On October 28, 2024, the Foundation for Sarcoidosis Research (FSR) hosted a groundbreaking Externally Led Patient-Focused Drug Development (EL-PFDD) meeting on sarcoidosis with the U.S. Food and Drug Administration (FDA). This virtual event attracted nearly 350 sarcoidosis patients, caregivers, healthcare providers, industry partners, and advocates, coming together to address the critical unmet needs and challenges faced by those living with sarcoidosis.
Hosted by Mary McGowan, FSR Chief Executive Officer, and moderated by John Carlin, FSR Sarc Fighter Podcast host, this interactive EL-PFDD meeting provided a platform for patients to share firsthand experiences about living with sarcoidosis, the urgent need for new sarcoidosis-specific therapies, and the symptoms and challenges they would like future therapies to prioritize. Participants engaged through sharing patient and caregiver stories, participating in topic-specific panels, live polls, call-in segments, and written submissions, making their voices heard on key issues such as symptom management, serious side-effects of some current therapies, impact on daily quality of life, and specific treatment needs and preferences.
Dr. Lisa Maier, Professor and Chief of the Division of Environmental and Occupational Health Sciences at National Jewish Health, a member of FSR’s Scientific Advisory Board, and Founding Member of the FSR Global Sarcoidosis Clinic Alliance, provided an overview of the complexity of the disease and highlighted opportunities for essential future research.
“This meeting marks a critical milestone for sarcoidosis patients and caregivers,” said Mary McGowan, CEO of FSR. “The insights shared point to the urgent need for better therapies for the approximately 175,000 individuals nationally living with sarcoidosis and will support the creation of improved treatments that can enhance their quality of life.”
As a follow-up to this impactful event, FSR will publish a Voice of the Patient report early next year, summarizing all the written and oral testimonies provided by the sarcoidosis community. The report will offer vital insights into the priorities of the sarcoidosis community and provide a powerful resource for those working toward more effective treatments for this rare and complex disease.
FSR is grateful to the FDA and all the patients and community members who participated in the EL-PFDD, shared their stories, and called in or submitted comments. FSR also extends thanks to sponsors aTyr Pharma, Boehringer Ingelheim, Kinevant, Mallinckrodt Pharmaceuticals, and Xentria for their support of this event.
FSR will be accepting comments from sarcoidosis patients and caregivers through December 2, 2024, by filling out the comment form found on their website. The video recording of the live streamed event is also available on demand. Click here to find the comment form and recording.