The government is getting involved in the push to find cures for rare diseases. The Orphan Drug Act of 1983 was a key initiative, providing tax incentives and other benefits for drug companies willing to explore treatments for rare diseases. Since its enactment, more than 340 treatments for rare diseases have been approved.
The National Institutes of Health (NIH) have also gotten involved in promoting new drug research for rare diseases. Their effort, the Therapeutics for Rare and Neglected Diseases program (TRND), aims to create a research pipeline that will integrate smaller and larger company efforts to develop treatments for orphan drugs. The National Institutes of Health Office of Rare Diseases Research will handle the oversight and governing of the new initiative, which will provide a facility administered by the National Human Genome Research Institute.
Patient Information
If you suffer from a rare disease or have a family member who is affected, you can get information from the Office of Rare Diseases Research online Genetic and Rare Diseases Information Center, which offers a searchable website. There are also information specialists available by phone, email, fax, TTY or postal mail.
Rare diseases often have support groups online. These non-profit organizations serve as a clearinghouse for research information, promote research and serve as political advocates for their constituents affected by particular rare diseases. Among the more prominent groups is the National Organization for Rare Diseases and the Genetic Alliance.
Unfortunately, an estimated 50 percent of rare diseases do not have such support groups.