Malignant mesothelioma is a rare disease. It is one of approximately 6,000 rare diseases that have been recognized. These diseases are often referred to as “orphan diseases” Rare diseases are those that affect fewer than 200,000 people in the United States, with a population of 330 million people. In other countries, the numbers are different –with numbers varying from 1 to 8 per 10,000 people. Having a rare disease, it follows that few medical professionals are experts as they see so few of these diseases in their practices. Some of the web sites that you can find information on are the National Organization of Rare Disorders (NORD) www.rarediseases.org. The United States government offers on its National Institute of Health web site, information on Genetics and Rare Disease Information Center www.rarediseases.info.nih.gov. GARD, created in 2002 by the National Human Genome Research Institute (NHGRI) and the Office of Rare Diseases Research (ORDR) to help people find information about rare diseases. Orphanet is another option for rare diseases and orphan drugs, the site is Europe focused and based.
It has been recognized that it is vital for patients and families to obtain, reliable up to date medical information for rare diseases. It is also important that they have the option of a supporting community, either on line or in person at conferences.
Some of the rare diseases we have all heard about such as mesothelioma, cystic fibrosis, Tourette’s Syndrome and Amyotrophic Lateral Sclerosis-“Lou Gehrig’s ” disease.
Rare diseases also have a day every year to increase awareness. It is held internationally on the last day of February. The theme for 2018 is research. February 28, 2018 is the next one planned.
Another issue when dealing with rare diseases is to get the pharmaceutical companies interested in research and development of drugs that will have a limited market. Research and development of drugs cost millions of dollars and many years to develop. According to the web site www.fda.gov/Drugs/ResourcesForYou/Consumers ; In 1983 Congress passed the Orphan Drug Act (ODA). The ODA created financial incentives for drug and biologics manufactures, including, tax credits for costs of clinical research, government grant funding, assistance for clinical research, and a seven-year period of exclusive marketing given the first sponsor of an orphan-designated product who obtains market approval from the Food and Drug Administration for the same indication. At the same time, federal programs at the FDA and the NIH began encouraging product development, as well as clinical research for products targeting rare diseases.
In 1983, the United States Food and Drug Administration established the Office of Orphan Product Development. Since this law was signed by President Reagan in 1983, more than 200 companies have brought almost 450 orphan drugs to market. Some of the drugs have been approved to treat other more common diseases and then are “repurposed” to treat rare diseases. For some rare diseases, the drug approval process is based on one well run clinical trial.
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