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HD InsightsFeatured Story: Conversations on Pre-HD

Conversations on Pre-HD

FDA Patient Listening Session Executed by Huntington’s Disease Community Advocates


B.J. Viau is the Director of Patient Advocacy at Horizon Therapeutics and is a lifelong Huntington’s disease advocate. He is co-founder of the HD Youth Organization, an international non-profit helping young people impacted by HD. B.J. has served as its board chairman for over 10 years.

Fellow HD patient advocate Seth Rotberg and I have weekly conversations about the HD community and how we can find ways to provide positive impact. A couple of years ago Seth, who is gene positive, pre-symptoms, was doing a bit of a speaking tour on podcasts and panels, raising awareness for HD. He would always send me the links for listening and ask for constructive feedback. One of the common themes I kept hearing Seth say was, “I know I will get HD one day, but it’s OK and I am living a good life right now.” Although this was a true statement, I suggested some alternative ways to say this in order to create more urgency with potential listeners to join the fight.

One of the small tweaks we made for his future speaking engagements was “I know I will get symptoms of HD one day, unless something is done about it.” A small change to say that the inevitable doesn’t have to be true as new treatments could save his life, and we need people’s help to accelerate this option!


Accelerating HD Treatments

As two non-scientists, we then started to ask ourselves what we could do to accelerate treatments to help Seth and our many friends who are also pre-symptomatic or who are still at risk. We started having many conversations with stakeholders that included clinicians, pharmaceutical partners, advocacy organizations and others. Although we weren’t going to create a new treatment in a lab ourselves, we did identify the opportunity to use our voices and community connections to accelerate the future potential treatments in a few ways.

The FDA developed a handful of patient-focused tools a few years ago in hopes of better understanding the needs of patients afflicted with any serious illness—for them and their surrounding families. The Huntington’s disease community was fortunate enough to get a patient-focused drug development meeting back in 2015 to discuss the impact of the disease on the patient and the caregiver.

Although this meeting was important, after watching the recording, we realized that the attendees and speakers represented symptomatic patients, but not those who are gene-positive but pre-symptomatic—individuals who didn’t have symptoms today but knew they would eventually get them. We sought advice from a few patient advocates affected by other rare diseases, and they suggested a different tool called an FDA patient listening session. Essentially, a listening session is an opportunity to share personal stories directly with FDA representatives to help them better understand a condition.

Seth and I drafted a rough agenda and followed the guidelines provided on the FDA website to submit our proposal back in November 2021. Our specific aim was to provide stories that share what it’s like to live knowing you will one day get HD, but having yet to be clinically diagnosed with any symptoms.

Our request was accepted with one condition set by the FDA: that we provide insight regarding risks this population is willing to take when considering joining a clinical trial or taking a newly approved medicine, and the types of benefits they would be looking to receive.

On July 25, 2022, we held the virtual patient listening session with the FDA, which included the stories for seven patient advocates and results from a community survey around risk/benefits and general feelings towards clinical trials. There were 50 individuals from the FDA who attended the session. Additional attendees from HSG, HDSA, CHDI, HDYO, Help4HD and Optio Bio attended the call as observers.

The seven advocates shared their personal stories and how HD has impacted their lives to date, even though they don’t have symptoms. They shared their hopes for future treatments and what the opportunity to participate in clinical trials before symptoms occur would mean to them and others impacted by their lives. A full recap of the event can be found on the HDYO website: https://en.hdyo.org.


The IRB-approved community survey we had created was meant to capture more data to better understand the impact HD has on patients before being clinically diagnosed. Within one month, we were able to get 164 qualified responses, where we learned:

  • 73% of respondents visited a clinician less than once a year.
  • 76% expressed a level of willingness to participate in
    pre-symptomatic clinical trials.
  • Using a hypothetical thought experiment, individuals were willing to accept a mortality risk between 30-42% for a gene therapy that would slow symptoms by 90%.

A poster of the data will be shared at the 2022 Huntington Study Group Annual Meeting in Tampa, Florida.

At the end of the call, the FDA’s Director of Neuroscience, Dr. Billy Dunn, spoke on behalf of the FDA and thanked everyone for sharing their stories. He shared the FDA’s commitment to accelerating trials for individuals with HD, and the ultimate goal of approving a treatment for individuals before they ever deal with symptoms. It meant a lot to hear this directly from Dr. Dunn, but we also understand there is a lot of work we need to do to get to that point.

Our main takeaways from the FDA patient listening session and the community survey are that a lot more needs to be done to attend to the needs of the pre-symptomatic patient community. We believe the survey we created was just a conversation-starter, but a more robust and professionally created survey needs to be executed next. We want to continue to partner with clinicians, pharmaceutical companies, advocacy organizations and other stakeholders to not just learn more from the pre-symptomatic community, but also to make them aware that their opinions are needed, now!

We believe the FDA is open to discussing more robust data from the pre-symptomatic HD community. A discussion around the data could lead to official guidance for pharmaceutical/biotech companies to follow when creating new treatment programs for patients who aren’t yet symptomatic. More data could also lead to the FDA better understanding what appetite for risk individuals have, even before they have diagnosed symptoms.



  • Accelerate knowledge of biomarker data by raising awareness about observational study opportunities like PREVENT-HD and ENROLL-HD to a younger population that isn’t yet aware of these.
  • Work with pharmaceutical companies to help them better understand the needs of younger people when they are creating clinical trial protocols, inclusion and exclusion criteria, and recruitment strategies.
  • Identify gaps in clinical trial awareness and education to individuals who don’t regularly go to a clinician for care and/or stay current with all the HD clinical trial updates.
  • Find opportunities to influence and educate the FDA about pre-symptomatic individuals (both gene positive and at-risk) to help them better prepare for future clinical trials programs in this population. 


We’d really appreciate collaboration of all types and welcome anyone’s input if you have an opinion or expertise in any of the below:

  • Writing risk/benefit market research questions
  • Background working with the FDA
  • Passionate about accelerating trials and treatments for pre-symptomatic individuals





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