Sacramento, CA Marie Waldron, State Assemblymember –As co-chair of the Assembly Rare Disease caucus, I am privileged to help recognize the struggle of patients and their families living with rare diseases. Among these are almost 7,000 diseases and conditions considered rare, with 80 percent of those considered ultra-rare.
By definition, a disease is considered rare when it affects fewer than 200,000 people. Since only a relatively small number of people are involved, treatments for these diseases can be limited, have sky-high costs or are completely unavailable. That’s where Orphan Drugs come in. In 1983, President Ronald Reagan signed the Orphan Drug Act to encourage development of medical treatments for rare diseases that affect small numbers of persons. These drugs are developed solely in response to public need, since the huge costs of developing a new drug are unlikely to be recovered.
The FDA reports that more than 600 drugs and treatments have been approved for treating these diseases in the United States. Even so, no treatments exist for the vast majority of rare diseases. Millions of American patients and their families bear a huge burden, facing long delays in diagnosis, in finding medical experts capable of providing treatment, a lack of access to ancillary services, with many going untreated simply because treatments aren’t available, or the costs are prohibitive. If you or a loved one suffer from a rare disease and require financial assistance, there may be resources available to you here.
An estimated 30 million people in the United States suffer from rare diseases. That’s why raising public awareness of this problem is so important. The National Organization for Rare Diseases has organized a national Rare Disease Day for many years, and I am happy to join Assemblymembers Bonta and Maienschein to jointly author House Resolution 25 proclaiming February 28 as Rare Disease Day in the State of California.
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