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

GBI Research, January 2012, Pages: 118

GBI Research, 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 Table of Contents
1.1 List of Tables
1.2 List of Figures

2 Endpoints-Clinical Trials in Orphan Diseases - Introduction
2.1 Disease Overview
2.1.1 The US
2.1.2 Europe
2.1.3 Japan
2.2 GBI Research Report Guidance

3 Endpoints-Clinical Trials in Orphan Diseases - Design of Orphan Diseases Clinical Trials and its Outcomes
3.1 Clinical Trial Design - Need For Adaptive Trial Designs In Orphan Diseases

4 Endpoints-Clinical Trials in Orphan Diseases: An Overview on Endpoints
4.1 Types of Clinical Trial Outcomes
4.1.1 Oncology Endpoints
4.1.2 Other Endpoints

5 Endpoints-Clinical Trials in Orphan Diseases - Marketed and Pipeline Products Assessment
5.1 Huntington’s disease
5.1.1 Primary Endpoints used in Huntington’s disease Clinical Trials
5.1.2 Secondary Endpoints used in Huntington’s disease Clinical Trials
5.1.3 Major Marketed Drugs-Safety and Efficacy Analysis
5.1.4 Phase III Clinical Trial Analysis
5.1.5 Phase III Primary Endpoint analysis
5.1.6 Phase III Secondary Endpoint analysis
5.1.7 Most Promising Drugs’ Profiles
5.1.8 Phase II Clinical Trial Analysis
5.1.9 Phase II Primary Endpoint analysis
5.1.10 Phase II Secondary Endpoint analysis
5.1.11 Terminated Trials Analysis
5.2 Acute Myelocytic Leukemia
5.2.1 Overview
5.2.2 Primary Endpoints used in Acute Myelocytic Leukemia Clinical Trials
5.2.3 Secondary Endpoints used in Acute Myelocytic Leukemia Clinical Trials
5.2.4 Major Marketed Drugs-Safety and Efficacy Analysis
5.2.5 Phase III Clinical Trial Analysis
5.2.6 Phase III Primary Endpoint analysis
5.2.7 Phase III Secondary Endpoint analysis
5.2.8 Most Promising Drugs’ Profiles
5.2.9 Phase II Clinical Trial Analysis
5.2.10 Phase II Primary Endpoint analysis
5.2.11 Phase II Secondary Endpoint analysis
5.2.12 Terminated Trial Analysis
5.3 Amyotrophic Lateral Sclerosis
5.3.1 Overview
5.3.2 Primary Endpoints used in Amyotrophic Lateral Sclerosis Clinical Trials
5.3.3 Secondary Endpoints used in Amyotrophic Lateral Sclerosis Clinical Trials
5.3.4 Major Marketed Drugs-Safety and Efficacy Analysis
5.3.5 Phase III Clinical Trial Analysis
5.3.6 Phase III Primary Endpoint analysis
5.3.7 Phase III Secondary Endpoint analysis
5.3.8 Most Promising Drugs’ Profiles
5.3.9 Phase II Clinical Trial Analysis
5.3.10 Phase II Primary Endpoint analysis
5.3.11 Phase II Secondary Endpoint analysis
5.3.12 Terminated Trials Analysis
5.4 Hodgkin Lymphoma
5.4.1 Primary Endpoints used in Hodgkin Lymphoma Clinical Trials
5.4.2 Secondary Endpoints used in Hodgkin Lymphoma Clinical Trials
5.4.3 Major Marketed Drugs-Safety and Efficacy Analysis
5.4.4 Phase III Clinical Trial Analysis
5.4.5 Phase III Primary Endpoint analysis
5.4.6 Phase III Secondary Endpoint analysis
5.4.7 Most Promising Drugs’ Profiles
5.4.8 Phase II Clinical Trial Analysis
5.4.9 Phase II Primary Endpoint analysis
5.4.10 Phase II Secondary Endpoint analysis
5.4.11 Terminated Trials Analysis
5.5 Multiple Myeloma
5.5.1 Overview
5.5.2 Primary Endpoints used in Multiple Myeloma Clinical Trials
5.5.3 Secondary Endpoints used in Multiple Myeloma Clinical Trials
5.5.4 Major Marketed Drugs-Safety and Efficacy Analysis
5.5.5 Phase III Clinical Trial Analysis
5.5.6 Phase III Primary Endpoint analysis
5.5.7 Phase III Secondary Endpoint analysis
5.5.8 Most Promising Drugs’ Profiles
5.5.9 Phase II Clinical Trial Analysis
5.5.10 Phase II Primary Endpoint Analysis
5.5.11 Phase II Secondary Endpoint analysis
5.5.12 Terminated Trials Analysis
5.6 Ovarian Cancer
5.6.1 Overview
5.6.2 Primary Endpoints used in Ovarian Cancer Clinical Trials
5.6.3 Secondary Endpoints used in Ovarian Cancer Clinical Trials
5.6.4 Major Marketed Drugs-Safety and Efficacy Analysis
5.6.5 Phase III Clinical Trial Analysis
5.6.6 Phase III Primary Endpoint analysis
5.6.7 Phase III Secondary Endpoint analysis
5.6.8 Most Promising Drugs’ Profiles
5.6.9 Phase II Clinical Trial Analysis
5.6.10 Phase II Primary Endpoint analysis
5.6.11 Phase II Secondary Endpoint analysis
5.6.12 Terminated Trials Analysis
5.7 Pancreatic Cancer
5.7.1 Overview
5.7.2 Primary Endpoints used in Pancreatic Cancer Clinical Trials
5.7.3 Secondary Endpoints used in Pancreatic Cancer Clinical Trials
5.7.4 Major Marketed Drugs-Safety and Efficacy Analysis
5.7.5 Phase III Clinical Trial Analysis
5.7.6 Phase III Primary Endpoint analysis
5.7.7 Phase III Secondary Endpoint analysis
5.7.8 Most Promising Drugs’ Profiles
5.7.9 Phase II Clinical Trial Analysis
5.7.10 Phase II Primary Endpoint Analysis
5.7.11 Phase II Secondary Endpoint analysis
5.7.12 Terminated Trials Analysis

6 Endpoints-Clinical Trials in Orphan Diseases - Company Profiling
6.1 Novartis
6.1.1 Overview
6.1.2 Major Orphan Diseases Molecules in Pipeline
6.2 Johnson & Johnson
6.2.1 Overview
6.2.2 Major Orphan Diseases Molecules in Pipeline
6.3 Sanofi
6.3.1 Overview
6.3.2 Major Orphan Diseases Molecules in Pipeline
6.4 Amgen
6.4.1 Overview
6.4.2 Major Orphan Diseases Products in Pipeline
6.5 Celgene Corporation
6.5.1 Overview
6.5.2 Major Orphan Diseases Molecules in Pipeline

7 Endpoints-Clinical Trials in Orphan Diseases - Appendix
7.1 Market Definitions
7.2 Abbreviations
7.3 Research Methodology
7.3.1 Coverage
7.3.2 Secondary Research
7.3.3 Primary Research
7.3.4 Expert Panel Validation
7.4 Contact Us
7.5 Disclaimer
7.6 Sources

1.1 List of Tables
Table 1: Huntington’s disease, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010
Table 2: Huntington’s disease, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010
Table 3: Acute Myelocytic Leukemia, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010
Table 4: Acute Myelocytic Leukemia, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010
Table 5: Acute Myelocytic Leukemia, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010
Table 6: Amyotrophic Lateral Sclerosis, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010
Table 7: Amyotrophic Lateral Sclerosis, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010
Table 8: Hodgkin’s lymphoma, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010
Table 9: Hodgkin’s lymphoma, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010
Table 10: Hodgkin’s Lymphoma, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010
Table 11: Multiple Myeloma, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010
Table 12: Multiple Myeloma, Global, Primary and Secondary Endpoints of Phase II Molecules , 2010
Table 13: Multiple Myeloma, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010
Table 14: Ovarian Cancer, Global, Approved Products, 1990-2010
Table 15: Ovarian Cancer, Global, Marketed Drugs Efficacy, 2010
Table 16: Ovarian Cancer, Global, Gemzar - Adverse Reactions, 2010
Table 17: Ovarian Cancer, Global, Hycamtin - Adverse Reactions, 2010
Table 18: Ovarian Cancer, Global, Major Marketed Product Comparison in Market, 2010
Table 19: Ovarian Cancer, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010
Table 20: Ovarian Cancer, Global, Primary and Secondary Endpoints of Phase II Molecules, 2010
Table 21: Ovarian Cancer, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010
Table 22: Pancreatic Cancer, Global, Gemcitabine Based Combinations, 2010
Table 23: Pancreatic Cancer, Global, Efficacy Results for Tarceva and Gemcitabine Versus Placebo, 2010
Table 24: Pancreatic Cancer, Global, Major Marketed Products , 2010
Table 25: Pancreatic Cancer, Global, Primary and Secondary Endpoints of Phase III Molecules , 2010
Table 26: Pancreatic Cancer, Global, Primary and Secondary Endpoints of Phase II Molecules, 2010
Table 27: Pancreatic Cancer, Global, Discontinued Drugs’ Primary and Secondary Endpoints, 2010
Table 28: Novartis AG, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010
Table 29: Johnson & Johnson, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010
Table 30: Sanofi, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010
Table 31: Amgen, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010
Table 32: Celgene Corporation, Global, Endpoints of Major Orphan Disease Pipeline Molecules, 2010

1.2 List of Figures
Figure 1: Huntington’s disease, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010
Figure 2: Huntington’s disease, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010
Figure 3: Huntington’s disease, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010
Figure 4: Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010
Figure 5: Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010
Figure 6: Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010
Figure 7: Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010
Figure 8: Amyotrophic Lateral Sclerosis, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010
Figure 9: Amyotrophic Lateral Sclerosis, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010
Figure 10: Amyotrophic Lateral Sclerosis, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010
Figure 11: Amyotrophic Lateral Sclerosis, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010
Figure 12: Hodgkin’s lymphoma, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010
Figure 13: Hodgkin’s lymphoma, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010
Figure 14: Hodgkin’s lymphoma, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010
Figure 15: Hodgkin’s Lymphoma, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010
Figure 16: Multiple Myeloma, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010
Figure 17: Multiple Myeloma, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010
Figure 18: Multiple Myeloma, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010
Figure 19: Multiple Myeloma, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010
Figure 20: Ovarian Cancer, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010
Figure 21: Ovarian Cancer, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010
Figure 22: Ovarian Cancer, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010
Figure 23: Ovarian Cancer, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010
Figure 24: Pancreatic Cancer, Global, Clinical Pipeline Phase III by Primary Endpoints, 2010
Figure 25: Pancreatic Cancer, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010
Figure 26: Pancreatic Cancer, Global, Clinical Pipeline Phase II by Primary Endpoints, 2010
Figure 27: Pancreatic Cancer, Global, Clinical Pipeline Phase II by Secondary Endpoints, 2010

GBI Research, the leading business intelligence provider, has released its latest research report entitled: “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 analysis for both completed and ongoing clinical trials, the most promising orphan drugs and their safety, efficacy and clinical trial details, and terminated trial analysis. The company profiles details 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.

Safety and Tolerability and Response Duration are Most Common Secondary Endpoints for Phase III Pipeline Molecules of Acute Myelocytic Leukemia (AML)

In general, multiple factors are considered for secondary endpoints. Most commonly seen secondary endpoints in Phase III molecules of Acute Myelocytic Leukemia include Safety and tolerability, Duration of Response and Complete remission rate, each of which account for 13% of secondary endpoints. Overall survival, used as a secondary endpoint in 12% of AML trials, is the next most common endpoint. Efficacy, EFS (Event-free survival,), DFS (Disease free survival), PFS (Progression free survival), QOL (Quality of life), Remission duration, and Toxicity each contribute 7% of secondary endpoints.

Acute Myelocytic Leukemia, Global, Clinical Pipeline Phase III by Secondary Endpoints, 2010

Note: QOL-Quality of life, OS-Overall Survival, DFS- Disease free survival, EFS-Event-free survival, PFS-Progression free survival, Efficacy and Safety endpoints refer to those trial endpoints where the specific details about the endpoints were not disclosed by the trial sponsors or in Clinicaltrials.gov website.

- Novartis
- Johnson & Johnson
- Sanofi
- Amgen
- Celgene Corporation

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