A Guest Post by CANDACE
This past February, I had the opportunity to advocate on behalf of ITP patients during Rare Disease Week. I traveled to Washington DC to learn about the 21st Century Cures Initiative, now House Bill 6. The legislation provides a lot of groundbreaking developments for the rare disease community, especially for those of us with Immune Thrombocytopenia. I’m a firm believer that certain things in our lives occur to inspire us to embark on great journeys. This trip was my wakeup call. I learned about a bill that would eventually be included into 21st Century Cures.
It was called the OPEN Act or Orphan Product Extensions Now Accelerating Cures and Treatments. The goal of the bill was to allow FDA-approved drugs to be repurposed for rare disease patients. This struck me, since I used Rituxan to control ITP. It worked and I have been in remission since December. Using Rituxan has saved my life and allowed me to build my “new normal” as a rare disease patient.
The OPEN Act would end “off-label” use of Rituxan for ITP patients in the US and allow more people to have access to the drug. I have met many ITP patients who were denied Rituxan by their insurance company because it was off-label and thus very, very expensive. I met with the Congressman Bilirakis who is from my home state of Florida. He introduced the OPEN Act in the Energy and Commerce Subcommittee and is a wonderful friend of the ITP community.
I told him I was living proof his bill could save and improve the lives of ITP and rare disease patients. The Congressman and his amazing staff have used my experience to push Congress to include the OPEN Act into 21st Century Cures, and were successful. None of this would have been possible without their help.
After the July 4th holiday, it is anticipated that the House of Representatives will vote on the 21st Century Cures bill. We believe it will pass with tremendous support from both parties. Our next challenge is encouraging the Senate to act with the same urgency. Time is running out and ITP patients need to have access to every treatment option available. Orrin Hatch and Amy Klobuchar furthering bi-partisan support of our efforts introduced the OPEN Act in the Senate at the end of May.
I am in contact with legislatives aides that are working diligently to get the Senate on a fast track. There is a lot of work that goes on behind the scenes and the Congressional staffers don’t receive a lot of credit. So far, I have had nothing but positive experiences working and advocating with them. They are interested in seeing that the ITP community benefits from this legislation.
Many ITP patients in other nations are probably shocked by this, and rightfully so. It is unfathomable to think that the United States would prevent rare disease patients with a life threatening blood disorder from obtaining a medication that may put their disease into a temporary remission. While Rituxan doesn’t work for all ITP patients, and doctors don’t know everything about the disease, every ITP patient has the right to try.
This is why I fight for the ITP community, I believe every ITP patient and rare disease warrior deserves the same chance I had. I also hope to have this legislation on President Obama’s desk before I need to use Rituxan again. My safe zone of 9 months is rapidly approaching, right around the time I begin law school. I am terrified to think of what will happen to me if my platelets start to fall again and I cannot have the same treatment. That’s why there is no time to waste in pushing our leaders to get this bill passed.