News Article
February 29, 2016

Rare Disease Day: A Giant Leap in the Push for #CuresNow

Rare Disease Week Launches on Capitol Hil


February 29, 2016

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Rare Disease Day: A Giant Leap in the Push for #CuresNow

Rare Disease Week Launches on Capitol Hill


No leaping over today – it’s Rare Disease Day, which kicks off Rare Disease Week. Meet Candace Lerman, an advocate from Florida who will be blitzing Capitol Hill this week. Candace, who has Immune Thrombocytopenia (ITP), recently explained why it’s so important we repurpose drugs and treatments. Why does this sound familiar? Because it’s a critical component of H.R. 6, the 21st Century Cures Act. She also highlights her visit with Rep. Gus Bilirakis (R-FL) last year in Washington, D.C., during Rare Disease Week.


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Follow the advocates and our Members during Rare Disease Week by monitoring #CuresNow,#Path2Cures, and #CuresIn4Words on your digital platforms.


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Repurposing Drugs Matters with Rare Disease

By Candace Lerman


In November 2014, I used the chemotherapy drug Rituxan off-label for my rare disease, Immune Thrombocytopenia (ITP). The decision was made after careful consideration of all the possible outcomes. I was desperate for relief since ITP causes internal bleeding that can be fatal. This thought stayed in the back of my mind as I watched my platelets drop and my bleeding episodes increase in severity.


Ultimately, I decided it was worth a shot to see if Rituxan could put my rare disease in remission. When it was successful, I was thrilled and shared my success with the rest of the ITP community. To my surprise, many ITP patients told me they did not have access to Rituxan because of the off-label status. When I first heard about the Orphan Product Extensions Now, Accelerating Cures and Treatments Act (OPEN ACT), I was thrilled! Finally, legislation was presented that would allow rare disease patients to have access to FDA approved drugs that are deemed safe and effective for other conditions.


During Rare Disease Week last year, I discussed my story with Congressman Gus Bilirakis of Florida, who introduced the OPEN ACT to the Energy and Commerce Committee. Congressman Bilirakis — a huge champion for patients — and his staff worked hard to have the OPEN ACT included in the 21st Century Cures Act. The bill passed the House in July 2015 by an overwhelming vote of 344-77. The momentum has stalled in the Senate though, so now patients must act. Recently, the Senate Health, Education, Labor and Pensions Committee (HELP), listed bills that were a priority for their version of 21st Century Cures, and for some reason omitted the OPEN ACT. I will be on the Hill for Rare Disease Week this year to push for the inclusion of the OPEN ACT on the HELP Committee’s priority agenda.


For the rare disease community and patients as a whole, the OPEN ACT will provide critically needed treatment options for underserved populations. It will help fill in the gaps between diseases with limited or no treatment options and new drugs under development. I encourage all patients, regardless of their illness to reach out to their Senators and express support for the bill. Repurposing FDA approved drugs will create new data that provides the foundation for further research. Ultimately, the OPEN ACT can unlock new treatments and potential cures for rare diseases. I am living proof that repurposing drugs can save lives. Looking back, the decision to use Rituxan paid off more than I could ever imagine.


I encourage everyone to join the conversation on social media using the hashtag #CuresNow on Twitter, Facebook and Instagram. Many of our representatives are participating with us. This is our time to show lawmakers that we may be rare when identified by specific disease or diagnosis, but together we are a movement!


Read the column online here.


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