Abstract

FDA Patient Listening Session: Using Multimedia Presentations to Share What it Feels Like to Live With LBSL 

Melody A. Kisor,*  Elizabeth F. McGinn,* Britt K. Faulkner,  Sarah L. Goone,# and Grace E. Hanson#
Affiliations: * Cure LBSL (Formerly “A Cure for Ellie”)  #Ink Factory Studios 

Introduction 

To share the critical patient voice with FDA, Cure LBSL hosted a Patient Listening Session on May 30, 2023.  As these small, private meetings with the FDA are only 90 minutes long, it can be difficult to fully convey the wide spectrum of lived patient experiences.  We wanted to create impactful and memorable presentations to share what it is truly like to live with LBSL.   

Materials & Methods 

All patients, caregivers, and families from the global LBSL community were invited to contribute their voice to this Patient Listening Session.  Information about participation went out via social media, the LBSL RareConnect community, the Cure LBSL website, and by direct invitations to individuals participating in the International LBSL Contact Registry.  Participants could share their pictures, videos, and stories via the method of their choice (email, texting, social media, surveys, community meetings, and during private interviews with the Director of Patient Engagement).  Select LBSL patients/caregivers (representing the full spectrum of the condition) were also invited to be speakers during the Patient Listening Session.   

Cure LBSL staff curated the submissions and included the content in one of three projects: video compilation, poster illustration, or speaker preparation.  We worked with a storytelling professional to transform small video clips of daily life with LBSL into one impactful video compilation, set to music and with poignant captions.  We also worked with illustrators to create a “building illustration” PowerPoint, with each slide sequentially adding additional content to a final poster.  Finally, we worked with each patient/caregiver speaker to create short presentations that incorporated pictures, videos, music, and other personal elements to most effectively tell their story. 

Results 

During the ninety-minute Patient Listening Session, the audience was able to learn about the full spectrum of experiences of living with LBSL.  

An unexpected result was the discovery of significant sleep disturbances across the majority of patients who participated in interviews. This represents a large burden to many patients and families, but was previously undocumented in the literature.   

Discussion 

During the LBSL Patient Listening Session, we were able to create the most complete representation of this complex, ultra-rare, neurodegenerative disease ever presented.  By inviting everyone, allowing families to share their choice of materials, and scheduling planning meetings and interviews to family convenience, we were able to get wide international participation.   

As a direct result of the insights discovered during the process of preparing for the Patient Listening Session, the clinical and research landscape has already changed.  Sleep questions will now be incorporated into clinical guidelines, natural history study protocols, and future quality of life surveys.  Cure LBSL is in the process of engaging sleep experts for future research.   

Using multimedia presentations kept the meeting interesting, appealed to different learning styles, and allowed visualization of experiences that could not otherwise be adequately conveyed.   These powerful presentations helped to highlight the magnitude of the effects of LBSL on the whole person and on families.