Energy and Water Development and Related Agencies Appropriations Act, 2016
Floor Speech
BREAK IN TRANSCRIPT
Mr. RUBIO. Madam President, I have two topics I want to talk about today--actually, three--but I want to begin with the Zika virus.
A few weeks ago I went back to Florida on a Friday and I sat down and met with officials from the Department of Health from Florida. I met with leaders from Puerto Rico in the health sector. I met with doctors who live in Miami-Dade County and also officials in Miami-Dade County. They are freaked out about the Zika thing. I don't know any other term to use. If they are freaked out, then I am very concerned about it as well. That is why I do support fully and immediately funding this situation, and I have asked our colleagues to do so as quickly as possible.
I want to speak briefly about the Florida experience with this. There are two things that are deeply concerning, and then I will speak to some of the things we should be doing.
First, the summer months are upon us. Anyone who has been in Florida, in the summer particularly, knows summer has basically already started in Florida if you go outside. The spread of mosquitoes as a threat virtually everywhere in the State is just massive. It is just a way of life. This very deadly disease is something we are still learning about, by the way. A few weeks ago, they said: Well, Zika impacts only a small population of people--a very significant population of people. We are learning this disease impacts whoever it touches. First of all, you don't have to be symptomatic to spread it. In Florida alone, we have had at least two cases of transmission sexually transmitted.
By the way, it is just a matter of time before someone in Florida gets bit by a mosquito. I am telling you, it is just a matter of days, weeks, hours before you will open up a newspaper or turn on the news and it will say that someone in the continental United States was bitten by a mosquito and they contracted Zika. When that happens, then everyone is going to be freaked out, not just me and not just the people who work for the health department in Florida. This is going to happen. There are just way too many mosquitoes to avoid it.
The second thing is that Miami-Dade County, in particular, but a lot of Florida, is a transit point for all of Latin America. So, for example, one of the places most impacted by Zika is Brazil. Well, this summer the Olympics are being held in Brazil, and there will be hundreds of thousands of people who cross through Florida to get to Brazil and back, on top of the normal number of travelers. It is just a matter of time. It is not a question of if, it is a question of when.
So I look at this from a Senate perspective and say: We are going to fund this. We are going to spend money on Zika in Washington, DC, No. 1, because we should. It is the obligation of the Federal Government to keep our people safe, and this is an imminent and real threat to the public safety and security of our Nation and our people. So the money is going to be spent. The question is: Do we do it now, before this has become a crisis or do we wait for it to become a crisis? Maybe that crisis happens in August, when everyone is back home doing their campaign stuff or maybe it happens on Monday, when everyone is back home doing whatever they do on recess. Then everyone will get pulled back to deal with this immediately, and I want to know what Members will say to those who say: Hey, this Zika thing has been in the news for months. Now there is a case.
It can be in any State in the country--any State in the country. You may hear: Oh, it is only in certain States that are warm. That is not true. It can be in any State in the country. I want to know what people are going to say when they are asked: What did you do about it? Are you going to say: Well, I had real problems. I wanted to make sure about this and that.
This is a serious thing. People's lives are at stake here. And by the way, this is now spreading into all sorts of other threats. Guillain- Barre was mentioned earlier. We know about the birth defects that are very significant. Do my colleagues realize what the cost will be of dealing with all of that? Are people aware of what Guillain-Barre is? It is a debilitating, often fatal, disease. The cost of treating someone that has it is extraordinary.
What about where the money is going to be spent? Look, it is possible at the end of the day that $1.9 billion will not even be enough. We don't know. But we have to start.
No. 1, we don't have a commercially available plan to test for Zika. You can't just go to Quest Diagnostics and get a Zika test. It doesn't exist. In Florida, if you want to get a Zika test, you have to go through the State department of health.
No. 2, a lot of people aren't being tested because they are not a pregnant woman so they do not think they have to be tested. False. If you have traveled anywhere at this point--I don't care who you are, how old you are, male or female--where there are mosquitoes in significant amounts, you probably should be tested. If you have traveled abroad into these danger zones, you can transmit this disease. You can be carrying it and not see manifestations of it for a while.
There is no commercially available plan. They talk about mosquito control. They have only been trying that for thousands of years, and mosquitoes have outlasted everything. It is important. It has to be a part of it. But one of the two mosquito species that spreads Zika is resistant to pesticides. It has become resistant to the pesticide, and that is why new technologies need to be developed.
There are some innovative ways out there to cut down on the mosquito population. There is an innovative program now, trying to start a pilot program in the Keys. That should be a part of this conversation. Researchers are pretty confident they can find a vaccine for this kind of disease, given its pathology. Maybe not next week, but they can find a vaccine for it. The government has a role to play in basic research that allows the private sector to commercialize that and make that possible.
I understand we want accountability for how this money will be spent. I believe that. I do. I think the administration should come forward and say: Here is our plan. Here is where every penny is going to be spent, and here is how we are going to spend it. We should hold them accountable, and if there are ways to improve on that, we should. But I think there should be a sense of urgency when dealing with this issue.
I honestly believe--I don't believe; I know--it is just a matter of time before there is a mosquito-borne transmission. By the way, does it really matter how you got it, whether it was from a mosquito or it was sexually transmitted? You have Zika. It acts the very same way once you have it. It is just a matter of time before there is a mosquito-borne transmission in the continental United States.
I also have heard--not that anyone here has said it--but I have heard others say there are no cases of Zika transmitted from a mosquito yet in the United States. That is false. Puerto Rico is in the United States. Puerto Ricans are American citizens. By the way, they travel in huge numbers to and from the United States. Many are moving here. Many work here during the week and travel back on the weekends. This is a catastrophe right now in Puerto Rico, which is a United States territory, and its people are American citizens. They are facing a catastrophe right now on this issue.
So I hope there is real urgency about dealing with this. I understand this is not a political issue. There is no such thing as a Republican position on Zika or a Democrat position on this issue because these mosquitoes bite everyone. They are not going to ask you what your party affiliation is or who you plan to vote for in November. This is a real threat, and it is not just in the tropical States. They may feel it first, but so can any State that has any significant travel, which is basically all 50 States in the Union. In a country where people travel extensively across the country and around the world, we are going to face a Zika problem in this country this summer and fall.
My advice to my colleagues is that we are going to deal with this, so I hope we deal with it at the front end. Not only is that better for our people, but that will be better for my colleagues. Otherwise, we will have to explain why it is that we sat around for weeks and did nothing on something of this magnitude.
The second topic I want to--
BREAK IN TRANSCRIPT
Mr. RUBIO. I will yield to the Senator from Washington.
BREAK IN TRANSCRIPT
Mr. RUBIO. Madam President, I appreciate the advocacy of the Senator from Washington, and I do look forward to working with the Senator on this as well. Hopefully, we can get a result on this.
There is going to be a recess now, and that means for 10 days people will be going back to their home States. So I hope when we come back a week from Monday, we will hear that we have a plan that we are going to be able to vote on and vote on it quickly. Duchenne Muscular Dystrophy and FDA Advisory Panel
Madam President, on a separate topic, I want to call attention to a remarkable group of advocates who are bound together, not by a common race or religion or political ideology but by the common hope of one day ridding the world of a rare disease named Duchenne muscular dystrophy.
Duchenne is one of multiple different forms of muscular dystrophy. It affects mostly boys, almost exclusively, at the rate of 1 per 3,600 individuals. Its primary symptom is the steady deterioration of muscle mass beginning early in childhood. By the age of 12, most boys with Duchenne have lost the ability to walk and eventually become paralyzed from the neck down. I am sad to say there is currently no cure for Duchenne, and the average life expectancy is around 25 years.
I am personally the parent of four children, including two boys, and I can only imagine--perhaps I can't imagine; that is how difficult it is--what it must be like to have a child receive this diagnosis. Few are called to do more for their child and to show greater courage in the face of the adversity that MD poses than a parent helping their child battle Duchenne.
I was recently inspired and humbled a few weeks ago to meet a young man struggling against this disease. His name is Austin, and his dad Joe is a hero in more ways than one. Joe helps Austin combat Duchenne, and he does it alone, as a single father. By the way, he also serves as an Active-Duty member of the United States Air Force.
Austin is 12 years old, and I was immediately impressed when I met him. I knew how difficult it must have been for him to travel all the way to Washington from his home in Tampa. This is the embodiment of courage that people living with this disease show every day.
Joe shared with me a few of the struggles they face. He told me how Austin is unable to attend school full time because he needs hours of daily physical therapy to stimulate his muscles. He told me how Austin is quickly losing the ability to walk and how he now needs help getting in and out of his wheelchair and other daily tasks. He needs help with eating.
Joe told me he spends hundreds of dollars each month on over-the- counter drugs that are not covered by insurance, and he spends hours every Friday attending doctors' appointments.
Joe shared the dreams he once had when Austin was born--dreams of being that proud father in the bleachers at little league games or cheering loudly and waving a big foam finger. With Duchenne, he tells me he has even more reasons to proudly cheer Austin on, though the reasons are different. He cheers when Austin is able to get out of bed without help or to walk to the restroom. These are moments of great pride for Joe, when he sees how resilient Austin is in the face of this disease.
Joe and Austin traveled to Washington as part of a coordinated effort to witness and participate in FDA action related to Duchenne. As advancements in medical science continue, targeted therapies to treat Duchenne are being developed and tested, and each one--even the ones that fail--is providing us greater insight into the way the disease operates and how it might ultimately be defeated.
The last couple of weeks in particular have brought about a display of extraordinary strength from Joe and Austin, and thousands of other parents, children, family, and friends who engage in activism on behalf of those with Duchenne. This Monday, scores of advocates from around the country attended a hearing of the FDA advisory committee, which welcomed them and spent almost an entire day listening to their testimony. What this committee was listening to was the result of a clinical study on a small group. Admittedly, this is a small group of people who have this disease, so any clinical trial will have a small number of people. It is not the same as you would have for another more common disease. So this FDA advisory panel was meeting to decide whether they were going to allow this testing to expand and this drug to be more available.
The panel should have reviewed this in the context of a law that was passed in 2012 called the Food and Drug Administration Safety and Innovation Act; call it FDASIA for short. This act gave the FDA the authority to consider the perspectives of patients when evaluating whether to approve a drug. In essence, it gave the FDA the authority to listen to people who are taking the drug and decide whether it works or not--not just to look at the clinical study.
This also provides real flexibility when evaluating drugs for life- threatening illnesses, such as Duchenne. It included multiple provisions to address the challenges of the rare disease patient community, which is by definition small--meaning clinical trials have a more difficult time finding enough participants to meet the FDA's usual requirements. Usually, when it is a drug for cancer or something like that, you have tens of thousands of people you can do a trial for. When it is a rare disease, you have a harder time finding enough people to test it on the way you would for a normal drug. And on top of that--on top of the perspective of a lesser number of people--it is also a disease that is fatal. In the end, all of these cases with Duchenne end the same way, with death, in a very predictable pattern.
They had a chance to meet this week and review this in the committee. In the words of someone who was there, who has a lot of experience in interacting with government agencies and bureaucracies, the word they used was ``jarring.'' They said it was jarring. This is from someone who has a lot of experience interacting with government agencies and bureaucracies. They said it was jarring how it went.
I want to paint the picture of what that place looked like on Monday. There was an entire community of parents whose kids have Duchenne, who are taking this experimental drug, who are seeing their kids improve. They are seeing it. They know these kids better than any scientist, any doctor, or any panelist at the FDA, and they see these kids are doing better. They see this. They are begging the FDA panel: Please allow us to continue to give these kids medicine. And, by the way, make it available to other kids because, No. 1, there has not been a single documented case of harm; no one using this experimental medicine has been harmed by it. No. 2, we, the parents, are telling you it works because we see it in our kids. And, No. 3, if you take it away, we are desperate; there is nothing left. They are going to die. It is very predictable.
The committee ignored them. The committee ruled against them, and it did so because they basically applied the same standard to this drug as they did to a normal one: Oh, you didn't have enough people in the clinical trial. No, there aren't enough people to do a clinical trial with. It is a rare disease. The result is they had this ruling, and I think the vote was 7 to 3.
What is interesting is that one of the board members was quoted as saying: Based on all I heard, the drug definitely works, but the question was framed differently. What that means is the way the FDA posed the question to this committee was not just whether the drug worked, but the question was the process: Did this clinical trial have enough people? Was it conducted the normal way--the way other drug tests are conducted? Of course not, because it is not treating a normal condition. It is one with a very small population.
The committee spent almost the entire time focused on how the clinical study was designed and not on whether it works. By the way, had the FDA followed FDASIA, the law passed a few years ago, and taken that into account--the small patient population and likewise--they might have reached a different result. Instead, what is happening now is these patients and families are on the verge of losing not just access to the drug but to other families as well.
Put yourself in the position of one of these patients. Your son has Duchenne, your son is taking this experimental drug, and you see how he is improving--because you do not improve with Duchenne. It is not one of these things where you get better, worse, better, worse. You get worse and then worse and then worse. It is a steady, predictable decline. So imagine your child is one of those impacted by this disease. You know what the outcome is. It is a predictable, guaranteed outcome. They are taking an experimental drug, and you know it is working because they are not declining. In fact, in many cases they are improving. You are begging the FDA: Please, allow us to continue to give our children this drug. They say: No, we reject it because the clinical trial was not conducted the way it is for normal drugs. Then you would understand the desperation of these parents.
There is one last chance. The senior leadership of the FDA has the ability to override this decision and allow this to move forward. I personally hope that is what they will do. In the end, the only thing to lose here is to do nothing.
The sad story here would be for these parents, who are already seeing the benefits, to lose access to this drug that they know is having an impact on their children. No one has been able to prove there is any threat that this drug poses to these children. This has been documented. CBS has done a report. Other entities have reported on it.
FDA senior leadership has the chance to overrule this committee, which didn't knock it down for purposes of safety or anything of that nature. They just said the clinical trials didn't meet their standard-- and say these kids are going to die anyway if we don't do something.
Here is a drug that is showing improvement, and families who are using it are begging them to allow them to use it. Thousands of people do not fly in from around the country or watch online for something that isn't working. If this weren't working, these parents would not be so adamant about it. They see it is working. They know people it is working for. They are desperate to keep it or to reach it. Listen to them. They know what they are talking about. They know. They are the primary caregivers for their children, and they know improvement when they see it.
I hope the FDA will consider moving in a different direction. These parents deserve better.
BREAK IN TRANSCRIPT
Source