Endpoints-Clinical Trials in Orphan Diseases - Highest Number of Terminated Trials Focused on Mulitiple Myeloma

Summary

Company, the leading business intelligence provider, has released its latest research report, “Endpoints-Clinical Trials in Orphan Diseases - Highest Number of Terminated Trials Focused on Mulitiple Myeloma” providing an insight into different endpoints that are used in orphan disease clinical trials. The report examines different aspects of clinical trial endpoints in orphan diseases, such as analysis of major marketed orphan drugs with an emphasis on safety and efficacy details, Phase II and Phase III clinical trial analyses for both completed and ongoing clinical trials, most promising orphan drugs with more emphasis on safety, efficacy and clinical trial details, and terminated trial analysis. The company profiling highlights the orphan drugs of different companies.

These rare diseases have a low rate of prevalence in the existing population and a physician rarely gets to see patients with these conditions. Most of the orphan diseases are often genetic and hence they persist throughout a person’s life. It is estimated that 80% of orphan diseases have genetic origins. The remaining diseases occur due to allergies, degenerative and proliferative causes and as a result of infection. The symptoms for these diseases are not immediate and it appear later for most of the conditions. The definition of orphan diseases varies with geography and is primarily dependent upon the prevalence of a disease.

This report “Endpoints- Clinical Trials in Orphan Diseases” highlights the seven major orphan diseases: Huntington’s disease, acute myeloid leukemia, amyotrophic lateral sclerosis, Hodgkin’s lymphoma, multiple myeloma, ovarian cancer and pancreatic cancer.

The term endpoint refers to an outcome or measure of a clinical trial. Endpoints can include all kinds of aspects, those related to the effectiveness of treatment and others. However, endpoint selection must take into account the need to obtain the most information of therapeutic interest with the least risk and discomfort for the individual. Also, the endpoints must be in line with the objective of the study and represent the most effective way of assessing a pharmacological response.

Scope

- Data and analysis on the marketed products and analysis of their efficacy and safety details
- Analysis of the seven major orphan diseases which are Huntington’s disease, acute myeloid leukemia, amyotrophic lateral sclerosis, Hodgkin’s lymphoma, multiple myeloma, ovarian cancer and pancreatic cancer.
- Analysis of the Phase III and Phase II clinical trials in terms of percentage of cases. An analysis of terminated trials is also included in this chapter. Only industry-sponsored studies are included in the report.
- Analysis on most promising molecules of the seven major orphan diseases with emphasis on their efficacy and safety details.
- Company Profiling details the companies with a strong market presence.

Reasons to buy

- Understand the trends in clinical trial endpoints used for orphan diseases
- Build effective strategies to launch pipeline products by identifying potential geographies
- Exploit in-licensing and out-licensing opportunities by identifying products that might probably fill their portfolio gaps
- Align your product portfolio to the markets with high growth potential
- Develop market-entry and market expansion strategies by identifying the leading therapeutic segments and geographic markets poised for strong growth
- Reinforce R&D pipelines by identifying new target mechanisms which can produce first-in-class molecules with improved efficiency and safety profiles
- Develop key strategic initiatives by understanding the key focus areas of leading companies

1.1 List of Tables 6
1.2 List of Figures 7
2 Endpoints-Clinical Trials in Orphan Diseases - Introduction 8
2.1 Disease Overview 8
2.1.1 The US 8
2.1.2 Europe 8
2.1.3 Japan 8
2.2 Company Report Guidance 8
3 Endpoints-Clinical Trials in Orphan Diseases - Design of Orphan Diseases Clinical Trials and its Outcomes 9
3.1 Clinical Trial Design - Need For Adaptive Trial Designs In Orphan Diseases 9
4 Endpoints-Clinical Trials in Orphan Diseases: An Overview on Endpoints 10
4.1 Types of Clinical Trial Outcomes 10
4.1.1 Oncology Endpoints 10
4.1.2 Other Endpoints 10
5 Endpoints-Clinical Trials in Orphan Diseases - Marketed and Pipeline Products Assessment 12
5.1 Huntington’s disease 12
5.1.1 Primary Endpoints used in Huntington’s disease Clinical Trials 12
5.1.2 Secondary Endpoints used in Huntington’s disease Clinical Trials 12
5.1.3 Major Marketed Drugs-Safety and Efficacy Analysis 12
5.1.4 Phase III Clinical Trial Analysis 13
5.1.5 Phase III Primary Endpoint analysis 14
5.1.6 Phase III Secondary Endpoint analysis 15
5.1.7 Most Promising Drugs’ Profiles 16
5.1.8 Phase II Clinical Trial Analysis 16
5.1.9 Phase II Primary Endpoint analysis 16
5.1.10 Phase II Secondary Endpoint analysis 17
5.1.11 Terminated Trials Analysis 17
5.2 Acute Myelocytic Leukemia 18
5.2.1 Overview 18
5.2.2 Primary Endpoints used in Acute Myelocytic Leukemia Clinical Trials 18
5.2.3 Secondary Endpoints used in Acute Myelocytic Leukemia Clinical Trials 19
5.2.4 Major Marketed Drugs-Safety and Efficacy Analysis 20
5.2.5 Phase III Clinical Trial Analysis 21
5.2.6 Phase III Primary Endpoint analysis 22
5.2.7 Phase III Secondary Endpoint analysis 23
5.2.8 Most Promising Drugs’ Profiles 24
5.2.9 Phase II Clinical Trial Analysis 25
5.2.10 Phase II Primary Endpoint analysis 29
5.2.11 Phase II Secondary Endpoint analysis 30
5.2.12 Terminated Trial Analysis 31
5.3 Amyotrophic Lateral Sclerosis 32
5.3.1 Overview 32
5.3.2 Primary Endpoints used in Amyotrophic Lateral Sclerosis Clinical Trials 32
5.3.3 Secondary Endpoints used in Amyotrophic Lateral Sclerosis Clinical Trials 33
5.3.4 Major Marketed Drugs-Safety and Efficacy Analysis 33
5.3.5 Phase III Clinical Trial Analysis 33
5.3.6 Phase III Primary Endpoint analysis 34
5.3.7 Phase III Secondary Endpoint analysis 35
5.3.8 Most Promising Drugs’ Profiles 35
5.3.9 Phase II Clinical Trial Analysis 36
5.3.10 Phase II Primary Endpoint analysis 37
5.3.11 Phase II Secondary Endpoint analysis 38
5.3.12 Terminated Trials Analysis 38
5.4 Hodgkin Lymphoma 39
5.4.1 Primary Endpoints used in Hodgkin Lymphoma Clinical Trials 39
5.4.2 Secondary Endpoints used in Hodgkin Lymphoma Clinical Trials 39
5.4.3 Major Marketed Drugs-Safety and Efficacy Analysis 40
5.4.4 Phase III Clinical Trial Analysis 40
5.4.5 Phase III Primary Endpoint analysis 41
5.4.6 Phase III Secondary Endpoint analysis 42
5.4.7 Most Promising Drugs’ Profiles 43
5.4.8 Phase II Clinical Trial Analysis 44
5.4.9 Phase II Primary Endpoint analysis 45
5.4.10 Phase II Secondary Endpoint analysis 46
5.4.11 Terminated Trials Analysis 47
5.5 Multiple Myeloma 47
5.5.1 Overview 47
5.5.2 Primary Endpoints used in Multiple Myeloma Clinical Trials 47
5.5.3 Secondary Endpoints used in Multiple Myeloma Clinical Trials 48
5.5.4 Major Marketed Drugs-Safety and Efficacy Analysis 48
5.5.5 Phase III Clinical Trial Analysis 52
5.5.6 Phase III Primary Endpoint analysis 56
5.5.7 Phase III Secondary Endpoint analysis 57
5.5.8 Most Promising Drugs’ Profiles 58
5.5.9 Phase II Clinical Trial Analysis 59
5.5.10 Phase II Primary Endpoint Analysis 64
5.5.11 Phase II Secondary Endpoint analysis 65
5.5.12 Terminated Trials Analysis 66
5.6 Ovarian Cancer 68
5.6.1 Overview 68
5.6.2 Primary Endpoints used in Ovarian Cancer Clinical Trials 68
5.6.3 Secondary Endpoints used in Ovarian Cancer Clinical Trials 68
5.6.4 Major Marketed Drugs-Safety and Efficacy Analysis 69
5.6.5 Phase III Clinical Trial Analysis 75
5.6.6 Phase III Primary Endpoint analysis 77
5.6.7 Phase III Secondary Endpoint analysis 77
5.6.8 Most Promising Drugs’ Profiles 78
5.6.9 Phase II Clinical Trial Analysis 79
5.6.10 Phase II Primary Endpoint analysis 85
5.6.11 Phase II Secondary Endpoint analysis 86
5.6.12 Terminated Trials Analysis 87
5.7 Pancreatic Cancer 88
5.7.1 Overview 88
5.7.2 Primary Endpoints used in Pancreatic Cancer Clinical Trials 88
5.7.3 Secondary Endpoints used in Pancreatic Cancer Clinical Trials 88
5.7.4 Major Marketed Drugs-Safety and Efficacy Analysis 89
5.7.5 Phase III Clinical Trial Analysis 92
5.7.6 Phase III Primary Endpoint analysis 94
5.7.7 Phase III Secondary Endpoint analysis 95
5.7.8 Most Promising Drugs’ Profiles 96
5.7.9 Phase II Clinical Trial Analysis 97
5.7.10 Phase II Primary Endpoint Analysis 100
5.7.11 Phase II Secondary Endpoint analysis 101
5.7.12 Terminated Trials Analysis 102
6 Endpoints-Clinical Trials in Orphan Diseases - Company Profiling 103
6.1 Novartis 103
6.1.1 Overview 103
6.1.2 Major Orphan Diseases Molecules in Pipeline 103
6.2 Johnson & Johnson 105
6.2.1 Overview 105
6.2.2 Major Orphan Diseases Molecules in Pipeline 105
6.3 Sanofi 108
6.3.1 Overview 108
6.3.2 Major Orphan Diseases Molecules in Pipeline 108
6.4 Amgen 110
6.4.1 Overview 110
6.4.2 Major Orphan Diseases Products in Pipeline 110
6.5 Celgene Corporation 112
6.5.1 Overview 112
6.5.2 Major Orphan Diseases Molecules in Pipeline 112
7 Endpoints-Clinical Trials in Orphan Diseases - Appendix 114
7.1 Market Definitions 114
7.2 Abbreviations 114
7.3 Research Methodology 115
7.3.1 Coverage 116
7.3.2 Secondary Research 116
7.3.3 Primary Research 116
7.3.4 Expert Panel Validation 117
7.4 Contact Us 117
7.5 Disclaimer 117
7.6 Sources 118

1.1 List of Tables

Table 1: Huntington’s disease, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010 13
Table 2: Huntington’s disease, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010 16
Table 3: Acute Myelocytic Leukemia, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010 21
Table 4: Acute Myelocytic Leukemia, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010 25
Table 5: Acute Myelocytic Leukemia, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010 31
Table 6: Amyotrophic Lateral Sclerosis, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010 36
Table 7: Amyotrophic Lateral Sclerosis, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010 38
Table 8: Hodgkin’s lymphoma, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010 40
Table 9: Hodgkin’s lymphoma, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010 44
Table 10: Hodgkin’s Lymphoma, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010 47
Table 11: Multiple Myeloma, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010 52
Table 12: Multiple Myeloma, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010 59
Table 13: Multiple Myeloma, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010 66
Table 14: Ovarian Cancer, Global, Approved Products, 1990-2010 69
Table 15: Ovarian Cancer, Global, Marketed Drugs Efficacy, 2010 69
Table 16: Ovarian Cancer, Global, Gemzar - Adverse Reactions, 2010 71
Table 17: Ovarian Cancer, Global, Hycamtin - Adverse Reactions, 2010 72
Table 18: Ovarian Cancer, Global, Major Marketed Product Comparison in Market, 2010 73
Table 19: Ovarian Cancer, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010 75
Table 20: Ovarian Cancer, Global, Primary and Secondary Endpoints of Phase II Molecules, 2010 79
Table 21: Ovarian Cancer, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010 87
Table 22: Pancreatic Cancer, Global, Gemcitabine Based Combinations, 2010 89
Table 23: Pancreatic Cancer, Global, Efficacy Results for Tarceva and Gemcitabine Versus Placebo, 2010 91
Table 24: Pancreatic Cancer, Global, Major Marketed Products , 2010 91
Table 25: Pancreatic Cancer, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010 92
Table 26: Pancreatic Cancer, Global, Primary and Secondary Endpoints of Phase II Molecules, 2010 97
Table 27: Pancreatic Cancer, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010 102
Table 28: Novartis AG, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010 103
Table 29: Johnson & Johnson, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010 105
Table 30: Sanofi, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010 108
Table 31: Amgen, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010 110
Table 32: Celgene Corporation, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010 112

1.2 List of Figures

Figure 1: Huntington’s disease, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010 14
Figure 2: Huntington’s disease, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010 15
Figure 3: Huntington’s disease, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010 17
Figure 4: Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010 22
Figure 5: Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010 23
Figure 6: Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010 29
Figure 7: Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010 30
Figure 8: Amyotrophic Lateral Sclerosis, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010 34
Figure 9: Amyotrophic Lateral Sclerosis, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010 35
Figure 10: Amyotrophic Lateral Sclerosis, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010 37
Figure 11: Amyotrophic Lateral Sclerosis, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010 38
Figure 12: Hodgkin’s lymphoma, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010 41
Figure 13: Hodgkin’s lymphoma, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010 42
Figure 14: Hodgkin’s lymphoma, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010 45
Figure 15: Hodgkin’s Lymphoma, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010 46
Figure 16: Multiple Myeloma, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010 56
Figure 17: Multiple Myeloma, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010 57
Figure 18: Multiple Myeloma, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010 64
Figure 19: Multiple Myeloma, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010 65
Figure 20: Ovarian Cancer, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010 77
Figure 21: Ovarian Cancer, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010 77
Figure 22: Ovarian Cancer, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010 85
Figure 23: Ovarian Cancer, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010 86
Figure 24: Pancreatic Cancer, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010 94
Figure 25: Pancreatic Cancer, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010 95
Figure 26: Pancreatic Cancer, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010 100
Figure 27: Pancreatic Cancer, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010 101

Companies Mentioned

Novartis
Johnson & Johnson
Sanofi
Amgen
Celgene Corporation