Myotubular & Centronuclear Myopathy
Externally-Led Patient-Focused Drug Development (PFDD)
August 3rd, 2021
10:00 am EDT-3:00 pm EDT
What is an "Externally-led Patient Focused Drug Development (PFDD) Meeting"?
The FDA states, “Patient-focused drug development is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. As experts in what it is like to live with their conditions, patients are uniquely positioned to inform the understanding of the therapeutic context for drug development and evaluation”.
So, a PFDD meeting is a platform for the patient community to share the stories of how their lives are impacted by the disease and their hopes and priorities when it comes to any potential therapies.
A PFDD meeting is not specific to any drug and therapy but rather a moment to hear the patient voice on the disease impact and what meaningful improvement looks like from the patient and caregiver perspective.
("Externally-led" means that the meeting is being run by the patient community rather than being hosted by the FDA.)
Agenda (all times Eastern)
10:00 Welcome & Introductions
10:10 Opening Remarks, Representative of the FDA
10:30 Overview of X-Linked Myotubular Myopathy & Centronuclear Myopathy
11:00 Patient & Family Voice Panel Topic 1: Impact of MTM & CNM
12:00 Current Therapeutic strategies for MTM & CNM
12:30-1:00 Lunch Break
1:00 Patient & Family Voice Panel Topic 2: Meaningful Outcomes
2:00 Patient Preferences & Risk/Benefit Considerations
2:30 Meeting Summary and Closing Remarks
We hope you can join us for this important event!
This PFDD is Co-Hosted and Moderated by the following MTM-CNM Patient Advocacy Organizations:
Celebrating a decade of connections!
Photo Credits: Sergio Vazquez