Patient advocacy plays a key role in bringing about systemic change in the development and approval of medical products. Ideally, patient engagement involves more than participation in clinical trials. It means active participation in providing input into patient-identified preferred treatment outcomes and risk benefit tolerance for new therapies. This strategy places the patient community in partnership with the pharmaceutical industry and regulators who make decisions that affect access to new therapies.HDSA has a major initiative to collect input from the HD community about the impact of living with HD and preferred treatment outcomes to inform regulatory decision-makers and medical product developers about the unmet needs of the community. Read further to learn about this initiative and view webinars, read reports or listen to podcasts.
HDSA HD Symptoms and Treatment Impact Survey
The first step in collecting input from the HD community was to develop the HDSA HD Symptoms and Treatment Impact Survey. A HDSA Family Council was convened to provide input on the survey questions and responses. View a webinar that highlights the survey results here.
HDSA EL-PFDD Meeting
On November 13, 2024, over 146 members of the HD community participated in an HDSA EL-PFDD Meeting for Pre-symptomatic and Early to Mid-Stage Adult-Onset HD. During this full-day hybrid meeting, participants shared their stories about the impact of living with HD and their approaches to treatment. Twenty-one FDA staff members were in attendance to listen and learn. Also in attendance were staff from pharmaceutical companies actively engaged in research to treat HD. This input can inform FDA’s decisions and oversight both during drug development and during the review and approval process for new therapies.
The HD community participated in facilitated conversations by providing responses to questions in person or online. You can view a recording of the entire meeting and hear the comments using these links: Part 1, Part 2. View the slide deck from the meeting here.
HDSA Podcast: Let's Talk about the HDSA HD EL-PFDD Meeting
Neekia Davis and Phyllis Foxworth dive deep into the highlights and critical discussions from the November 13, 2024 Externally-led Patient Focused Drug Development (EL-PFDD) meeting. They break down the key takeaways and provide an inside look at the collaborative efforts shaping the next steps. Listen here.
Next Steps
The participants’ comments will be consolidated into a Voice of the Patient Report. This report will be submitted to the FDA for posting on the agency’s website providing medical product developers, researchers and clinicians access to the HD community’s perspectives. The voice of people living with HD and their families will also be amplified by submitting the consolidated community input to academic journals for publishing and presented at HD conferences.