The new year is in full swing, and I’m excited for what 2023 has in store for the Huntington’s disease (HD) community.
Looking back, it feels like the past three years haven’t gone in the right direction. We still lack disease-modifying therapies, legislative support for the HD Parity Act, and a lot of scientific knowledge about the disease to support clinical trials for people who don’t yet have symptoms.
Regardless of what we don’t have, we will have a lot of steps in the right direction that are coming this calendar year. From new pharmaceutical updates to ongoing advocacy for this community, my columns will continue to focus on what I — and you — can do to make a difference.
Last July, I co-hosted a patient listening session with the US Food and Drug Administration (FDA). We discussed the importance of clinical trials becoming available to people who know they have the HD gene mutation but don’t qualify for clinical studies because their symptoms haven’t progressed enough.
We conducted a community survey of over 150 respondents. We learned that people who are presymptomatic not only want to participate in clinical trials, but they are also willing to take on a large risk to potentially eliminate or prolong the onset of HD symptoms.
If this subset of patients were to qualify for a clinical study, however, we discovered a handful of roadblocks they would encounter that might deter them from participating. These included the need for paid time off at work, the cost of taking time away from work and the home, and the need for good information about a trial, as many of these patients aren’t regularly seeing an HD doctor.
The patient listening session was a nice exercise, but looking back, it feels a little like an empty experience. We had hoped it would serve as a springboard to prompt conversations with stakeholders across the patient community, as well as with advocacy organizations, pharmaceutical companies, clinical practitioners, and regulators. But that hasn’t seemed to be the case.
So we have a new year with new energy to figure out how to move forward and build momentum that leads to change. Our ultimate hope is for the FDA to provide clear guidance to the pharmaceutical industry regarding clinical endpoints for people who don’t yet have symptoms. We believe that more guidance from the FDA would prompt pharmaceutical companies to open trials for people affected by HD, earlier in their journey.
The path to achieving this goal isn’t clear yet, but I, along with a handful of advocates, look forward to convening with key HD community stakeholders early in 2023 to brainstorm ways to move forward. We welcome any ideas about how to make it happen.
Notes: Huntington’s Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Huntington’s Disease News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Huntington’s disease.