Externally-Led Patient-Focused Drug Development Meeting for STXBP1-related Disorders
On October 20, 2023, the STXBP1 Foundation brought the STXBP1 patient community together for a first ever Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD), to inform the FDA and other stakeholders about the patient perspective of living with STXBP1-related disorders. We achieved record attendance with 287 unique attendees!
Thank you to the STXBP1 patient community for your participation in making this a successful meeting!
Many thanks to the patient panelists, people who called in with live comments, and individuals who submitted email comments. During the meeting, parents, caregivers and family members shared the realities of living with STXBP1, highlighting the profound impact on children and families living with STXBP1-related disorder and hopes for future treatments. This information was included in the Voice of the Patient report.
Externally led patient-focused drug development (EL-PFDD) meetings bring together patients and caregivers, US Food and Drug Administration (FDA) representatives, pharmaceutical companies, doctors who are experts in the particular disease, and other stakeholders. For our meeting on STXBP1-related disorders, our goal was to hear from patients on what it's like to live with the condition to better understand and document the patient experience. This information can help the FDA to make informed decisions on approvals of potential medicines for STXBP1-related disorders, and pharmaceutical companies to design clinical trials that are meaningful for patients.
This highly successful event not only demonstrated the community's motivation but also educated both the FDA and industry about the arduous journey of STXBP1 families and the pressing need for effective treatments.
Voice of the Patient Report
The "Voice of the Patient" Report summarizes the Externally Led Patient-Focused Drug Development (EL-PFDD) meeting for STXBP1-related disorders. This document is an extensive summary of the patient community comments and feedback shared at the meeting. This will be a reference for future decisions about potential medicines for STXBP1-related disorders. This report is being shared with the FDA and published on our website. Future applications for therapy approvals will use the report as a reference when evaluating the effectiveness of the treatment.
Download the Voice of the Patient Report.
Who Benefits from EL-PFDD Meetings?
FOOD AND DRUG ADMINISTRATION (FDA)
The FDA gains an understanding of what it's like to live with a particular disease.
The FDA becomes informed of side effects and risks patients may be willing to accept to gain a certain level of symptom relief or slowing of their disease progression. The FDA learns about patients' needs regarding new drugs, and what their preferences are for clinical trials for their disease.
EL-PFDD meetings assist the FDA in knowing if a new drug addresses patient needs.
PATIENTS
Patients know the FDA and drug sponsors have heard their voices.
Patients' experiences are validated, reducing feelings of isolation. Hearing other patients voice their experiences and needs helps patients to better self-advocate.
EL-PFDD meetings can help to bring new treatments to the market, which benefits patients.
PATIENT ADVOCACY GROUPS
EL-PFDD Meetings help these groups identify what needs exist for patient education and advocacy.
More effective advocacy increases public awareness and knowledge of the disease.
In addition, these meetings help patient advocacy groups connect patients with their peers.
PHARMACEUTICAL COMPANIES
Drug sponsors gain insights into the major concerns of patients. This helps the companies develop treatments and design clinical trials that match patients' needs and preferences.
Drug sponsors learn which disease symptoms or treatment side effects are, or are not, tolerable by the patients. This helps the companies develop drugs that matter to patients.
With the knowledge gained from EL-PFDD Meetings, pharmaceutical companies receive advice from the FDA on developing potential drugs and therefore help to advance medicines that meet patients' needs.
