NEWS

We are happy that after hosting the Externally Led Patient-Focused Drug Development (EL-PFDD) meeting, in July of 2022, the Voice of the Patient report has been submitted to the FDA’s Center for Drug Evaluation and Research Division of Neurology Products, Office of Neuroscience, for inclusion in the framework used to evaluate future Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD) therapies. Patients’ views carry great weight with the agency when it considers whether to approve a new therapy.

The report reflects the success of the EL-PFDD meeting that was held to educate the FDA and other stakeholders on how SSADHD has impacted their life and the life of their loved ones. Over a hundred patients, caregivers, researchers, and clinicians came together from around the world to share the impact that this ultra-rare disorder has on their daily life. Additionally, we were joined remotely by families, from across the world, whose patients were too fragile to travel and attend the meeting in-person.

In the year leading up to the meeting, we made every effort to encourage patients, caregivers
and family members, at every stage of disease progression to get involved.

SSADHD is highly underdiagnosed and has a varying phenotype, so we are grateful to the more than 130 individuals who participated in the extensive survey describing the disease burden in their affected children. Additionally, we appreciate those who participated in the small group meetings, with caregivers and patient interviews attempting to narrow in on the most significant symptoms of the disease.

Our community owes a deep debt of gratitude to the patients and family members who courageously shared their stories. This landmark Voice of the Patient report documents the severe disease burden and unmet medical need in patients’ own voices. It includes a written summary of a year’s work around the EL-PFDD meeting.

Click here to view the Voice of the Patient report.