Creating unbiased and equitable access to the HD community to expedite key questions for research.

Date: Sunday, 15 September
Time: 8 a.m. – 12 p.m.
Location: AC Marriott, Strasbourg, France

Email at jenna@hdyo.org for more information!

As clinical research and trials are progressing with the aim of either preventing the development of HD or treating HD as early in life as possible, there is a growing need for industry partners, academic researchers, and regulators to rely on the advocacy communities to understand patients’ and families’ perspectives around these novel therapies and treatment paradigms. This is especially critical for understanding the different risks communities are willing to endure based upon the benefits possible with emerging treatments. In depth feedback cannot be collected in a simple format like a survey because there is a need to measure the community’s understanding around these complex topics first.

A round table format supports the need to engage the community in a meaningful conversation around broad risk-benefit considerations, which may be highly variable between individuals. In addition, HD is a generational disease with many serving as caregivers and future patients; we need to further understand how they would consider the risk-benefit balance both for themselves as well as for their loved ones. The community’s feedback on these general risk-benefit questions is essential to advancing therapeutic treatments into clinically asymptomatic patient populations. This forum is intended to engage as many partners as possible at one time on thematic considerations to help industry and research partners further develop studies, trials and advocacy efforts. Follow-up discussions may then be held to focus on specific development programs or therapies.

  • Understand general risk/benefit topics for the global HD community based on how they identify their connection to HD.
  • Collaborate across industry and advocacy partners to answer key questions.
  • Understand if there is a strategic way to disseminate a larger-scale study to gather even more data around these topics across the global community.
  • Decrease roadblocks to get treatments approved even if a trial that shows benefit doesn’t meet all clinical endpoints.
  • Understand the perspectives of young people who have been underrepresented.

Pre-Meeting:

  • Participating partners can submit their questions to HD-CAB
  • HD-CAB will structure the meeting around these questions and enlist specialists to help with any educational components needed for advocates.
  • Sample questions may include topics around willingness to participate with varying levels of Nfl, feelings around placebos, medical procedures, symptom treating vs disease modification, etc.
  • HD-CAB will generalize questions to be as encompassing as possible for multiple interests.

During Meeting:

  • Event with 10-15 advocates present to share their perspectives. This group will represent different countries and identities in the HD community.
  • Industry and research professionals who have been approved can attend the meeting. Their role is to listen to the advocates, but there won’t be an opportunity for questions on the spot from the audience. However, there will be a feedback portal that will go to the HD-CAB team where questions can be asked, and we may work them into the conversation.

Post-Meeting:

  • Detailed report and analysis will be distributed among partners who attended.
  • Specific follow up per company available with HD-CAB Leadership.
  • Future advisory boards offered to address any trial-specific questions and needs.

Pharmaceutical Companies – $5,000

  • 2 staff members’ attendance.
  • Ability to ask questions ahead of the discussion and request certain community member profiles.
  • Early Access to post-event analysis and follow up meeting to discuss analysis to plan future advisory boards.

Researcher, Regulators or Additional Industry Participants – $250

  • Access to attend the meeting.
  • Access to post-event analysis.

Advocacy Association, HD Community Members and Young Investigators – $50

  • Access to attend the meeting.
  • Access to post-event analysis.

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