“From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation.” The US Senate Special Committee on Aging will convene a hearing with the FDA. Submit your statement!

On Thursday, February 26, Chairman Rick Scott of the U.S. Senate Special Committee on Aging will convene a hearing entitled “From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation.” This hearing will explore how regulatory processes and evolving standards at the Food and Drug Administration (FDA) can unintentionally delay patient access to safe and effective therapies, particularly for individuals living with rare diseases. Members and expert witnesses will share the life-altering impacts of these delays on patients and their families. The hearing will also explore opportunities to improve regulatory clarity and predictability, discuss how an efficient review process that puts patients first can strengthen U.S. biomedical leadership and ensure that lifesaving therapies reach patients faster.

This hearing follows a November 2025 letter to FDA Commissioner Dr. Martin Makary expressing the committee’s interest in understanding how the agency is supporting innovation in rare disease drug development and increasing the speed and number of rare disease approvals. Chairman Scott remains committed to ensuring that the FDA fulfills its mission to protect patients while fostering innovation and maintaining America’s leadership in biomedical research.

Here is Help 4 HD Internationals written statement to the committee:

Witten Testimony of Katie Jackson

President and CEO, Help 4 HD International

Before the U.S. Senate Special Committee on Aging

Hearing: "From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation"

February 26, 2026

Chairman Scott, Ranking Member Casey, and distinguished members of the Committee, thank you for the opportunity to submit my testimony. My name is Katie Jackson. I am the President and CEO of Help 4 HD International, a nonprofit organization serving the Huntington’s disease community and I am an HD family member.

My late husband was diagnosed with Huntington's Disease at 24 years old. After a long, 14-year battle, he passed away six years ago. I have children who are at risk. For nearly 20 years, I have been an advocate for the Huntington's Disease community — and what I have learned from thousands of families across this nation is that for us, time is both precious and a source of constant fear.

I am writing today because the Huntington's Disease community feels let down by the very agency that is supposed to protect us.

The Unmet Need: A Disease Without Hope

Huntington's Disease is a fatal, genetic neurological illness that causes the progressive breakdown of nerve cells in the brain. It destroys a person physically, cognitively, and psychiatrically — robbing them of every ability until death. There is no cure. There is no disease-modifying therapy. There is nothing approved that slows, stops, or reverses the progression of this disease.

Let me say that plainly for the record: in the year 2026, there is not a single FDA-approved treatment that changes the course of Huntington's Disease. Not one.

HD does not just take away everything from the person who is living with HD, it does the whole family. It is a family disease. Every child of an affected parent has a 50 percent chance of inheriting it. I have spoken to mothers who are gene-positive, caring for a dying parent, while wondering when their own symptoms will begin — and whether their children will face the same fate. I have spoken to young adults who serve as 24-hour caregivers for a parent, knowing the disease may hit them even younger. We see generational trauma in a way that is hard to comprehend. Sometimes, their own symptoms begin almost as soon as their loved one's battle ends.

When we surveyed our community and asked, "what is the number one thing you would like to see in drug development?" the answer was overwhelming and unified: something — anything — in their lifetime that will slow down the progression of HD.

You might ask, "what amount of slowing progression is meaningful?" The answer is: any amount of time. Our lives are defined by moments. A slowing of progression means people can work longer, drive longer, share more holidays with their families. It means more time before the person living with HD feels like they are a burden on the ones they love the most.

The Urgency: A Community Running Out of Time

The urgency of this moment cannot be overstated. For years, the HD community has had no viable treatment options — until now. The gene therapy AMT-130, developed by uniQure, has shown significant promise in slowing disease progression. The FDA itself acknowledged this potential by granting AMT-130 both Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy designations — designations reserved for drugs that may demonstrate substantial improvement over available therapy for serious conditions.

The FDA and uniQure had previously agreed on an accelerated approval pathway. This was not a casual suggestion. It was a framework built on collaboration, regulatory science, and precedent. Accelerated approval exists specifically for devastating diseases like HD — diseases that are fatal, irreversible, and have no alternatives.

Yet the FDA reversed course.

To give this Committee a sense of how our community responded, We started a petition alongside four sister HD organizations — Help 4 HD, the Huntington's Disease Society of America, HD Reach, the Huntington's Disease Foundation, and the Huntington's Disease Youth Organization. As of today, that petition has over 40,000 verified signatures and nearly 200 supporter voices. We analyzed the sentiment and common themes of the comments. Eighty-one percent expressed urgency and the need for regulatory flexibility.

I want to share some of their words directly, because these are the people whose lives hang in the balance:

"A delay of a few years is literally the difference between life and death for me and others. Knowing there are drugs like AMT-130 that have gone through two trial phases and shown slowing of progression and are safe — and then not following through with accelerated approvals — is heartbreaking. I'm watching HD slowly take my dad. Please don't be a reason for that same fate for me." — gene-positive for HD

"My husband qualifies for this treatment right now — but if the FDA delays approval, he likely won't by the time they revisit it. We have three boys that want their dad around 75% longer as this treatment can offer them." — HD caregiver

"For many of these people, another long clinical trial does not just delay access — it eliminates it completely."

"We aren't asking for a cure without evidence. We are asking you to use regulatory flexibility which you possess. Do not let 'perfect' data be the enemy of the good when time is a luxury that HD families do not have." — HD family member

"I have lived with this devastating disease for over 40 years as I've watched it slowly but surely destroy my family. First, it killed my husband and two of his three brothers, one by suicide, in 2000, 2001, and 2002. We were told there would be a cure in 10 years. Our son, my only child, tested positive in 2001... To rip away the only hope we have is unconscionable." — 24/7 caregiver

These are not abstract policy concerns. These are parents, spouses, and children who are watching the people they love die — slowly, irreversibly — while a promising therapy sits in regulatory limbo.

Do Not Move the Goalposts

Members of the Committee, I want to be very direct about what happened. The FDA agreed to an accelerated approval pathway for AMT-130. The HD community organized around that agreement. Families participated in clinical trials. They endured brain surgery, MRIs, and invasive procedures. They traveled long distances. They also contributed to one of the most robust natural history databases in all of rare disease — the Enroll-HD study, with data from over 30,000 individuals mapping the natural progression of this disease.

Then the FDA changed its position. The Agency began reconsidering its acceptance of natural history data as an external control — a scientifically valid and widely accepted method for rare diseases where using a placebo is unethical. This is not a new or untested approach. It has strong precedent. And it was previously agreed upon.

The FDA is now signaling that it may not allow a BLA submission. This may mean more clinical long trials needed.

Let me be clear about AMT 130 specifically: the science has not changed. The data has not changed. What changed was the FDA's stance. When a regulatory agency moves the goalposts after families have already organized their lives, their hope, and their participation around an agreed-upon framework, it does more than delay a drug. It breaks trust. It discourages pharmaceutical investment in rare disease. And it sends a devastating message to families who have given everything they have to support research.

As one petition signer wrote:

"Changing the rules in the middle of the game sows nothing but distrust. The US government should honor its agreements." — HD community member

"The FDA's current reluctance isn't about saving people — because those of us who have HD are already dying. I urge the FDA to follow through on its previous commitments and give people the chance to fight for their lives." — gene-positive for HD

Our Ask

The Huntington's Disease community is not asking for corners to be cut. We are not asking for approval without evidence. We understand that accelerated approval comes with continued data collection and responsibility — and we accept that, because the alternative is having no chance at all.

We are asking for three things:

First, honor previous guidance. The FDA should fully uphold the previously agreed-upon accelerated approval pathway for AMT-130 and any future therapies that are granted accelerated approval, including the use of external control data from natural history studies. Agreements matter. Consistency matters. Predictability in the regulatory process is essential — not just for HD, but for every rare disease community watching this unfold.

Second, recognize the urgency. Huntington's Disease is fatal, irreversible, and has zero disease-modifying therapies.

Third, expedite review. Make the review and approval of AMT-130, and any future HD therapies, a top priority. Every day of delay is a day that someone loses function they will never get back. Every month of delay is a month where a patient may progress past the point of eligibility. For a community that has waited decades with no options, the clock is not metaphorical. It is real.

Closing

I want to close with this. Over 40,000 people signed our petition. Families from across the United States raised their voices because this is the first real hope the HD community has ever had. Many of them may not have time to submit their testimony because they are home caring for a loved one who can no longer speak, walk, or feed themselves.

One of our petition signers, a young woman wrote:

"Everyone who did not pass this through that sits on the FDA board, I want you to sit on the fact that your delays not only taking my future away — you are senselessly killing me, and you are killing others like me. Real human beings, not stats on your board."

Senators, I am asking you to hold the FDA accountable. Do not allow procedural hesitation to become a death sentence. The HD community has done its part. We have participated in research, contributed data, and organized with an urgency that reflects the stakes of our lives. We are asking the FDA — and now we are asking you — to do the same.

Thank you.

Katie Jackson President/CEO, Help 4 HD International www.help4hd.org

You can also submit your statement at: RareDiseaseStatements@Aging.Senate.Gov