Dr. Marty Makary on estrogen deficiency episodes and the role of the FDA
FDA Commissioner Dr. Marty Makary discusses the rise in demand and shortage of estrogen patches. She explains the FDA’s decision to remove the black box warning, leading to increased demand, and its impact on women’s health and menopause.
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I have spent over 40 years battling a deadly disease that killed my brothers and put my 14 year old son in a wheelchair. This week, I asked the Commissioner of the Food & Drug Administration (FDA) Dr. Martin Makary to leave his shiny office and visit my home, because I fear he has lost touch with terminally ill children like Ryu.
I am Ryu’s full-time caregiver. Like many rare disease families, we rely on one income and have limited financial resources. What we have is astronomical medical costs; Ryu’s steroids alone are $30,000 a month. My husband and I thankfully found a non-profit that covered the remaining $2,000 after our insurance paid part of it – but the rest of the cost falls entirely on us.
It’s easy to overlook these struggles when you’re a powerful executive in Washington, DC In my house, Ryu struggles to breathe because of Duchenne muscular dystrophy, the same disease that I watched kill my brothers Angelo and Antonio at the ages of 20 and 22. Experimental drugs may allow Ryu to avoid their fate, but we cannot access them under the control zone that allows children to have priority in children’s lives.
I AM A THERAPIST AND I AM CONCERNED THAT OUR HEALTH ORGANIZATIONS ARE FACING INCREASING CHAOS
Last year, the rare disease community thought that Dr. Makary will be our most powerful assistant. He promised to “remove barriers and use regulatory flexibility” to bring meaningful treatment to patients. The families listened and had the courage to believe that this plan would finally show the urgency of the situation we are facing.
But our hopes have been dashed time and time again as treatments have been subjected to an extended cycle of review, accelerated approval processes have been ignored, and previously approved treatments have been suspended, discontinued, or effectively sidelined.
The upcoming departure of the FDA’s Director of the Center for Biologics Testing and Research, Dr. Vinay Prasad, the man Makary assigned to make these life and death decisions, creates an opportunity to reset. He chaired the committee responsible for the approval of the treatment, so he is seen by many in the rare disease community as responsible for delaying treatment rather than bringing it.
Now, as Dr. Makary considering the successor of Dr. Prasad, you still have a chance to nominate someone who understands that families should have the right to make their own decisions about which medications are “harmful” to their debilitated loved ones.
LUCKILY LOUISIANA IS FIGHTING STATE RED TAPE PREVENTING CHILDREN WITH SPECIAL NEEDS FROM THE MORNING
Risks are a big part of Ryu’s day to day, and our whole family is familiar with them. At night, he relies on a machine to keep his lungs working because they could 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 it is important that Dr. Makary knows what that looks like, because when he and Dr. Prasad talking about the accident, they did so based on what the papers told them. They make decisions, in their offices, it seems that they can be reviewed and reconsidered.
But when I hear “danger,” I see Ryu’s health level irreversibly dropping by the day. His calves are so stiff that he can’t make his feet flat, so he wears special supports to keep them comfortable. During the day he wears a mobility belt just to keep his hips aligned as his muscles atrophy, and at night, he can’t even turn himself around without our help.
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Under Ryu’s current treatment, these changes are irreversible. That’s why treatment options must be expanded – not at the speed of the FDA, but at the speed of rare, deadly diseases.
Families understand risk better than anyone. We are not asking for reckless approval or abandonment of scientific standards. What we need is a system that recognizes that doing nothing is a fatal decision, 100% of the time.
If the FDA wants to rebuild trust, it needs to start with burned families. That means involving patient communities early in the process and being transparent about decision-making. Control periods should be consistent with the urgency of the diseases being treated, to treat current and ongoing discrepancies.
FDA policies can be reviewed, revised, and reversed. Leadership comes and goes. But in families like mine, there are no do-overs. The expedited approval process was created for a reason – because rare disease patients don’t get a reset.
That is why this moment is important: because even if the ship is sailing badly, a great leader can save the voyage.
I invited Commissioner Makary to come sit in my living room and meet with Ryu, so he could see for himself what it means when decisions are delayed and hope suspended. Not as a symbolic gesture, but as a reminder that behind every app, every dataset, every rejected treatment, there is a child whose future is being decided.
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In the end, regulators will not be judged by how vigilant they were, but by how their actions kept our children alive.
Families like mine cannot afford more delays.
