Huntington’s Disease Community Perspectives on Desired Characteristics of Disease Modifying Therapies

Background: Promising disease modifying therapies for Huntington’s disease are now entering pivotal trials, raising questions of what patients and families consider successful outcomes. Consistent with an ongoing movement to incorporate patient preferences into the development of new therapies, we conducted a pilot study to assess Huntington’s disease community views on emerging DMTs to assist in planning large-scale studies of patient preferences.

Methods: Semi-structured interviews were conducted with members of the Huntington’s community (manifest disease, at-risk, and family/caregivers). Participants were asked which symptoms they believed should be targeted with novel treatments, as well as potential benefits and tradeoffs of delaying symptom onset versus prolonging late-stage disease.

Results: Participants (N = 14) emphasized the need for treatments improving cognitive and/or behavioral symptoms. Many wanted treatments that delayed symptom onset up to 5–10 years, though some considered shorter delays acceptable due to potential value in advancing research to help future generations. Concern regarding potential for prolonging later-stage disease was variable, with some participants uncertain if they would want a treatment that delayed onset but prolonged later-stage disease. Others stated that any delay in onset would be desirable, regardless of potential prolongation of later stage disease.

Discussion: This study demonstrates a breadth of opinions among the Huntington’s disease community surrounding both the benefits and complex tradeoffs that might occur with disease modifying treatments. These preliminary findings will inform future large-scale studies of attitudes toward disease modifying treatments, which may ultimately guide the design and outcome measure selection for clinical trials.

Highlights: In-depth interviews with the Huntington’s disease community were used to explore patient and family preferences regarding potential disease modifying therapies. Many wanted symptom delay of 5–10 years, though some considered shorter delays acceptable for altruistic reasons. Opinions on trade-offs varied, suggesting larger preference studies are needed to inform trial design.