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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