XLRP-Patient Focused Drug Development
Webinar for the X-Linked Retinitis Pigmentosa (XLRP) Community
The Foundation Fighting Blindness recently held a webinar to provide details about an upcoming Externally-Led Patient Focused Drug Development (EL-PFDD) meeting for XLRP, and to provide background information about the Food and Drug Administration’s (FDA) drug review process.
The EL-PFDD meeting was held on June 7th, 2022. The goal of the June 7th meeting was to educate FDA staff and other key stakeholders such as biotech and pharmaceutical companies about what it is like to live with XLRP, with perspectives from both affected individuals as well as caregivers and family members of affected individuals. The FDA staff may use this information when reviewing new clinical trials or new drug applications for XLRP.
EL-PFDD meetings are unique among public meetings with the FDA and other stakeholders, with a format designed to engage patients and elicit their perspectives on two topic areas:
The most significant symptoms of their condition and the impact of the condition on daily life; and
Current approaches to management or treatment.
Below is a recording from the recent webinar.
Recording from the June 7th EL-PFDD meeting.
Voice of the Patient Report
The Foundation Fighting Blindness first and foremost wishes to thank all those living with X-linked retinitis pigmentosa and their loved ones and caregivers who attended the June 7, 2022 meeting. Thank you for sharing your experiences and insights about living with XLRP. Thank you also to those who contributed by phoning in or contributing their insights online. We dedicate this report to all of you.