Dr. Marty Makary on estrogen patch shortages and the role of the FDA
FDA Commissioner Dr. Marty Makary discusses the surge in demand and shortage of estrogen patches. He explains the FDA’s decision to withdraw the black box warning, which led to increased demand, and its impact on women’s health and menopause.
I spent over forty years on the front lines against a terminal illness that killed my brothers and left my fourteen-year-old son in a wheelchair. This week I have asked Food & Drug Administration (FDA) Commissioner Dr. Martin Makary will leave his gilded office and come visit my home because I fear he has lost touch with terminally ill children like Ryu.
I am Ryu’s full-time caregiver. Like most families with rare diseases, we rely on one income and have limited financial resources. What we do have are astronomical medical costs; Ryu’s steroids alone cost $30,000 a month. My husband and I luckily found a nonprofit that covered the remaining $2,000 after our insurance paid their share – but other costs are entirely our responsibility.
It’s easy to ignore these struggles when you’re a powerful official in Washington, DC. At my house, Ryu has trouble breathing because of Duchenne Muscular Dystrophy, the same disease that saw me kill my brothers Angelo and Antonio at the ages of 20 and 22. Experimental drugs could allow Ryu to avoid their fate, but we don’t have access to them in a regulatory environment that seems to prioritize bureaucratic caution over the lives of dying children.
I am a doctor and I fear that our healthcare institutions are facing increasing chaos
A year ago, the rare disease community thought Dr. Makary would be our strongest ally. He pledged to “remove barriers and exercise regulatory flexibility” to provide meaningful treatments for patients. Families listened and dared to believe that the system would finally reflect the urgency of the circumstances we face.
But our hopes were dashed time and time again as therapies were subjected to extensive review cycles, fast-track approval pathways were ignored, and previously approved treatments were paused, limited, or effectively sidelined.
The impending departure of FDA’s director of the Center for Biologics Evaluation and Research, Dr. Vinay Prasad, the man Makary appointed to make these life-and-death decisions, creates an opportunity for a reset. He oversaw the committee responsible for approving treatments, which is why he is seen by many in the rare disease community as responsible for delaying treatments rather than delivering them.
Now Dr. Makary the successor to Dr. Prasad believes he has an opportunity to appoint someone who understands that families should have the right to make their own decisions about which medications are “risky” for their declining loved ones.
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Risk is a big part of Ryu’s daily work, and our entire family is intimately familiar with it. At night he relies on a machine to keep his lungs functioning because they could fail at any moment. He always dreamed of becoming a Navy SEAL; now he just hopes to live long enough to cast his first vote.
Despite this, he is the happiest child I know. And it’s important that Dr. Makary knows what that looks like, because when he and Dr. Prasad talk about risk, they do so based on what the paperwork tells them. They make decisions that seem subject to review and revision from their offices.
But when I hear “risk,” I see Ryu’s quality of life irreversibly declining day by day. His calves are so tight that he can’t put his feet flat, so he wears special braces for some relief. During the day he wears a treadmill to realign his hips when his muscles deteriorate, and at night he can’t even turn around without our help.
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Under Ryu’s current treatment, these changes are irreversible. Therefore, treatment options must expand – and not at the speed of the FDA, but at the speed of terminal rare diseases.
Families understand risk better than anyone. We are not asking for reckless approvals or abandoning scientific standards. What we need is a system that recognizes that doing nothing is a decision that kills 100% of the time.
If the FDA wants to rebuild trust, it must start with the families who were burned. This means that patient communities are involved earlier in the process and are transparent about decision-making. Regulatory timelines must match the urgency of the diseases being addressed so that the current and ongoing discrepancy can be addressed.
FDA policies may be revisited, revised, and reversed. Leadership comes and goes. But for families like mine, there are no transfers. The accelerated approval process was created for a reason: because patients with rare diseases don’t get a reset.
That’s why this moment is important: because even if a ship is completely off course, a great leader can save the journey.
I invited Commissioner Makary will come sit in my living room and meet Ryu, to see firsthand what it means when decisions are postponed and hope is put on hold. Not as a symbolic gesture, but as a reminder that behind every request, every data set, every rejected therapy, there is a child whose future is determined.
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Ultimately, regulators will not be judged on how careful they were, but on whether their actions kept our children alive.
Families like mine cannot afford any more delays.
