Externally-Led Patient-Focused Drug Development Meeting & The Voice of the Patient Report

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. 

In case you missed the EL-PFDD meeting, you can watch it below!

Voice of the Patient Report

This report summarizes the Externally- Led Patient-Focused Drug Development (EL-PFDD) meeting that we held on November 15th, 2022. The EL- PFDD meeting was designed to communicate to FDA the impacts of hypophosphatasia on individuals daily lives, what types of treatment benefits make the most impact on peoples lives, and individuals and caregivers perspectives on how well available therapies are working.

This landmark report includes written statements and transcripts which are summarized in the report and shared in full in the appendices. 

This report is dedicated to the courageous patients, caregivers, and families impacted by HPP. 

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To download the report click below.

softbones icons_grey_soft bones publication copy

A summary of the Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD) on hypophosphatasia.

Public meeting: November 15, 2022  Report submitted: April 21, 2023
Hosted by: Soft Bones, Inc., The U.S. Hypophosphatasia Foundation

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