HDSA thanks the HD community for your outstanding contributions to yesterday’s FDA Patient Focused Drug Development proceeding. Whether you were able to participate in person, via webcast or just in spirit, know that the hundreds of caregivers, persons with HD, those living at risk as well as advocates representing loved ones who are no longer with us gave a strong and resounding voice to HD as meeting participants discussed their struggles to manage the wide range of symptoms affecting those with HD and JHD while also calling upon the FDA to fast track new treatment options.

While we are grateful to the community for rallying for this face to face meeting with FDA, our work is not done. FDA has opened a 60 day Open Comment period wherein you may add your voice to the discussion. To submit a comment, go to: http://bit.ly/FDA-comments and on the right side of the page you will see a box that says “SUBMIT A FORMAL COMMENT.”

You may submit more than one comment during the 60 day period beginning today and ending on November 23, 2016. The FDA will read every comment submitted – YOUR voice will be heard.

At the end of the meeting, the FDA asked the HD community to provide information on the following question: At all stages of HD (those living at risk, gene positive but no symptomatic, early, middle, late stage HD/JHD), if a treatment could be developed, what symptom would you most like to stop or what ability would you like to regain – what is most important to you?

Other questions posed by the FDA which you may also want to comment upon were:

Topic 1: Disease symptoms and daily impacts that matter most to patients

  1. Of all the symptoms that you experience because of your condition, which 1-3 symptoms have the most significant impact on your life? (Examples: ability to control movements, balance/coordination, difficulty concentrating, sleeping, mood/behavior etc.)
  2.  Are there specific activities that are important to you that you cannot do at all or as fully as you would like? (Examples: sleeping through the night, daily bathing/showering, cooking, eating, dressing, shopping etc.)
    a) How do your symptoms affect your daily life on the best days? On the worst days?
  3. How has your condition and its symptoms changed over time? a) Do your symptoms come and go? If so, do you know of anything that makes your symptoms better? Worse?
  4. How has your condition affected your social interactions, including relationships with family and friends?
  5. How has your condition affected your mood (for example; depression, apathy, patience/tolerance for frustration)?


Topic 2: Patients’ perspectives on current approaches to treating Huntington’s disease

  1. What are you currently doing to help treat your condition or its symptoms? (Examples: prescription medicines, over-the-counter products, and other therapies including non-drug therapies such as diet modification, exercise.)
    a) What specific symptoms do your treatments address?
    b) How has your treatment regimen changed over time, and why?
  2.  How well does your current treatment regimen treat the most significant symptoms of your disease?
  3.      a) How well do these treatments improve your ability to do specific activities that are important to you in your daily life?
    b) How well have these treatments worked for you as your condition has changed over time?
  4.  What are the most significant downsides to your current treatments, and how do they affect your daily life? (Examples of downsides: bothersome side effects, interacts with other medications, need to visit your doctor more frequently etc.)

5. Assuming there is no complete cure for your, what would you look for in an ideal treatment for a specific aspect of your condition?


HDSA will announce when the recorded webcast of yesterday’s proceedings is available and will provide the link as well as the link for the transcript when it becomes available. Please contact Deb Lovecky, Director of Programs and Services (dlovecky@hdsa.org) if you have any questions.