NEWS

Over a hundred patients, caregivers and doctors gathered from across the US, and from as far away as Asia, Europe and Greece to participate in the Externally Led – Patient Focused Drug Development (EL-PFDD) meeting. The meeting was proceeded by a family dinner on July 7, 2022 followed by the meeting on July 8, 2022 at the Marriott Bethesda, in Bethesda, Maryland.

Additionally, we had participants join us virtually who were unable to travel because of the logistical challenges of traveling with their affected children.

The PFDD initiative was developed for the Food and Drug Administration (FDA) to systematically obtain the patient perspective on specific diseases, and currently available treatments.

At the PFDD meeting the families highlighted the most significant symptoms of SSADH Deficiency and shared stories to reflect the impact that this disorder has on the patient’s daily life while, emphasizing the lack of treatment options.

Below is a list of those who attended either virtually or in-person on behalf of the FDA to hear our perspective:
Billy Dunn – CEDR, Director of Office of Neuroscience
Cynthia Welsh – CDER Medical Officer
Eleanor Dixon-Terry – Regulatory Health
Emily Freilich – Clinical Team Lead
John Troiani – CDER Medical Officer
Julienne Vailancourt – Center for Biologics Evaluation and Research
Karen Jackler – Center for Biologics Evaluation and Research
Quyen Tran – JD at FDA
Robyn Bent – CDER, Director of Patient-Focused Drug Development
Sally Yasuda – CDER, Lead Pharmacologist
Mary Jo Salerno – Senior Regulatory Health Project Manager at FDA
William Lewallen – CDER, Patient-Focused Drug Development
Michelle Campbell – CDER, Patient-Focused Drug Development

We are happy to provide the videos below of the SSADH EL-PFDD Meeting, for those who were unable
to attend in person.

SSADH Association Welcome and Morning Session 1
EL-PFDD Meeting, Video 1 of 4.
Morning Session 2
EL-PFDD Meeting, Video 2 of 4.

Afternoon Session 1
EL-PFDD Meeting, Video 3 of 4.

Afternoon Session 2
EL-PFDD Meeting, Video 4 of 4.

The “Voice of the Patient” report will provide vital input towards regulatory decisions at the FDA for new drugs and possible treatments for SSADH.