Two years ago, I held a series of roundtable meetings in Lutz, Florida with physicians, advocates, patients, and families from our area to hear about the challenges of finding cures for the thousands of deadly and chronic diseases impacting millions of Americans. Diseases like cancer, ALS, Alzheimer’s and the 7,000 rare conditions. These discussions were eye-opening to me. I got a true sense of how difficult it is to get new medicines to market, and how painful it can be for families desperate for treatment.

Out of these conversations with folks like Ashleigh Pike from Palm Harbor, who lives with Dysautonomia, Dr. Thomas Sellers, a top researcher with Moffitt Cancer Center, and Patricia Stanco, an advocate with the ALS Association Florida Chapter, along with many others, came the 21st Century Cures Act.

The 21st Century Cures Act is a comprehensive legislative package that will help speed up the development, discovery, and delivery of lifesaving cures and treatments for patients in need. During the early stages of working on 21st Century Cures, I became especially inspired by those fighting rare diseases.

Rare diseases affect 30 million Americans, and yet 95 percent of rare diseases have no approved treatment. Right now, drug makers do not have a major incentive to repurpose mainstream treatments for rare disease patients because each disease impacts a small number of patients—even though many of these conditions are life-threatening.

We need to give people fighting rare diseases a voice. That is why I introduced a provision to 21st Century Cures called the OPEN Act, an initiative to help leverage the free market and promote repurposing major market drugs for rare diseases. The OPEN Act has the potential to result in hundreds of new drugs and treatments for individuals with rare diseases, as well as a new surge in

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