In 2018, Congress finally passed one of Donald Trump's priorities in his first campaign – the Right to Try Act. Trump had picked up on frustration with an overly bureaucratic Food and Drug Administration and its response to terminal or near-terminal diseases. While the process used by the FDA for drug approvals emphasizes safety, it can take a decade or more to get approvals, especially for rarer diseases that make multiple double-blind studies difficult to complete. Trump's legislation allowed patients and families access to drugs in the study process and/or pending approval while waiving the risks of experimental treatment.
Or so we thought. Angeline Olivera has had a much different experience with the FDA, and she wants answers. She wrote an op-ed for USA Today a week ago, revealing all of the obstacles and bureaucratic logjams she has faced to get medication for her son Ryu. Ryu suffers from a rarer form of muscular dystrophy called Duchenne's (DMD), and at 14 is beginning to run out of time to get the necessary help to slow down the progress of the disease:
Eight years ago, President Donald Trump signed the Right to Try legislation to give people with terminal illnesses a chance at life. Now his Food and Drug Administration has undermined those principles by stealing the chance for my son to live past his early 20s. ...
I’m a mother of three living in El Paso, Texas. My 14-year-old son, Ryu, lives with Duchenne muscular dystrophy – a disease that killed two of my brothers. I said goodbye to my youngest brother, Antonio, as Ryu, who was diagnosed as a newborn, was sitting at my feet. ...
After spending much of my life in clinics and hospitals, I’ve seen people bravely face a difficult future. I also know that hope lives when elected officials, regulators and researchers prioritize the principles of Right to Try. Kids with Duchenne muscular dystrophy and their families deserve the chance to decide their own future.
The prognosis for DMD without medical intervention is grim, Angelina explains. Most will die in their early twenties or sooner. That means the clock is ticking for Ryu, and the good news is that the FDA has at least one medication in trials. The bad news is that the FDA halted the trial after two adverse outcomes, and with that, so Ryu's access to Elevidys has been cut off. Rachel Alexander explained it at Townhall before Angelina's essay was published:
Elevidys was approved for a few types of patients, but since a couple of non-ambulatory patients in the trial treatment died of acute liver failure afterwards, the FDA halted access to those in wheelchairs like her son, who lost his ability to walk at age 9. All AAV-delivered gene therapies have a risk of liver injury. The risk is small – liver injury leading to death has occurred in two out of more than 1,200 treated patients and both were older patients with more advanced disease. Furthermore, the risk is manageable with monitoring and enhanced immunosuppression. The two DMD patients who died were in advanced stages, and a third fatality occurred in a separate trial of a higher-dose gene therapy for a different muscle disease.Their son has staggering medical costs, including a $30,000 power wheelchair and steroid medications that average $30,000 per month. The Muscular Dystrophy Association used to cover the costs that insurance did not pick up, but recently stopped the reimbursements, stating that the money would be going to research instead. Olivera said this is unfair since they're not allowed to receive the benefit of the research.
Needless to say, this has frustrated and frightened Angelina, who relied on the Right to Try legislation to maintain access to Elevidys. This is not a question of long-term safety, Angelina argues, but of short-term survival:
The Trump administration once championed agency and choice. Where is that spirit now? The FDA should be guided by the principle that patients have a say over what happens to their own bodies. Somewhere along the way, that clarity has been lost.
Trump has already said that families deserve a voice in life-and-death medical decisions. Duchenne families are simply asking that this principle be carried through in practice. We are not asking for shortcuts.
We are asking for the same agency that was once championed as a fundamental right of terminal patients.
Rachel Alexander asked the same question, and aimed it at HHS Secretary Robert F. Kennedy Jr:
Maybe U.S. Secretary of Health and Human Services Robert F. Kennedy Jr., who has pushed for faster drug approvals in areas like rare diseases, can apply some pressure. He could replace FDA leaders and overrule or influence advisory committee recommendations and center-level decisions. Sen. Ron Johnson (R-WI) proposed a Right to Try 2.0 earlier this year, which would build on his original 2018 law to address rapid scientific advancements, speeding up approval of treatments for rare diseases that don't fit traditional large-scale FDA trials.
Why are the bureaucrats at the FDA so timid? People die from taking risky legal substances like alcohol all the time, and we don't restrict alcohol. Why not let someone who is going to die young in a few years take a chance?
On Friday, Angelina Olivera met with me to discuss the issue and her son Ryu's need for access to Elevidys. She understands the risk, and so does Ryu, who is now confined to a wheelchair.
"My son is 14," Angelina tells me. "Without medications, without a treatment, he could die tomorrow. The FDA seems more focused on bureaucracy versus patients' lives. And," she continued, "I don't know why it's such a slow process to get anything approved with the FDA. And I'm hoping by speaking up and other families speaking up, we can finally get even that answer."
Angelina's a fighter. If anyone can get those answers, she can – but she needs support to succeed. Contact your member of Congress and/or Senator to push for those answers, while Ryu can still benefit from them. Ryu is why Trump pushed for the Right to Try legislation, and if it's not helping him, is it doing any good at all?
Follow Ryu's fight on Instagram, and watch Angelina's fight right here in this interview.
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