EL-PFDD

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EL-PFDD Meeting On-Demand

Submit a Written Comment

You have until December 15th to submit a comment below on the topics that we touched on in the meeting and topics that we didn’t get to talk about. Comments should be submitted individually.

Of all the symptoms of Hypophosphatasia, which 1-3 symptoms have the most significant impact on you or your loved one’s life?

How does Hypophosphatasia 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 Hypophosphatasia?

How has your loved one’s ability to cope with the symptoms changed over time?

A  few key considerations before submitting comments:
1. The purpose of this meeting is to educate the FDA about HPP symptoms and benefits/challenges of current treatments.
2. Comments should be about personal experiences and perspectives living with HPP.
3. The FDA assesses drugs in terms of safety and efficacy. It does not have any authority or control over drug pricing. Comments about pricing or access are not appropriate for this EL-PFDD meeting. 

What is an EL-PFDD meeting? 

EL-PFDD meetings are designed to educate members of the FDA and other key stakeholders about rare diseases so that decision makers have a better understanding of these conditions, as they consider new therapeutic options as they are presented to them. Since there are several new treatment approaches in development, it is prudent that we host this meeting and continue to foster education of the spectrum of severity, including first-hand accounts of patients who are living with HPP.   

Why is it important?

An EL-PFDD meeting enables us to share with key FDA officials and other stakeholders the journey of hypophosphatasia. This includes patient experience living with the disease including symptoms and current treatments used to help manage the condition. The goal is to give patients a platform to describe their experiences and to explain any gaps in the current standard of care. It is important for all stakeholders to understand how patients are impacted by HPP, the current treatment landscape and where there is room for improvement. One of the main goals of the meeting is to systematically obtain patient perspectives on HPP, which are critical to provide context when the FDA makes regulatory decisions on new drugs. The EL-PFDD meeting will be recorded and can be viewed after the event for educational purposes, and its content will be packaged in a Voice of the Patient Report. To learn more about EL-PFDD meetings, click here. 

How does it work?

EL-PFDD meetings feature people living with hypophosphatasia, their caregivers and patient advocates who directly share their stories. Panelists discuss topics such as disease symptoms that are most important to them, the impact of a disease on their daily lives (and quality of life) and their experiences with current treatments. A moderator facilitates a guided discussion through sharing of patient videos, audience polling questions, written submissions and live callers who all can engage in discussion. 

FAQ’s

Q: What happens after the meeting?
A: Voice of the Patient Report will be published and shared with the FDA and other key stakeholders. The report will also be publicly posted on the Soft Bones website.

Q: Will the meeting be recorded?
A: The Soft Bones EL-PFDD meeting will be available after the meeting ends on the Soft Bones website and YouTube channel.

 

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