My son is dying — families like mine can’t afford FDA delays and red tape

I spent more than 40 years on the front lines fighting a deadly disease that killed my siblings and confined my 14-year-old son to a wheelchair. this week me he asked Food and Drug Administration (FDA) Commissioner Dr. I want Martin Makary to leave his gilded office and visit my home because I fear he has lost touch with terminally ill children like Ryu.

I’m Ryu’s full-time caretaker. Like most rare disease families, we rely on a single income and have limited financial resources. We have astronomical medical expenses on our hands; Ryu’s steroids alone cost $30,000 a month. My husband and I thankfully found a nonprofit that covers the remaining $2,000 after our insurance pays its share — but other expenses fall directly on us.

It’s easy to ignore these struggles when you’re a powerful official in Washington, D.C. In my home, Ryu has trouble breathing due to Duchenne muscular dystrophy; This is the same disease that I watched kill my brothers, Angelo and Antonio, at ages 20 and 22. Experimental drugs may allow Ryu to avoid their fate, but we cannot access them under a regulatory environment that prioritizes bureaucratic vigil over the lives of dying children.

I AM A PHYSICIAN AND I AM CONCERNED THAT OUR HEALTH INSTITUTIONS FACE INCREASING CHAOS

A year ago, the rare disease society, Dr. He thought Makary would be our strongest ally. He promised to “remove barriers and implement regulatory flexibility” to deliver meaningful treatments to patients. Families listened and dared to believe that the system would eventually reflect the urgency of the circumstances we face.

But our hopes were dashed again and again as treatments were subjected to extended review cycles, accelerated approval pathways were ignored, and previously authorized treatments were paused, restricted, or virtually sidelined.

FDA’s Center for Biologics Evaluation and Research Director Dr. The impending departure of Vinay Prasad (the man Makary tasked with making these life-or-death decisions) creates an opportunity for a reset. He oversaw the committee responsible for treatment approvals, so he is viewed by many in the rare disease community as responsible for delaying treatments rather than delivering them.

Now Dr. Makary, Dr. As he sees himself as Prasad’s successor, he has the opportunity to appoint someone who understands that families should have the right to make their own decisions about which drugs are “risky” for their loved ones.

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Risk is a big part of Ryu’s daily life, and our entire family knows it intimately. He relies on a machine to keep his lungs working at night because they can fail at any time. He always dreamed of becoming a Navy SEAL; Now he hopes to live long enough to cast his first vote.

Despite this, he is the happiest child I know. And Dr. It’s important for Makary to know what this looks like, because he and Dr. When Prasad talks about risk, they do so based on what the paperwork tells them. They make decisions that seem like they could be reviewed and reconsidered from their offices.

But when I hear the word “risk” I see that Ryu’s quality of life is irreversibly decreasing with each passing day. His calves are so tight he can’t straighten his feet, so he wears special braces to get some relief. He wears a walking belt to align his hips during the day because his muscles are weakening, and at night he can’t even turn around on his own without our help.

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Based on Ryu’s current treatment, these changes are irreversible. That’s why treatment options need to be expanded, not at the pace of the FDA, but at the pace of deadly rare diseases.

Families understand risk better than anyone. We do not seek reckless approval or abandonment of scientific standards. What we need is a system that recognizes that doing nothing is 100% a fatal decision.

If the FDA wants to rebuild trust, it needs to start with the hurting families. This means involving patient communities earlier in the process and being transparent about decision-making. Regulatory timelines should be appropriate to the urgency of the diseases being treated and address existing and ongoing non-compliance.

FDA policies can be revisited, revised, and reversed. Leadership comes and goes. But for families like mine, there’s no redoing. There was a reason for the accelerated approval process: No zeroing in on rare disease patients.

That’s the importance of this moment: Because even if a ship is wildly off course, a great leader can save the voyage.

I invited Commissioner Makary will come and sit in my living room and meet with Ryu to see firsthand what it means to postpone decisions and suspend hopes. Not as a symbolic gesture, but as a reminder that behind every application, every data set, every rejected therapy, there is a child whose future has been decided.

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Ultimately, regulators will be judged not on how careful they are, but on whether their actions keep our children alive.

Families like mine can’t afford any more delays.