Registration is now open for the SMA Patient-Focused Drug Development (PFDD) meeting with the FDA. The PFDD meeting will be held at the College Park Marriott, near Washinton DC, on Tuesday, April 18.
There are two primary ways to participate in this meeting:
Attend in person. Registration for in-person attendance is free, but seating is limited and will be on a first-come, first-served basis. Please note that, while registration is free, you will be responsible for your own travel and hotel accommodations.
View the webcast. A live-streaming webcast of the meeting will be available for those who are not able to travel to the meeting. The webcast is free, but you must register in advance.
Registration for in-person attendees will close on Friday, March 31. If capacity is reached before then, a waiting list will be opened up. We strongly encourage in-person attendees to register well in advance in order to secure a spot.
Registration for the webcast will close on Friday, April 14.
We thank our industry and advocacy partners who have helped make this meeting possible. Please visit our partners page to see a complete list.
More About the Meeting
Through this Patient-Focused Drug Development (PFDD) meeting, the FDA provides a forum where SMA patients, families and caregivers are invited to share their unique insight on the impact of SMA on their day-to-day lives. The FDA is also interested in gathering the SMA patients’ perspective on available treatment options and how well these may help to treat symptoms, strategies for managing their SMA, and expectations for approved treatments for SMA, among other important topics.
At this meeting, several different groups of panelists, representing all types, ages and stages of SMA, will bring their voices and stories to depict the real and specific ways in which their lives are impacted by SMA. Each round of panelists will be followed by polling questions and a period of facilitated discussion. Both in-person and webcast attendees will have an opportunity to submit comments during the discussion periods.
Prior to the meeting, all registrants will receive a discussion guide explaining in more detail the topics that will be covered at the meeting. Registrants can use this discussion guide to help shape their comments.
If you have any questions on the PFDD meeting, please email [email protected].