Externally Led - Patient Driven Drug Development

 (EL-PFDD)


Patient lived experience will convey the importance and urgency of new therapies to the Food & Drug Administration (FDA), researchers, pharma, healthcare providers, and federal regulators, leveraging the unique power of patient experience.


This meeting is hosted by the Hypertrophic Cardiomyopathy Association in partnership with the Danon Foundation, FARA, the DCM Foundation, SADS Foundation, and ARC. It’s an important opportunity to help inform how the FDA considers treatments for genetic cardiomyopathies. 


Do you want to talk to us live during the discussion sessions?
Please call 1 703-844-3231 when prompted.
*Due to time restrictions, we may not be able to get to all phone calls.


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Discussion Questions - Share Your Experiences

Topic 1: Genetic Cardiomyopathies Symptoms & Daily Impacts

  1. Of all the symptoms and health effects of Genetic Cardiomyopathy, which symptoms have the most significant impact on you or your loved one’s life?
  2. How does Genetic Cardiomyopathy affect you or your loved one on your best and worst days? Describe your or your loved one’s best days and worst days.
  3. How have you or your loved one’s symptoms changed over time? How has the ability to cope with the symptoms changed over time?
  4. Are there specific activities that are important to you or the loved one that you/they cannot do at all or as fully because of Genetic Cardiomyopathy?
  5. What do you fear the most as you or your loved one gets older? What worries you most about your or your loved one’s condition?


Topic 2: Perspective on Current and Future Approaches to Treatment

  1. What are you currently doing to manage your or your loved one’s Genetic Cardiomyopathy symptoms?
  2. How effectively do these treatments address the most significant symptoms and health effects of your or your loved one’s Genetic Cardiomyopathy?
  3. What are the most significant downsides to your or your loved one’s current treatments, and how do they affect daily life? (Examples may include bothersome side effects, hospital visits, etc.)
  4. Short of a complete cure, what specific things would you look for in a new treatment for Genetic Cardiomyopathy? What factors would be important in deciding whether to participate in a new research trial?


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