Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 3 | 5 |
| List of Tables | 6 | 1 |
| List of Figures | 7 | 1 |
| Endpoints-Clinical Trials in Orphan Diseases - Introduction | 8 | 1 |
| Disease Overview | 8 | 1 |
| The US | 8 | 1 |
| Europe | 8 | 1 |
| Japan | 8 | 1 |
| GBI Research Report Guidance | 8 | 1 |
| Endpoints-Clinical Trials in Orphan Diseases - Design of Orphan Diseases Clinical Trials and its Outcomes | 9 | 1 |
| Clinical Trial Design - Need For Adaptive Trial Designs In Orphan Diseases | 9 | 1 |
| Endpoints-Clinical Trials in Orphan Diseases: An Overview on Endpoints | 10 | 2 |
| Types of Clinical Trial Outcomes | 10 | 1 |
| Oncology Endpoints | 10 | 1 |
| Survival | 10 | 1 |
| Progression-free survival (PFS) | 10 | 1 |
| Time-to-Tumor Progression (TTP) | 10 | 1 |
| Disease-Free Survival (DFS) | 10 | 1 |
| Other Endpoints | 10 | 1 |
| UHDRS motor score | 10 | 1 |
| Clinical Global Impression Score | 11 | 1 |
| Endpoints-Clinical Trials in Orphan Diseases - Marketed and Pipeline Products Assessment | 12 | 91 |
| Huntington s disease | 12 | 1 |
| Primary Endpoints used in Huntington s disease Clinical Trials | 12 | 1 |
| Secondary Endpoints used in Huntington s disease Clinical Trials | 12 | 1 |
| Major Marketed Drugs-Safety and Efficacy Analysis | 12 | 1 |
| Xenazine | 12 | 1 |
| Phase III Clinical Trial Analysis | 13 | 1 |
| Phase III Primary Endpoint analysis | 14 | 1 |
| Phase III Secondary Endpoint analysis | 15 | 1 |
| Most Promising Drugs Profiles | 16 | 1 |
| Dimebon | 16 | 1 |
| Phase II Clinical Trial Analysis | 16 | 1 |
| Phase II Primary Endpoint analysis | 16 | 1 |
| Phase II Secondary Endpoint analysis | 17 | 1 |
| Terminated Trials Analysis | 17 | 1 |
| Acute Myelocytic Leukemia | 18 | 1 |
| Overview | 18 | 1 |
| Primary Endpoints used in Acute Myelocytic Leukemia Clinical Trials | 18 | 1 |
| Secondary Endpoints used in Acute Myelocytic Leukemia Clinical Trials | 19 | 1 |
| Major Marketed Drugs-Safety and Efficacy Analysis | 20 | 1 |
| Cytarabine (cytosine arabinoside) | 20 | 1 |
| Daunorubicin | 20 | 1 |
| Idarubicin | 20 | 1 |
| Mitoxantrone | 20 | 1 |
| Phase III Clinical Trial Analysis | 21 | 1 |
| Phase III Primary Endpoint analysis | 22 | 1 |
| Phase III Secondary Endpoint analysis | 23 | 1 |
| Most Promising Drugs Profiles | 24 | 1 |
| Clolar (clofarabine) | 24 | 1 |
| Quizartinib (AC220) | 24 | 1 |
| Phase II Clinical Trial Analysis | 25 | 4 |
| Phase II Primary Endpoint analysis | 29 | 1 |
| Phase II Secondary Endpoint analysis | 30 | 1 |
| Terminated Trial Analysis | 31 | 1 |
| Amyotrophic Lateral Sclerosis | 32 | 1 |
| Overview | 32 | 1 |
| Primary Endpoints used in Amyotrophic Lateral Sclerosis Clinical Trials | 32 | 1 |
| Secondary Endpoints used in Amyotrophic Lateral Sclerosis Clinical Trials | 33 | 1 |
| Major Marketed Drugs-Safety and Efficacy Analysis | 33 | 1 |
| Rilutek (Riluzole) | 33 | 1 |
| Phase III Clinical Trial Analysis | 33 | 1 |
| Phase III Primary Endpoint analysis | 34 | 1 |
| Phase III Secondary Endpoint analysis | 35 | 1 |
| Most Promising Drugs Profiles | 35 | 1 |
| Olesoxime (TRO 19622) | 35 | 1 |
| Phase II Clinical Trial Analysis | 36 | 1 |
| Phase II Primary Endpoint analysis | 37 | 1 |
| Phase II Secondary Endpoint analysis | 38 | 1 |
| Terminated Trials Analysis | 38 | 1 |
| Hodgkin Lymphoma | 39 | 1 |
| Primary Endpoints used in Hodgkin Lymphoma Clinical Trials | 39 | 1 |
| Secondary Endpoints used in Hodgkin Lymphoma Clinical Trials | 39 | 1 |
| Major Marketed Drugs-Safety and Efficacy Analysis | 40 | 1 |
| Intron A | 40 | 1 |
| Phase III Clinical Trial Analysis | 40 | 1 |
| Phase III Primary Endpoint analysis | 41 | 1 |
| Phase III Secondary Endpoint analysis | 42 | 1 |
| Most Promising Drugs Profiles | 43 | 1 |
| Brentuximab Vedotin (SGN-35) | 43 | 1 |
| Phase II Clinical Trial Analysis | 44 | 1 |
| Phase II Primary Endpoint analysis | 45 | 1 |
| Phase II Secondary Endpoint analysis | 46 | 1 |
| Terminated Trials Analysis | 47 | 1 |
| Multiple Myeloma | 47 | 1 |
| Overview | 47 | 1 |
| Primary Endpoints used in Multiple Myeloma Clinical Trials | 47 | 1 |
| Secondary Endpoints used in Multiple Myeloma Clinical Trials | 48 | 1 |
| Major Marketed Drugs-Safety and Efficacy Analysis | 48 | 1 |
| Velcade (bortezomib) with Melphalan and Prednisone | 48 | 1 |
| Revlimid (lenalidomide) with Dexamethasone | 49 | 1 |
| Thalomid (thalomide) with Dexamethasone | 50 | 1 |
| Doxil (doxorubicin HCl liposome injection) with Bortezomib | 50 | 1 |
| Mozobil (plerixafor injection) with Granulocyte colony-stimulating factor (G-CSF) | 51 | 1 |
| Phase III Clinical Trial Analysis | 52 | 4 |
| Phase III Primary Endpoint analysis | 56 | 1 |
| Phase III Secondary Endpoint analysis | 57 | 1 |
| Most Promising Drugs Profiles | 58 | 1 |
| Actimid (pomalidomide) | 58 | 1 |
| Carfilzomib | 58 | 1 |
| Phase II Clinical Trial Analysis | 59 | 5 |
| Phase II Primary Endpoint Analysis | 64 | 1 |
| Phase II Secondary Endpoint analysis | 65 | 1 |
| Terminated Trials Analysis | 66 | 2 |
| Ovarian Cancer | 68 | 1 |
| Overview | 68 | 1 |
| Primary Endpoints used in Ovarian Cancer Clinical Trials | 68 | 1 |
| Secondary Endpoints used in Ovarian Cancer Clinical Trials | 68 | 1 |
| Major Marketed Drugs-Safety and Efficacy Analysis | 69 | 1 |
| Altretamine (hexalen) | 70 | 1 |
| Yondelis | 70 | 1 |
| Gemzar (gemcitabine) | 71 | 1 |
| Hycamtin (topotecan hydrochloride) | 72 | 3 |
| Phase III Clinical Trial Analysis | 75 | 2 |
| Phase III Primary Endpoint analysis | 77 | 1 |
| Phase III Secondary Endpoint analysis | 77 | 1 |
| Most Promising Drugs Profiles | 78 | 1 |
| Farletuzumab (MORAb-003) | 78 | 1 |
| Abagovomab | 78 | 1 |
| Phase II Clinical Trial Analysis | 79 | 6 |
| Phase II Primary Endpoint analysis | 85 | 1 |
| Phase II Secondary Endpoint analysis | 86 | 1 |
| Terminated Trials Analysis | 87 | 1 |
| Pancreatic Cancer | 88 | 1 |
| Overview | 88 | 1 |
| Primary Endpoints used in Pancreatic Cancer Clinical Trials | 88 | 1 |
| Secondary Endpoints used in Pancreatic Cancer Clinical Trials | 88 | 1 |
| Major Marketed Drugs-Safety and Efficacy Analysis | 89 | 1 |
| Gemzar | 89 | 1 |
| Gemcitabine Based Combinations | 89 | 1 |
| Gemcitabine + 5-FU | 90 | 1 |
| Gemcitabine + Platinum Analogues | 90 | 1 |
| Gemcitabine + Topoisomerase I Inhibitors | 90 | 1 |
| Tarceva | 90 | 2 |
| Phase III Clinical Trial Analysis | 92 | 2 |
| Phase III Primary Endpoint analysis | 94 | 1 |
| Phase III Secondary Endpoint analysis | 95 | 1 |
| Most Promising Drugs Profiles | 96 | 1 |
| Mesupron | 96 | 1 |
| Phase II Clinical Trial Analysis | 97 | 3 |
| Phase II Primary Endpoint Analysis | 100 | 1 |
| Phase II Secondary Endpoint analysis | 101 | 1 |
| Terminated Trials Analysis | 102 | 1 |
| Endpoints-Clinical Trials in Orphan Diseases - Company Profiling | 103 | 11 |
| Novartis | 103 | 1 |
| Overview | 103 | 1 |
| Major Orphan Diseases Molecules in Pipeline | 103 | 2 |
| Johnson &Johnson | 105 | 1 |
| Overview | 105 | 1 |
| Major Orphan Diseases Molecules in Pipeline | 105 | 3 |
| Sanofi | 108 | 1 |
| Overview | 108 | 1 |
| Major Orphan Diseases Molecules in Pipeline | 108 | 2 |
| Amgen | 110 | 1 |
| Overview | 110 | 1 |
| Major Orphan Diseases Products in Pipeline | 110 | 2 |
| Celgene Corporation | 112 | 1 |
| Overview | 112 | 1 |
| Major Orphan Diseases Molecules in Pipeline | 112 | 2 |
| Endpoints-Clinical Trials in Orphan Diseases - Appendix | 114 | 5 |
| Market Definitions | 114 | 1 |
| Abbreviations | 114 | 1 |
| Research Methodology | 115 | 3 |
| Coverage | 116 | 1 |
| Secondary Research | 116 | 1 |
| Primary Research | 116 | 1 |
| Expert Panel Validation | 117 | 1 |
| Contact Us | 117 | 1 |
| Disclaimer | 117 | 1 |
| Sources | 118 | 1 |
