'Rare' disease awareness is important

As co-chair of the Assembly Rare Disease caucus, I 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% 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. Because these diseases affect relatively few people, the usual economic incentives for drug development don’t apply. 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 Food and Drug Administration said 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 and a lack of access to ancillary services, with many going untreated simply because treatments aren’t available.

An estimated 30 million people in the United States suffer from rare diseases/conditions. That’s why raising public awareness about this problem is so important. The National Organization for Rare Diseases has organized a national Rare Disease Day for many years, and I was happy to join Assemblymembers Rob Bonta and Brian Maienschein to jointly write House Resolution 25, proclaiming Feb. 28 as Rare Disease Day in California.