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Mr. WHITEHOUSE. Mr. President, I rise today to speak in support of the Food and Drug Safety and Innovation Act. This measure includes a number of important reforms to promote the development of new treatments for patients in need and to ensure that drugs and other medical products are safe and effective for American families. I commend Chairman Harkin and Ranking Member Enzi for their hard work and leadership on this bill.
As a participant in the drug supply chain integrity working group, along with the chairman and ranking member and Senators Bennet, Burr, and Grassley, I am especially proud of the strong, bipartisan measures to protect patients that have been included in this bill. The not-too-distant incidents concerning adulterated Heparin and counterfeit Avastin demonstrate the critical importance of protecting Americans from unsafe medical products manufactured overseas. The new tools and authorities in this law should help safeguard Rhode Island families from dangerous drugs, while leveling the playing field for U.S. manufacturers and providing more transparency and accountability across our drug supply chain.
I particularly want to thank the chairman and ranking member for working with me to include the Expanding and Promoting Expertise in Rare Treatments Act of 2012, or EXPERT Act, which I introduced earlier this year, in the bill on the floor.
During my time in office, I have been moved by the personal stories of dozens of Rhode Islanders with rare conditions. In the last year, I have met with Rhode Island advocates who have or whose family member has a rare disease, like Fragile X, spinal muscular atrophy, and CLOVES syndrome, among many others. Treatments for these rare conditions often do not exist or are so early in the development pipeline that it will take years for patients to benefit. Rather than simply waiting for the products to come to market, these families want to play a role in educating others about the rare disease that affects their loved one and working toward a successful treatment.
The EXPERT Act is intended to give patients and experts a role in strengthening and expediting the FDA's review of new treatments for rare diseases. The measure encourages the agency to take advantage of the wisdom and insights of rare disease experts in order to speed the development of therapies for patients suffering from rare diseases. The bill also gives rare disease patients and their advocates a role in consulting with the FDA on topics like the severity of the disease, unmet medical needs, and the benefits and risks of therapies to treat the disease.
We have seen that when the FDA gets the technical and scientific assistance it needs from rare disease experts, incredible progress can be made. The Cystic Fibrosis Foundation's recent work with Vertex Pharmaceuticals on a treatment named Kalydeco, which specifically targets the underlying causes of the disease in some patients, is a good example. As a result of close consultation with the CF Foundation and renowned experts, FDA approval for this treatment was one of the fastest in the agency's history.
Rhode Islanders are already benefiting from Kalydeco. Sheri, a former resident of Narragansett, was diagnosed with cystic fibrosis when she was 16 years old. This past year, Sheri was surprised with the news that she is one of the 4 percent of cystic fibrosis patients who can be treated by the newly approved Kalydeco. For the past months Sheri has been on Kalydeco and says that she already feels the difference in her health, and, most importantly, it has given her hope to start thinking about her future. Recently engaged in February, Sheri shared, ``I can think about having children and seeing them grow up . . . even living to see my grandchildren!''
I hope the EXPERT Act will lead to more good stories for other Rhode Island patients and families afflicted with rare diseases. I have great admiration for the determination and optimism of the Rhode Islanders with rare disease I have met over the years, and I wanted to share a few more of those stories here today.
I heard from Susan, a Providence resident and mother of 3 1/2 -year-old Phoebe. Susan describes her daughter as a ``bright, happy, and beautiful'' child. When Phoebe was 5 months old, Susan and her husband noticed that their daughter did not reach for or look at objects placed on the left side of her body. After numerous tests and doctor's visits, Phoebe was finally diagnosed with developmental dyspraxia, a motor-processing disorder. Because of the rarity of their daughter's condition, Susan and her husband found that specialists ``looked at us like we had two heads when we told them what her diagnosis was.'' Phoebe is reaching milestones in her development and is continuing to improve, but because so little is known about dyspraxia, Susan and her husband have encountered several hurdles to getting Phoebe the treatment and therapy she needs. Susan said, ``It breaks our hearts to think that Phoebe is being held back from reaching her full potential because of lack of awareness and education about her disease.''
Dorrie, from Warwick, wrote to share her family's story with me. Her youngest son was diagnosed with an extremely rare disorder called atypical non-ketotic hyperglycinemia, or NKH, when he was 4 years old. He is the only child living in Rhode Island with this disorder, which has no known cure or treatment. However, doctors have found several products can be used off label to improve their son's speech and alertness. Dorrie notes that ``he has progressed farther than we could ever have hoped possible. He is not only walking, but riding a two-wheel bicycle and playing kickball with his peers.'' Because they are using products off label, their private insurance will not cover their costs, and so they are forced to shoulder the burden of paying for their son's treatments out-of-pocket. This has caused anxiety and extreme stress on their family. As her son grows older, Dorrie is faced with more uncertainty about his future and says they are ``living on eggshells'' as he experiences increased and more severe symptoms.
For these Rhode Islanders and others like them, the challenge of having a rare disease or having a family member with a rare disease comes not just from the symptoms of the disease but the loneliness of having something that so few people understand, let alone have. The EXPERT Act is one step toward empowering patients and their families with an opportunity to participate in a process that is critically important for their future. I am pleased that the act is supported by 64 national organizations, including the Rhode Island Rare Disease Foundation. I again thank the chairman and ranking member for including this measure in this legislation so that more families in Rhode Island and around the country can receive the same kind of good news that Sheri and many other cystic fibrosis patients received earlier this year.
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