Frequently Asked Questions about the Symposium
What is patient focused drug development? (PFDD)
It's a newer way of thinking about how to optimize patient care and treatment by providing patients the opportunity to be involved in the FDA's decision making process.
What is a PFDD meeting?
A Patient Focused Drug Development meeting is a way for the FDA to hear from patients directly about what matters to them.
An Externally-Led Patient Focused Drug Development meeting, like our Symposium, just means that the FDA is not managing the meeting themselves, but they will have representatives in attendance.
But we have Crysvita now. Why do we need more options?
Crysvita is a huge step forward for the treatment of XLH. But there is no reason to stop there. Exploring additional options for treatment, especially for other hypophophatemias, will work towards optimizing the quality of care for other patients.
How will the information from the survey and meeting be used?
The XLH Network, Inc. will develop a report summarizing the information from the meeting as well as the pre-event survey. The report will focus on the impact of hypophosphatemia on adults and what treatment endpoints matter most to patients. This report, along with a video of the Symposium, will be available to the public via our website and social media outlets.
This sounds important. How can I help?