Energy and Water Development and Related Agencies Appropriations Act, 2016

Floor Speech

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Ms. KLOBUCHAR. Madam President, I rise today to discuss this urgent public health emergency. I am honored to be here with Senator Murray, Senator Mikulski, Senator Heitkamp, and Senator Hirono as we look at this serious crisis facing our Nation, and that is the Zika virus.

The World Health Organization has declared that Zika is spreading explosively and will affect nearly all countries in North America and South America. The virus has already infected nearly 400 Americans who have traveled abroad from 40 States, including my home State of Minnesota. Over 500 people in Puerto Rico have the disease. Nearly all of them contracted the virus locally. These numbers will only continue to grow as the warmer months bring more mosquitoes that transmit this disease. In fact, researchers calculate that 60 percent of the people in our country live in an area that will likely be affected.

Zika is a rapidly evolving mosquito-borne virus. Most infected patients develop mild flu-like symptoms that last for a week. However, the virus has devastating consequences for growing families. Researchers have now confirmed what many feared was true: A pregnant woman infected with Zika is at risk of giving birth to a child with microcephaly. This heartbreaking, lifelong condition results in newborns with abnormally small heads. These children will need increased access to health care and developmental services, such as speech therapy, occupational therapy, and physical therapy. There is no known cure for this disease or even standard treatment for this condition.

It is crucial that physicians have the knowledge and tools essential to diagnose and care for pregnant women who may be infected with Zika. It is crucial that moms with Zika and children with microcephaly have access to the services they need. It is crucial that we take steps now to ensure that our health care system and all levels of government are prepared for the imminent spread of the Zika virus.

We are here today to continue to stress the urgent need to ensure that our country is as prepared as possible to mitigate the spread of Zika and respond to outbreaks of this virus.

The administration submitted a request for nearly $2 billion in emergency funds to provide immediate support. This is about research. This is about a vaccine. This is about therapeutics and diagnostics. This is about a medical health crisis that primarily--but not only-- affects women and children. That is why the women Democrats of the Senate have gathered on the floor today to speak out, to speak out and say this is a crisis that must be funded. This is a crisis that must be responded to.

Simply because it mainly affects women and children right now--and we have no idea what other effects it will have--is no reason to shirk our duties in the Congress and not fund this. Our foremost duty is to protect the health and safety of Americans. Zika is a rapidly evolving disease with severe public health implications. I ask my colleagues to support this effort. We cannot afford to delay action.
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Ms. KLOBUCHAR. Mr. President, I rise today as the cochair of the Rare Disease Congressional Caucus in recognition of patients with Duchenne muscular dystrophy and the loved ones who care for them.

Duchenne is a devastating, rare disease that primarily affects boys and young men. There is no cure. It is 100 percent fatal. There are no approved disease-modifying treatments at this time, but we want to give them hope. In 1999, there were no human clinical trials for Duchenne. Today, there are 22 observational trials currently underway. Life expectancy rates have increased by about 10 years in just the past decade. The FDA has more tools in its toolbox than ever to accelerate approvals of safe and effective Duchenne therapies, but we would like more therapies to be approved in the future.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 male children. The disease results in the gradual loss of muscle strength, usually beginning before age 5. The progressive muscle weakness leads to serious medical problems, particularly issues related to the hearts and lungs. By age 14, over 80 percent of these boys are using wheelchairs.

My work on Duchenne muscular dystrophy began when I was elected to the Senate. It was an issue my dear friend and former Minnesota Senator, Paul Wellstone, championed. Paul was instrumental in getting the Muscular Dystrophy Community Assistance Research and Education Act--or as it is known, the MD-CARE Act--signed into law back in 2001.

The bill dramatically increased investment at the National Institutes of Health for muscular dystrophy research and included funding for the creation of six centers of excellence. In recognition of his work, all of the centers share Senator Paul Wellstone's name. The bill also supported public health policies designed to improve quality of life and boost life expectancy of children and adults diagnosed with muscular dystrophy.

Since passage of the MD-CARE Act, $500 million has been leveraged for muscular dystrophy research and education programs, half of which is Duchenne-specific. I then led the reauthorization of the MD-CARE Act in 2008, and it passed the Senate by unanimous consent. In 2014, Senator Roger Wicker and I led the MD-CARE Amendments of 2014, which built upon the progress by ensuring that efforts are focused on the most critical needs of doctors, patients, and researchers. These are important accomplishments, but more needs to be done.

The Food and Drug Administration Safety and Innovation Act of 2012 gave the FDA increased flexibility to grant accelerated approval for rare disease treatments that have proven to be beneficial. The bill also directed the FDA to use patient-focused drug development tools during the drug approval process. The idea is simple: Patient experience should be a factor when the FDA considers a drug for approval. This gives the FDA the opportunity to hear directly from patients, their families, and caregivers about the symptoms that matter most to them, the impact the disease has on patients' daily lives, and their experiences with treatments.

To build upon that progress, Senator Wicker and I introduced the Patient-Focused Impact Assessment Act. The bill would help advocates understand how the FDA uses patient-focused drug development tools and how it engages patients, including those with rare diseases, such as Duchenne, as it reviews drugs and therapies. Last month this bipartisan bill unanimously passed the Senate Health, Education, Labor, and Pensions Committee, bringing us one step closer to ensuring strong patient engagement throughout the FDA review process.

At an FDA meeting on Monday, there was one example of patient involvement in the drug approval process. It was a meeting that broke records. According to advocates, it was the largest gathering of Duchenne families in history. More than 900 members of their community were there. In fact, turnout was so large the FDA changed the meeting location to accommodate everyone.

Many stories were shared during the daylong meeting--stories of hope, stories of progress. Even seemingly small improvements--such as the ability to open a bottle of water on their own or lift their arm a little higher--make a huge difference in the quality of these boys' lives. These small victories have a ripple effect across a lifetime.

Monday's historic event shows the strength of the Duchenne community, the passion of the families, and the hope that treatments are on the horizon. This particular treatment was not approved that day, but we continue to hold hope that change will be on the horizon.

The fight against muscular dystrophy will not be won overnight, but we have already seen incredible progress in the last few years. I am confident that by working together--by bringing families to the table with policymakers and health care experts--we can accomplish some truly remarkable things.

One of the reasons Senator Wicker and I fought so hard to have the FDA officials listen directly to the families is that when you know your child has a disease that is 100 percent fatal, you might take different risks. You might see different improvements in a different way than a medical professional who does not have this experience. We hope going forward this kind of experience and testimony and information will make for better decisions by the FDA.

We need to continue to ensure the FDA has the tools and flexibility it needs to increase the number of safe, effective, and affordable treatments that are available for people with rare diseases. I also thank Senator Hatch, who has done a lot of work with me on the rare disease issue, and we will continue to push for cures for people who have so little hope.

I thank the Chair, and

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