Williamstown, MA -- (SBWIRE) -- 01/26/2012 -- 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.
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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.
- 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.
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