First, I was a political appointee and know the Congressman/Congresswoman is not reading this letter. As an important member of his/her staff, I ask you to please take a moment to seriously listen to my message. You are more than aware that he/she is ultimately responsible for your response.
The National Institute of Health defines a “rare disease” as one that affects fewer than 200,000 people in the United States, which was included by Congress in the Orphan Drug Act of 1983. There are approximately 7,000 diseases or disorders that qualify for this designation. Alone, each disease is rare. It is only when counted together that they are not. Because of the Congressional Orphan Drug Act of 1983, the term “orphan disease” is often used interchangeably with “rare disease.” Anyone who suffers from a rare disease or, in our case, our three-year-old daughter, Katherine Belle, who suffers from one, can understand the harsh poetry of that term. All too often, the sufferers are children. Statistically, 30% of children with rare and genetic diseases will not live to see their fifth birthday.
In many ways, individuals with rare disorders are orphans of the scientific, medical, and political communities. It is understandable that we donate our charitable dollars to studying conditions we know all too well. There is not much money to be made from a few children compared to the billions to be made off of each new cancer therapy or even a minor improvement in a heart stint.
So, yes, in this sense, we are orphans. In most cases, genetic testing is not covered by insurance companies because they are in the business of collecting premiums, not paying claims. Simply put: rare disease patients are easy to ignore and expensive to hear. It costs nothing for the insurance company to let our daughter die. More often than not, rare disease patients are figurative orphans left to die by harsh economic realities. However, this is not just an insurance issue. There are many layers and barriers at work in this failing system. Our children deserve better.
My request to you is simple: Please take a moment to read our story at www.hopeforkatherinebelle.com, where you will see the face of rare disease and learn about more barriers patients face. After you do so, ask yourself how you can help change things for the better? Right off hand, I know there is an opportunity to co-sponsor H.R. 1591, the Charles August Long UNDIAGNOSED DISEASES RESEARCH AND COLLABORATION ACT OF 2013. Likewise, I strongly encourage you to join the Rare Disease Caucus to brainstorm ways Congress can help the rare disease community. If nothing else, this is actually for your benefit. At a 10% overall rare disease rate in the United States, it will affect your family. It is a matter of when, not if. You are in a position to help us change things for the better before you discover you or your own “Katherine Belle” is among that 10%.
*I will send a hard copy of this letter to all members of Congress with their names added.