Externally Led Patient-Focused Drug Development (EL-PFDD) Meeting on Fabry Disease
Submit Comments After the EL-PFDD Meeting on Fabry Disease
Comments will be accepted up to October 20, 2022 and will be incorporated into the Voice of the Patient report.
- Of all the symptoms of Fabry disease, which 1-3 symptoms have the most significant impact on you or your loved one’s life?
- How does Fabry disease affect you or your loved one on best and on worst days? Describe your best days and your worst days.
- Are there specific activities that are important that you or your loved one cannot do at all or as fully as you would like because of Fabry disease?
- How has your loved one’s ability to cope with the symptoms changed over time?
- What information would you want to know to inform your decision on enrolling in a clinical trial Fabry disease?
- In which type of trial would you be more likely to enroll – a trial for a drug designed to:
a. Benefit the kidney alone but not slow or stop the overall disease progressionb. Benefit the heart alone but not slow or stop the overall disease progressionc. Reduce symptoms (e.g., pain, GI involvement, fatigue) alone but not slow or stop the overall disease progression
- For parents of pediatric patients: are you interested in enrolling your child in a clinical trial? If so, what factors would you consider in deciding whether to participate (e.g., age, whether symptoms have begun, whether already on ERT)? If not interested, why not?
- What are you currently doing to help treat your condition or its symptoms?
- How well does your current treatment regimen treat the most significant symptoms of your disease?
- What are the most significant downsides to your current treatments and how do they affect your daily life?
- Assuming there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition?
- You'll be able to submit comments on your experience with Fabry disease before the meeting
- The link to join the meeting will be available on the day of the meeting (September 19th)
Take the patient survey
Who benefits from EL-PFDD meetings?
Food and Drug Administration (FDA)
Patient advocacy groups
Hear from Previous Patient and FDA Participants
I can say with certainty…that the stories that you shared with us today, your experiences with the disease will certainly impact how we at FDA will think about this disease, will approach our discussions with sponsors moving forward, and will really shape the development in the future of therapies for this disease.
Being able to tell my story, be heard, and people empathizing and understanding the struggles this disease faces you with is indescribable.
I think that your stories really highlight the urgency with which we need to find effective treatments for this disease, and also treatments that have better safety profiles than those that are currently out there.
I liked that everyone was invited to speak freely and participate. It was an excellent opportunity to tell our stories.
If I take the Patient Survey, what will you do with the information I provide?
Is there a charge to attend the meeting?
How do I register to attend the EL-PFDD meeting?
When and for how long is the EL-PFDD Meeting on Fabry disease?
Is this EL-PFDD Meeting on Fabry disease for pediatric patients?
What happens after the meeting?
The National Kidney Foundation (NKF) is the oldest and largest non-profit health organization dedicated to preventing kidney diseases and improving the health and well-being of individuals and families affected by kidney disease. Education of patients and healthcare providers is a big part of the NKF’s work, and we support kidney patients by providing all sorts of educational materials, programs, and patient services.
The mission of the Fabry Support & Information Group (FSIG) is to raise awareness of Fabry disease and its symptoms, to advocate for community needs and to lead the fight for the most effective treatment possible and eventually a cure. FSIG provides a link for the Fabry community to information, each other, the medical community, researchers, the pharmaceutical industry, and regulatory authorities. FSIG strives to support affected families and individuals lead fuller lives.