The Food & Drug Administration’s job is to make sure safe treatments and drugs are available for Americans. Chief science officer Dr. Vinay Prasad instead spent almost a year blocking Americans in the rare disease community from accessing life-changing new drugs. His recent departure from the FDA was a rare piece of good news from the agency.
But, as a Wall Street Journal op-ed noted last week, one man’s departure can’t change an agency’s culture.
Just days after Prasad announced he was leaving, Andrew Nixon, Deputy Assistant Secretary for Media Relations at the U.S. Department of Health and Human Services and a regularly cited spokesperson about FDA matters, used his X Account to refer derisively to advocates for Huntington’s disease treatments — that is, the sons, daughters, spouses, and parents fighting to keep the people they love alive — as “the swamp.”
Although that appalling post is now deleted, screenshots of his comment are still circulating online. And it raises serious questions about how patients and their families are viewed inside the very institutions meant to serve them.
I’m an American living with a disability. I worked at HHS for President Donald Trump in his first Administration and am an advocate who knows that people facing conditions like Huntington’s disease, Sanfilippo syndrome, and Duchenne muscular dystrophy are not lobbying for convenience or profit. They are not playing politics. Their advocacy is a fight for more time, longer lives, better treatment, and the basic dignity of hope.
Over the last year, patients, caregivers, and rare disease advocates have voiced concerns about the FDA delaying promising therapies. These concerns are grounded in the daily reality of parents watching their children lose function as terminal conditions take their cruel toll — families who have no choice but to endure the ticking clock while potential treatments remain out of reach.
Recent developments suggested that those concerns were finally being taken seriously. Amid growing public and congressional pressure, leadership changes at the FDA — including the departure of Dr. Prasad from the Center for Biologics Evaluation and Research, where approval decisions live or die — signaled accountability might be on the way.
Thankfully, some leaders in Washington are paying attention. Sens. Rick Scott (R-Fla.) and Ron Johnson (R-Wis.) have used their work through the U.S. Senate Special Committee on Aging to shine a necessary spotlight on the struggles facing rare disease sufferers and their families. Their willingness to ask tough questions and demand accountability is a reminder that patient voices are finally being heard in places that matter.
President Trump has also demonstrated a real sensitivity to the suffering of people confronting life-threatening conditions. His support for the Right to Try initiative recognized a simple but powerful truth: when time is running out, access to promising treatments can mean everything. The patients with those diseases and their families, not a bureaucrat in Washington, should be able to decide whether a drug is too “dangerous” to take.
Rare disease communities have long been supporters of innovation. Their persistence has driven research, accelerated development timelines, and, in many cases, made medical breakthroughs possible. They are not the problem, but a big part of the solution.
It’s too bad that the FDA’s leadership and senior staff don’t seem to recognize that. And it’s why leadership changes alone are not enough if the culture inside powerful institutions does not change with them.
Patients and their advocates are not the swamp — and no one can drain the love that drives us to fight for hope and a better life for people living with rare diseases.
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