Rare Disease Day 2010
Nearly 1 in 10 Americans Has A Rare Disease
On February 28, IHRF will join the effort organized by the National Organization of Rare Disorders (NORD) to raise awareness of rare diseases, including chronic intracranial hypertension, that affect nearly 30 million Americans and countless others worldwide.
In the United States, a disease is considered rare if it is believed to affect fewer than 200,000 Americans. According to the National Institutes of Health (NIH), there are almost 7,000 rare diseases. While some have names that are familiar to the public, such as hemophilia and Lou Gehrig’s disease (ALS), have names that are familiar to the public, many others, like intracranial hypertension, do not.
Some facts:
•Nearly one in 10 Americans has a rare disease.
•Many of these diseases are genetic and present at birth (although some genetic diseases may not become apparent until much later in life).
•More than half of the people known to be affected by rare diseases at this time are children.
•One of NORD’s continuing themes is that everyone knows someone affected by a rare disease.
With so many different types of diseases, even though each one is rare, the impact on the American population as a whole is significant. For that reason, rare diseases are an important public health concern.
Many rare diseases still have no approved treatment. And many are not even being studied by medical researchers, at this time. Often, people with rare diseases are treated “off-label” with treatments that are not approved by FDA for their specific disease. This is the case with Diamox (acetazolamide)—a drug originally developed to treat heart failure—that is now used to treat IH. Increasingly, patients are experiencing reimbursement problems with insurance (including Medicare and Medicaid) for off-label treatments.
Some of the problems experienced by people who have rare diseases:
• Difficulty in obtaining an accurate diagnosis
• Limited treatment options
• Little or no research being done on the disease
• Difficulty finding physicians or treatment centers with experience in treating a particular rare disease
• Treatments that are generally more expensive than those for common diseases
• Reimbursement issues related to private insurance, Medicare, and Medicaid
• Difficulty accessing medical, social, or financial services or assistance because those making the decisions are not familiar with the disease
If you have chronic IH or know someone affected by this illness, this information is not surprising. Rare Disease Day 2010 is an opportunity to make your voice heard. IHRF is launching a grassroots media campaign to educate the public about rare diseases and chronic IH; if you are interested in participating, please .(JavaScript must be enabled to view this email address)