Washington, D.C. – In a Senate floor speech today, U.S. Senator Marco Rubio (R-FL) urged the Food and Drug Administration (FDA) to consider patients in its ruling on a drug intended to slow the progression of Duchenne Muscular Dystrophy (DMD).
In his speech, Rubio tells the personal story of Tampa native Austin Wilcher, who struggles with DMD, and his dad, Joe, who Rubio describes as “a hero in more ways than one.” Earlier this month, Rubio had the opportunity to meet with Austin and Joe to discuss ways to raise awareness about the fatal disease.
U.S. Senator Marco Rubio
Senate Floor Speech
April 28, 2016
Senator Marco Rubio: “I’d like to call attention today 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 the multiple different forms of muscular dystrophy. It affects mostly boys, at a rate of about 1 in 3,600 individuals.
“Its primary symptom is a steady deterioration of muscle mass beginning early in childhood. By age 12, most boys with Duchenne have lost the ability to walk. They eventually become paralyzed from the neck down.
“I’m sad to say there is currently no cure for Duchenne, and the average life expectancy is around 25 years.
“I am personally a parent of four children, that includes two boys, and I can only imagine -- perhaps I can’t imagine, that’s 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 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 named Austin, and his dad, Joe, who 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 up to Washington from his home in Tampa. But this is the embodiment of courage that people living with this disease show every day.
“So 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 with other daily tasks. He needs help with eating. Joe told me how 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 doctor’s appointments.
“Joe shared the dreams he once had when Austin was born – dreams of being that proud father in the bleachers at a Little League game, cheering loudly and waving a big foam finger. With Duchene, 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 is able to walk to the restroom. These are moments of great pride [for] Joe, when he sees just 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, are providing us greater insight into the way Duchenne 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 results of a clinical study on a small group admittedly, because this is a small group of people that have this disease. So any clinical trial is going to have a small number of people, not the same as you would have for another more common disease.
“And so this FDA advisory panel was meeting to decide whether or not they were going to allow this testing to expand and this drug to be more available.
“And it should have reviewed this in the context of a law that was passed in 2012, it’s 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. And 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.
“Because when it is a rare drug, usually if it’s a drug for cancer or something like that, you have tens of thousands of people that you can do a trial from. When it’s a rare disease, you have a harder time to find enough people to test it on the way you would on a normal drug.
“And on top of that, on top of the perspective of the less number of people, it’s also a disease that’s fatal. In the end, all of these cases of Duchenne end the same way - with death, in a very predictable pattern.
“So they had a chance to meet this week and review this in the committee and in the words of someone who was there, who has a lot of experience in interacting with government agencies and bureaucracy, this is the word they used, they said, it was ‘jarring.’ This is from someone who says he’s been exposed to a lot of different bureaucratic agencies. It was ‘jarring’ how it went.
“So I want to paint the picture of what that place looked like on Monday.
“You had an entire community of parents whose kids have Duchenne, who are taking this experimental drug, who are seeing their kids improve. They’re seeing it. They know these kids better than any scientist, than any doctor, than any panelist at the FDA and they see these kids are doing better. They see this.
“And they are begging with the FDA panel, ‘Please, allow us to continue to give these kids this medicine. And by the way, make it available to other kids.
“‘Because in the end, number one, there’s not been a single documented case of harm. No one using this experimental medicine has been harmed by it.
“‘Number two, we, the parents, are telling you it works because we see it in our kids.
“‘And number three, if you take it away, we’re desperate, there’s nothing left. They’re gonna die. It’s very predictable.’
“The committee ignored them. The committee ruled against them. And it did so because they applied basically 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, because they aren’t enough people to do a clinical trial with that is a rare disease.
“And the result is, they had this ruling, I think the vote was 7-3.
“What’s interesting is, one of the board members was later quoted as saying, ‘Based on all I heard, the drug definitely worked, but the question was framed differently.’
“What it means is, the way the FDA posed the question to this committee was not does whether the drug work or not, but the question was the process.
“Was this clinical trial… ‘Did it have enough people? Was it conducted the normal way, the way other drug tests are conducted?’ Of course not. Because it’s not treating a normal condition, it’s 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.
“Had the FDA, by the way, followed FDASIA, the law that was passed a few years ago, taken that into account, the small patient population, and likewise, they might have reached a different result.
“And so instead, what’s happening now is that these patients and these families are on the verge of losing, not just access to the drug, but other families as well.
“So I want you to put yourself in the position of one of these patients. Your son has Duchenne. Your son is taking this experimental drug. You see how they are 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’s a steady, predictable decline.
“So imagine if your children or one of those children is impacted by this disease and you know what the outcome is. It is [a] predictable, guaranteed outcome and they are taking an experimental drug and you know that it’s working because they’re not declining.
“In fact, in my cases they’re improving. And you are begging the FDA, ‘Please, allow us to continue to give our children this drug.’ And they say to you, ‘No, we reject it because the clinical trial was not conducted the way it is for normal drugs.’
“And you understand the desperation of these parents. There’s one last chance.
“The FDA has the ability, the senior leadership, to override this decision and allow this to move forward. I personally hope that’s what they’ll do.
“The only thing to lose here is to do nothing.
“The sad story here would be for these parents who are already seeing benefits of it. To lose access to this drug that they know is having an impact on their children.
“And no one has been able to prove to any threat that this drug poses to these children.
“This has been documented, and I encourage people to go on… CBS has done a report on this, other entities over the last few days have reported on it.
“The FDA has a chance now, at its senior leadership, 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 anyways, if we don’t do something. And here’s a drug that is showing improvement and families that are using it are begging you 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 stuff wasn’t working, these parents would not be so adamant about it. They see that it’s working. They know people that it’s working for. And they are desperate to keep it or to reach it.
“Listen to them. They know what they’re talking about. They are the primary caregivers of their children and they know improvement when they see it.
“And I hope that the FDA will consider moving in a different direction. These parents deserve better.”