83 CLINICAL PIPELINE AS OF 12/31/ CHAPTER EIGHT: PHARMACEUTICAL COMPANIES ABBOTT LABORATORIES TABLE 84 CLINICAL PIPELINE HIGHLIGHTS AS OF 8/ ASTRAZENECA PLC TABLE 85 CLINICAL PIPELINE AS OF 12/31/ TABLE 85 (CONTINUED) TABLE 85 (CONTINUED) TABLE 85 (CONTINUED) BAYER SCHERING PHARMA AG TABLE 86 PHASE II/III CLINICAL PIPELINE AS OF 12/31/ TABLE 86 (CONTINUED) BOEHRINGER INGELHEIM GMBH TABLE 87 CLINICAL PIPELINE AS OF 12/31/ BRISTOL-MYERS SQUIBB COMPANY TABLE 88 CLINICAL PIPELINE, SELECTED PHASE I/II CANDIDATES AS OF 12/31/ TABLE 88 (CONTINUED) TABLE 89 INVESTIGATIONAL CANDIDATES IN PHASE III CLINICAL TRIALS ELI LILLY AND COMPANY TABLE 90 CLINICAL PIPELINE HIGHLIGHTS AS OF 8/ GLAXOSMITHKLINE PLC TABLE 91 CLINICAL PIPELINE AS OF 12/31/ TABLE 91 (CONTINUED) TABLE 91 (CONTINUED) TABLE 91 (CONTINUED) TABLE 91 (CONTINUED) JOHNSON & JOHNSON TABLE 92 SELECTED PHASE III CLINICAL PIPELINE AS OF 12/31/ MERCK & CO., INC TABLE 93 CLINICAL PIPELINE AS OF 12/31/ TABLE 93 (CONTINUED) MERCK SERONO TABLE 94 CLINICAL PIPELINE AS OF 12/31/

10 NOVARTIS AG TABLE 95 CLINICAL PIPELINE AS OF 12/31/ TABLE 95 (CONTINUED) TABLE 95 (CONTINUED) TABLE 95 (CONTINUED) TABLE 95 (CONTINUED) PFIZER, INC TABLE 96 CLINICAL PIPELINE AS OF 12/31/ TABLE 96 (CONTINUED) TABLE 96 (CONTINUED) TABLE 96 (CONTINUED) ROCHE TABLE 97 CLINICAL PIPELINE AS OF 12/31/ TABLE 97 (CONTINUED) TABLE 97 (CONTINUED) SANOFI-AVENTIS TABLE 98 CLINICAL PIPELINE AS OF 12/31/ TABLE 98 (CONTINUED) TAKEDA PHARMACEUTICAL COMPANY LIMITED TABLE 99 CLINICAL PIPELINE AS OF 12/31/ TABLE 99 (CONTINUED) TABLE 99 (CONTINUED) TABLE 99 (CONTINUED) CHAPTER NINE: MEDICAL EQUIPMENT AND DEVICES TABLE 100 MEDICAL EQUIPMENT AND DEVICE COMPANIES IN THE U.S. WITH ANNUAL REVENUES EXCEEDING $1 BILLION TABLE 100 (CONTINUED) TABLE 100 (CONTINUED) TABLE 100 (CONTINUED) CHAPTER TEN: CLINICAL RESEARCH ORGANIZATIONS TOP 10 CROS TABLE 101 TOP 10 CROS BY REVENUE (ACCOUNTED FOR MORE THAN 50% OF TOTAL INDUSTRY REVENUE IN 2008/2009) SECONDARY CROS (ALPHABETICAL ORDER) TABLE 102 SECONDARY CROS (ALPHABETICAL ORDER) TABLE 102 (CONTINUED) NICHE CRO SPECIALIZED BY REGION OR THERAPEUTIC FOCUS (ALPHABETICAL ORDER) TABLE 103 NICHE CRO SPECIALIZED BY REGION OR THERAPEUTIC FOCUS (ALPHABETICAL ORDER) TABLE 103 (CONTINUED) TABLE 103 (CONTINUED) TABLE 103 (CONTINUED)

11 TABLE 103 (CONTINUED) TABLE 103 (CONTINUED)