Celeste Graham: “Cerebral Creatine Deficiency Syndromes (CCDS) Externally Led Patient Focused Drug Development (EL-PFDD) Meeting Voice of the Patient Report”
SHORT SUMMARY
A summary of highlights from the Voice of the Patient Report (VOP). The VOP is a written account of the January 2023 EL-PFDD Meeting, polls conducted, and comments submitted by those in attendance.
ABSTRACT
Externally-Led Patient Focused Drug Development (ELPFDD) meetings are a methodical way to make sure that patient and caregiver perspectives related to living with a disease are considered throughout the drug development process. In addition to patients and caregivers, these meetings are attended by regulatory agencies, industry, researchers, and clinicians. Following the ELPFDD, an extensive Voice of the Patient (VOP) report is compiled, capturing all of the data and a summary of the information shared throughout the meeting. This report is submitted to the FDA, to be used as a reference point when drugs or therapies are reviewed. The Association for Creatine Deficiencies (ACD) hosted a virtual ELPFDD meeting in January 2023 on Cerebral Creatine Deficiency Syndromes (CCDS). Following a brief introduction with remarks from the FDA and a clinical overview of CCDS, the meeting was divided into two main sessions – one focusing on living with CCDS and the other focusing on current and future approaches to treatment for CCDS. The CCDS VOP is a comprehensive summary from the meeting. It includes key insights, a wealth of data, and impactful quotes from patients and caregivers throughout. This poster aims to provide a visual summary of the CCDS VOP.